FDA's Role in U.S. Leadership in Biomedical Innovation and Drug Development

i like a whole chicken

Sorry. Subcommittee will come to order. The chair recognizes himself for five minutes for an opening statement. Today's hearing will examine ways to maintain American leadership in biomedical innovation. This hearing is a great opportunity to learn more about what is working, where improvements can be made, and how Congress can ensure FDA continues to serve patients well, promote innovation, and maintain our nation's leadership in biomedicine. The United States has always been the global leader in biopharmaceutical research and development, and it's important that we remain leaders in this space. However, other countries are catching up to the United States. It is important for Congress to re-examine our current landscape to ensure we are staying up to date with the latest biopharmaceutical advancements, and examine what role Congress could play in making these processes more efficient. This administration has proposed efforts to do so, such as the recent launch of FDA's Operation Trailblazer, or trial blazer, excuse me, announced in June. This is an, you know, you rehearse this stuff, you still get it wrong. Trial blazer announced in June. This is an HHS wide effort that comes in response to the growing competitiveness of the global pharmaceutical landscape, which promotes cross-agency collaboration in support of research from the earliest stages of innovation. Operation trial blazer provides a set of in Initiatives and reforms to modernize and accelerate medical product development, aiming to remove the unnecessary hurdles and ambiguity that sometimes hinders patient access to innovative treatments. Streamlining clinical trial requirements, encouraging use of modern trial designs and real-world evidence where appropriate and reducing administrative burden can shorten development timelines and lower costs without compromising science or safety. These improvements have the potential to bring new treatments to patients faster, increase participation in clinical research, and make the United States even more attractive for investment in medical breakthroughs. It is important that we begin to have these conversations as this subcommittee looks ahead to FDA's user fee reauthorizations next year, especially as initiatives like Operation Trial Blazer demonstrate uh, that an approach to maintaining Ameri- America's competitive edge involving improving the efficiency and efficacy of drug development. For more than three decades, user fee programs have helped provide FDA with the resources to review innovative drugs and medical devices while maintaining the agency's rigorous standards for safety and effectiveness. Rather than relying solely on taxpayer dollars, user fees are paid by manufacturers that submit products for review, helping ensure the FDA has the scientific expertise and capacities necessary to keep pace with the rapidly advancing medical innovations. Ultimately, user fees serve to improve health outcomes for patients. Every day that a safe and effective therapy reaches the market is another day that patients living with cancer, rare diseases, or other serious conditions may have access to life-changing or even life-saving treatments. The predictability and performance goals established through the fee agreements have helped reduce review times, improve communication between FDA and product developers, and provide greater standardization through the review process. However, there is still more work that can be done and reauthorization is not limited to just extending the status quo. Reauthorization gives Congress the opportunity to explore and write legislation so we can continue to work to streamline regulatory processes, reduce unnecessary administrative burden, strengthen communication with product developers and ensure the agency has the tools it needs to make the best possible use of its resources. We can also continue to look for opportunities to modernize clinical trial net networks embedding them in routine care and expanding access to innovative treatments in rural areas. Maintaining America's position as the global leader in biomedical innovation requires a regulatory system that is both thorough and efficient, I am eager to look for ways that we can help ensure FDA can review drugs in a timely and predictable manner, not only to expand patient access to innovative treatments, but to also encourage companies to invest, develop, and manufacture domestically. Thank the witnesses for being here this morning, and I look forward to the discussion. I now recognize the subcommittee ranking member, uh, DeGette for her five minutes for an opening statement.

Thank you so much, Mister Chairman, I am really excited about this hearing today because I've um really worked a lot on biomedical research issues um over my career, and I have to tell you I think that there is I've never seen a more precarious time for the US leadership around the world in biomedical research. Um s- the FDA plays a critical role in regulating c- clinical trials. centering on the safety of trial participants and making sure that trials are asking the right questions. Over the past year and a half, FDA's staff has been decimated, political appointees have been making decisions that should be left to scientists, and industry and researchers have been left in the dark about what current agency policy actually is. The disastrous reign of Elon Musk and Doge last year, resulted in thirty five hundred jobs being eliminated at the fda willy-nilly without respect to what they were the most of any hhs agency low morale was pervasive at the agency and it showed fda shed senior leaders and rank and file scientists alike even the ones who weren't just summarily dismissed under commissioner mccary an agency famously insulated from political pressures became instead captured by them. He started the Commissioner's National Priority Voucher program, which provided incentives to companies with no predefined criteria, simply rewarding general alignment with administration policies. Without set public criteria, companies don't know what to focus on for the program, and the public doesn't know what, legitimate or not, is being rewarded. In another example, Vowing to Secretary Kennedy's anti-vaccine nonsense, FDA Commissioner McCary refused to even review an MRNA-based flu vaccine bef- before being forced to change course by public outcry. And who can forget FDA's initiation of a label change for Tylenol, indicating a lack of safety for use in pregnancy, contrary to established scientific evidence and against the advice of medical organizations. Simply because Secretary Kennedy wanted something to blame for increased documented rates of ADHD and autism. All of these decisions placed ideology above silence. FDA's inability and shoot-from-the-hip approach to policy also impacted industry. Commissioner McCary and FDA leadership made major policy announcements through journal articles, press releases, and even podcasts. circumventing FDA's policy-making processes and making it so, researchers did not know what the official policy was. FDA would even contradict its own past guidance as in the case of experimental gene therapy for Huntington's disease. FDA reversed direction, demanding a new trial for their product, even though advocates and scientists believe the new trial to be unethical. Luckily, with the evidence that Macquarie is gone, Things do seem to be slowly improving, and this is what I'm hearing from the community. The agency is now in the process of hiring thousands of staff to fill critical gaps, though it's hindered by the reputational damage to the agency from McCurry and Doge. There are responsible leaders atop the critical centers at FDA, and Acting Commissioner Kyle Diamantis has shown every indication that he will empower career experts to do their jobs, and protect American patients and get good markets to to products to market. In February, I sent a letter to FDA regarding the agency's failure to follow its own good guidance pra practices, announcing policy through journal articles, et cetera, et cetera, which is expressly prohibited in regulation. Last week, the agency finally responded, indicating the announcements we asked about were not in fact guidance and did not communicate

Without objection, so ordered.

So

I helped us as a turning point, back to transparency, to consistency, and to a focus on good science. This is particularly important for developers trying to get promising medicines into the clinic. And it's also important to our reputation in the world. so we can get clinical trials back into the US and stop this brain drain that we've seen going overseas. Innovative researchers will need to trust the FDA to treat their applications fairly, and without political favor or disfavor. As science, a- a- as as our capabilities evolve, so must our regulators. And so needless to say, Mister Chairman, I'm looking forward to hearing more from the witnesses on just how we can work with the agency to strengthen it so that we can get cures to patients quickly and safely as possible, and maintain our leadership in biotechnology and medicine as we go through the twenty-first century. And I yield back.

General, he yields back now, I recognize the Chairman of the full committee, Chairman Guthrie, for five minutes for his opening statement.

Good morning, and thank you, Chairman Griffiths, for holding this hearing on this important topic, and I can remember to get. Thank you for doing this. Additionally, Thank you to all of our witnesses for being here. Um, we appreciate your expertise as we examine how Congress and the FDA can strengthen America's leadership in biomedical innovation by supporting early stage drug development clinical research in domestic manufacturing. For decades, the United States has set the global standard, a gold standard for developing safe and effective medical products, which benefit patients around the world with Americans who gain earlier access to groundbreaking therapies. This is possible because innovators have chosen to develop them here first, but our leadership cannot be taken for granted. Other countries, particularly China, are making significant investments to attract bi- biotechnology companies, their clinical trials and domestic manufacturing. Our ability to develop and manufacture lifesaving medicines, vaccines, and other medical products here in the US depends on a strong domestic biotechnology sector in a regulatory environment that supports innovation while preserving FTA's standards for safety and effectiveness. Bringing new therapies to patients requires clinical trials that are e- efficient and modern leveraging new digital technologies and novel evidence, supported by sound science. This committee has a strong record of advancing those goals. Through the FTA user fee authorization, this committee has worked in a bipartisan way to modernize FTA's authorities improve regulatory predictability, and help Americans access new cures without compromising FDA's gold standard of review. Today's hearing is an opportunity to continue that work, learning from our witnesses where barriers to innovation remain, how FDA can further improve the efficiency and predictability of early stage drug development, and what legislative changes Congress should consider as we prepare for the next FDA user fee re- reauthorization cycle. Thank you, Mister Chairman, I appreciate our witnesses for being here, and I yield back.

Chairman yields back, now recognize the ranking member of the full committee, Representative Malone, for his five minutes for an opening statement.

Thank you, Chairman Griffith. I'm pleased that we're here to talk about advancing the US's role in drug development, particularly at a time when that role is threatened by the actions of the Trump administration. For a long time, the US has been the world leader in drug development. But this did not just happen. It was thanks to the cooperation between public health agencies, namely the Food and Drug Administration and the National Institutes of Health, and industry partners to ensure that our goal, standard research and regulatory review process is appropriately tailored to bring safe and effective products to Americans. Other countries have looked to our public health agencies as the gold standard leaders in research therapeutic innovation and development and regulatory oversight of medical product assessment. In order to remain that global leader, we need to preserve the strength of our public health infrastructure and investment. in research. However, we have heard many experts sound the alarm that the United States is at risk of losing its competitive edge in research and development to other countries, especially China. Unfortunately, actions we have seen from the Trump administration will only fuel the fire, in allowing other countries to overtake our position, threatening the advancement of lifesaving cures. The Trump administration has slashed billions of dollars in critical research funding. This funding was already congressionally approved, but Trump wasted no time. and abruptly and arbitrarily cutting the funding from research institutions and dismantling federal scientific agencies. Experts warn that these funding cuts de-stabilize the scientific research, that is the foundation of drug discovery. In many instances, these funds were slated to go towards scientific research that would develop basic research, that complements industry's applied research for drugs that they submit to FTA for approval. Without this initial public investment into NIH, the pipeline of drugs will slow. In fact, the Congressional Budget Office estimated that a ten percent cut to NIH's budget would result in twenty fewer lifesaving drugs approved over ten years. But the administration's threats to advancing drug development did not end there. Early last year, the Elon Musk led Doge indiscriminately and illegally cut thousands of federal employees from our public health institutions. It's still unclear which positions or programs were cut in the mass layoffs. specifically for this information for over a year and still have not received satisfactory responses. Just yesterday I asked the government accountability office whether FDA has violated the law in spending user fee dollars on unauthorized expenditures. And as we approach the user fee reauthorizations next year, I expect answers to those questions. Beyond these terminations, the administration has created upheaval at the FDA despite attempts to rehire, stay up, have shown that the fda applicant pools are not as robust as they used to be this is concerning given reports of brain drain with leading scientists leaving the united states to work for competing countries so i'm great we are having this hearing today but we cannot have a productive conversation about how best to move forward without honestly evaluating the real harm that the trump administration has done administration's actions threaten availability of new cures for for american patients And I look forward to hearing from our witnesses about what else is needed to address this critical problem as we begin the process of reauthorizing user fees. So thank you, Chairman Griffin, and I yield back.

Chairman yields back. We now conclude with member opening statements. The chair would like to remind members that pursuant to committee rules, all members opening statements will be made a part of the record. We want to thank our witnesses for taking their time to testify before the subcommittee today. Although it is not the practice of this subcommittee to swear in witnesses, I would remind our witnesses that knowingly making materially false statements to the legislative branches against the law under title eighteen section one zero zero one of the United States code you will have the opportunity to give an opening statement followed by questions from members and our witnesses today are uh doctor syntheny verst who is the president of design and delivery innovation r and d solutions at iqvi a miss susan uh winkler uh who is the executive officer of the reagan udall foundation for the fda and i will now yield to my friend and colleague, Ms. DeGette of Colorado, to introduce our next witness.

Thank you. Thank you, Mister Chairman, I'm so happy to introduce my friend, Doctor Aaron Kowalski, who's the Chief Executive Officer of Breakthrough T one D, the which is the leading global organization driving research advocacy and cures for type one diabetes. Many people on this committee know my daughter, Francesca, who was diagnosed with type one diabetes very early in my congressional career. And also, Doctor Kowalski and his brother both have it. Um, they frankly, when they were all diagnosed, um, everybody was saying there's a cure around the corner, no problem, and all these years have gone by. But all of us have shared a lifetime commitment to advancing treatments, therapies, and ultimately cures. And it has been extraordinary to see what has transpired. And this is why I'm so happy Doctor Kowalski is here, and it'll blow your mind. when he talks to you about some of this research but also the others here today what's happening in biomedical research in this country um and i know that under his leadership Breakthrough T one D continues to bring researchers industry regulators and advocates together and um i i just wanna say while i have the floor thank you to doctor Kowalski but thank you to all of our witnesses here today and everybody in the room uh we are on the cusp of exciting exciting discoveries that and this committee needs to partner with all of you in how we do it. Okay, I'll let you.

And I and I appreciate it, and I'd also appreciate all the witnesses being here today. The next witness to be introduced is uh, Doctor Thomas Wang, did I get it right? All right, he says I got it right. And is an Assistant Professor of of Medicine at Brigham and Women's Hospital and Harvard Medical School. And last but not least, Doctor Thomas Boice uh, is the is the Bloomberg Chair of in Global Health, Senior Fellow for International Economics, Law and Development, and Director of the Global Health Programme, Council on Foreign Relations. Uh, per committee custom, each witness will have the opportunity for a five-minute opening statement, followed by a round of questions from members. The light on the timer in front of you will turn from green to yellow when you have one minute left. It is now my honor and privilege to recognize our first witness, Doctor Synthony, Cynthia Vers. for her five minute opening statement.

Chairman Griffith, Ranking Member DeGette, and members of the subcommittee, thank you for the invitation to present to you today. Uh, I'm Doctor Cindy Verst, uh, President of R and D Design and Delivery Innovation for IQVIA, a leading clinical research, service and commercial insight provider to the life science industry. Our ninety thousand employees work every day uh to serve over a thousand biopharmaceutical sponsors from large to small, bringing medicines to patients faster. We connect globally with thousands of investigators to engage patients and deliver high quality clinical data to global regulators. IQVIA's vantage point today on the US competitiveness is both practical, pragmatic, um and data driven. We help sponsors design and execute global clinical trials And we see in real time why companies choose one country over another for their trials. So I'd like to share a few insights and and data points and recommendations. Firstly, the United States remains the most sought-after market for new medicines, and thus a critical destination for clinical trials. And although our data does confirm that China surpassed the US share of phase one innovative industry trials starts in twenty twenty one. At the end of twenty twenty five, China accounted for thirty five percent, in the US, twenty seven percent of phase one trial share. In looking more closely at the data, I'd like to share other insights. China's phase one growth is fueled by Chinese headquartered sponsors, that is, by China, for China. and where their home grown information uh relative to innovation is translating at scale from bench to the clinic. In fact, eighty-nine percent of phase one uh trial country uses by Chinese companies in twenty twenty five were actually in China, whereas China's inclusion as a country for US and EU headquartered companies in phase one trials were only about five percent each. Some US headquartered sponsors are indeed initiating early trials in China, but even more so in Australia, to confirm the asset's viability before coming to the US due to cost and speed to signaling advantages. Second, it is not the FDA review timelines, but rather the IND package preparation that drives X US first in human starts. Consistently, our sponsors, particularly emerging biopharma companies who drive over seventy-five percent of the global phase one starts cite time and resources to assemble the IND package itself as the barrier to initiate trials in the US. Many start their first trial XUS while concurrently building their full IND package, here in the US. And we appreciate that FDA and HHS are taking meaningful steps to address this through the operation trial blazer. Congress should consider encouraging the definition and use of fit for first in human minimum data sets based on complexity and risks to support the safe to proceed decisions and that would in essence speed the US starts without lowering the standards or compromising patient safety. Third, this fit-for-first in human focus at the IND application review will defer important conversations later in development. However, this will require more flexibility and capacity for FDA reviewers to provide that scientific advice as assets advance. Emerging biopharma companies and their investors place an an extremely high value on FDA feedback to inform their development decisions. and to attract further, often staged, investments beyond first in human. Without this support, we may certainly gain more first in human trials with speed to signal, but see costly rework and delays in later stages. Congress should consider supporting the FDA efforts to adapt rolling review practices and increase capacity for scientific advice, perhaps offering additional interactions for early stage companies who commit to start their trials firstly in the US. A modernized IND review framework with tailored first in human expectations, and an FDA empowered to provide more iterative scientific engagement to help our sponsors start more early phase trials here in the US while maintaining the patient protections and and data integra- integrity that make the FDA the global gold standard. And of course IQVIA is continuing to be committed to help the Congress, FDA, HHS, and of course uh other stakeholders to enhance this clinical research efficiency, and most importantly, helping our patients receive medicines faster. Thank you.

And lady yields back and I appreciate that. Now I recognize Miss Winkler her for her five minute opening statement.

Chairman Griffith, Ranking Member DeGette, and members of the subcommittee, thank you for the opportunity to testify today. I am Susan Winkler, Chief Executive Officer of the Reagan Udall Foundation for the FDA. I'm a pharmacist and an attorney by training and have observed drug development from uh many vantage points including as FDA's Chief of Staff under both, uh, Republican and Democratic commissioners. Part of my role there um involved in negotiating product safety agreements with China. The foundation is a nonprofit created by congress in two thousand seven to advance regulatory science and support fda s mission. So thank you for creating uh the organization. The insights I offer today uh result from some of our recent convening activity. Each produced dozens of specific stakeholder vetted recommendations, many of which offer insight into how the United States can make its own drug discovery and development system faster, clearer, and more capable. We brought together clinical trial sites, research organizations, FDA regulated industry, patient groups, and regulators to deliberate shared challenges with candor. The resulting recommendations align with much of Operation Trial Blazer. This is encouraging. When the people who run trials treat patients and generate evidence arrive on their own as the at the same conclusions as the regulatory agency, it's a strong signal that the reforms are sound and ready to implement. But more can be done. The goal is a stronger American system, and the surest way to lead is to be the world's best place to bring therapies from discovery to patients. The single most cited reform across our convenings was the need for clear, phase-appropriate FDA requirements and more opportunities to interact with and learn from the FDA. Specifically, adapting investigational new drug submission requirements according to the risk presented as Doctor Verst mentioned, and triaging the review can accelerate routine INDs and focus limited FDA resources to higher risk INDs to m- higher risk INDs uh to more novel interventions and emerging science. There is also benefit in informal interactions between sponsor and regulator, shared learning involving multiple parties, and simply using clearer language. For example, using the phrase clinical hold when FDA pauses an IND application before any human has received a dose can lead people to think that clinical work has stopped. It's a simple notice of additional information required would be phrasing that is clearer and less alarming both to patients and investors. FDA can clarify guidance and streamline review, and it should. But innovation is not advanced simply by refining regulations. FDA cannot build physical and human infrastructure and some needed reforms sit with other HHS divisions such as CMS. Congress, including this subcommittee, has a considerable role to play. I share three areas for subcommittee exploration. First, we lack a clear national picture of America's phase one trial capacity, which academic medical centers and units can safely activate first-in-human trials and how much of that capacity is at risk. How does that landscape compare with the independent for-profit sites that may focus more on healthy volunteer studies. The stakeholders we gathered recommend building a dedicated network of phase one capable sites including centers of excellence that apply policies and practices intended to speed time to trial such work requires federal resources and evaluation of the center of excellence concept. Second, where trial sites are located is uneven across the country. The number of trial sites is also declining under financial pressure. In addition to expanding opportunities, like the hub and spoke model for clinical trials, it's also an opportunity to re-imagine the screen ema screening mechanisms for clinical trial participation. Re-imagine them to be patient-centric rather than trial-centric. Further, harmonizing legal structures regarding permissible incentives and support for clinical trial participants which can be essential for offsetting the burden in research participation. would serve patients and innovation alike. Third, current contracting and budget negotiations between trial sponsors and research sites, simply take too long and add unneeded complexity to trial management. Congress could encourage encourage sponsors and trial sites to generate and use common processes and multi-sponsor multi-site templates to help shorten the time from IND approval to patient dosing. In closing, we need to create a regulatory system that keeps pace with scientific advances. HHS has charted a sound course, and what remains is squarely Congress's opportunity, including assessing and strengthening our national phase one capability, removing barriers, and where needed, amending the Food Drug and Cosmetic Act. Our foundation uh will continue to support this ecosystem. We need we stand ready to help.

Thank you very much. I now recognize Doctor Kowalski for his five minute opening statement.

Good morning, Chairman Guthrie, Ranking Member Pallone, Chairman Griffith, and Ranking Member DeGette and members of the subcommittee. And thank you, uh, Ranking Member DeGette, for your kind introduction and for all you've done for your daughter in our community. Thank you for the opportunity to test be testify before you today on a subject that is critical not only for our nation's competitiveness but for every American who struggles with chronic and serious illness. I'm Doctor Aaron Kowalski, Chief Executive Officer of Breakthrough T one D, formerly JDRF. the leading global research and advocacy not-for-profit organization for type one diabetes or as we say T one D. Our purpose is to make everyday life with type one diabetes better while driving towards cures. We do this by investing in promising research, educating the public, and working with the government, like this esteemed committee, to address critical issues in the T one D community. I'm a scientist by training, but I also live with type one diabetes as does my brother Steve. So I know firsthand the constant challenges of managing this disease, as well as the promising science on the horizon. I've also worked with FDA for nearly twenty years, and I can say with pride that my family and our broad type one diabetes community has benefited directly from American innovation and FDA's standards. There's work to do, but I want to commend the FDA's new leadership. In just a short time, they've shown a real commitment to becoming forward-leaning partner in innovation, that the T one D community needs. Type one diabetes is an autoimmune disease that can affect uh anyone at any age. Once you have type one diabetes, you rely upon insulin for the rest of your life. Without insulin, type one diabetes is fatal. Insulin, though, is not a cure for type one diabetes. The disease is unrelenting to manage, even with the amazing progress that we've made uh over the last number of years. It's twenty four seven, three sixty five, and it can have serious complications and consequences. High blood sugar can lead to diabetic complications such as heart disease, strokes, amputations, and blindness. But, on the flip side, too much insulin at the wrong time can lead to hypoglycemia or low blood sugar, which can be life-threatening in just minutes. So Breakthrough TND has invested over two and a half billion dollars in type one diabetes research to try to solve these problems. But we can't cure type one diabetes alone. We're so grateful for the bipartisan leadership of representatives DeGette and Bill Arrakis and the strong support of this committee for continuing support of T one D research and the special diabetes program. America has a long and proud history of leading T one D research innovation and treatments. But the rest of the world is catching up. I saw this firsthand during recent visits to China in December. in Australia in April. That said, our global uh leadership in T one D innovation is ours to lose. America is well equipped to continue to lead. NIH research funding and FDA regulatory approvals are the linchpins to that success. It would be a tragedy to fall behind because we failed to keep pace with the speed of innovation. But that's begun to happen. Transplantation of deceased donor islet cells is an example of this. These transplants have been safely performed abroad for decades, benefiting benefiting select patients who qualify. But these same deceased donor islets here in the United States remain largely inaccessible for research and transplantation because FDA regulates them as drugs instead of organs. Clinical clinical trials are another challenge. C-peptide, for example, is the best, simplest measure of your body's insulin production. Yet in clinical trials, S- FDA hasn't granted full approval for using C-peptide as an endpoint. Antiquated requirements needlessly lo- prolong and complicate clinical trials and can deter innovation and investment. I commend the recently announced trial blazer pilot aimed at monitor- modernizing clinical trials. Breakthrough T one D has also stepped in to help by publishing road maps for T one D cell therapies, giving more clarity to navigate FDA pathways. A robust FDA, one that is stable and well-resourced, consistent and transparent, modern in its clinical trial and endpoint policies, and that incorporates patient perspectives is essential to our global global leadership in biopharmaceutical innovation and manufacturing. An FDA with these qualities will be ready for the next wave of T one D innovation, which is already right here at our doorstep. Cell therapies are already resulting in insulin independence. One pivotal trial is happening in the US right now. With the right approach, we can be the first country to approve and manufacture scalable cures for T one D. My message is simple. We have the capacity to end type one diabetes and many other chronic and debilitating illnesses if FDA adapts and modernizes to keep pace with scientific progress. Thank you again for inviting me to testify and I look forward to questions.

Thank you and now recognize uh Doctor Wang for his five minute opening statement.

Chairman Griffith, the ranking member to get, Chairman Guthrie, ranking member Pallone, and members of the subcommittee. Thank you very much for the opportunity to testify. My name is Thomas Wang. I'm a physician and health policy researcher at Harvard Medical School and the Brigham and Women's Hospital in Boston where I lead the Cancer Innovation and Regulation Initiative. My research focuses on the regulation, clinical development, and payment of new medicines, medical devices, and other interventions in cancer care. All views here are my own only. The United States' leadership in biomedical innovation is built on strong and stable investments in research. Nearly every drug entering the US market is linked to NIH-supported science. Early stage clinical testing is at the leading edge of this innovation. These studies have special regulatory and signe- scientific significance for three main reasons. First, early stage studies of new therapies are often located, although not always, where the scientific discovery occurs. Second, first in human studies have heightened safety obligations because they traditionally test healthy volunteers. In oncology, early stage trials um may enroll affected patients and can serve as a pivotal study supporting regulatory approval. And third, unforeseen safety issues can and do occur when preclinical models fail to predict actual human response. So where early stage trials are conducted matters both for the safety of our patients and for innovation leadership more broadly. Our research reviewed the phase one studies and other early stage testing for novel cancer drugs approved by the FDA. We found that the share of these studies with at least one US trial site declined substantially over the past seven years. During this time, early stage trial activity in China increased. As recently as twenty eighteen, cancer drug approvals did not involve any first in human study site in China. Since then, the share of early stage cancer trials with any Chinese trial site nearly tripled and the share of first in human studies against cancer with any Chinese site doubled. Our research indicates that rising Chinese contributions to early stage clinical development now extend to the first in human and pivotal early stage studies that underpin FTA approval. Our patients benefit from access to the world's best scientific discoveries that will ultimately generate safe, effective, and affordable treatments for their conditions. We should welcome competition in the global research enterprise, but such competition has to be fair, transparent, and ethically sound. Recently the FDA has raised questions about data from single country and non-U.S. clinical trials that may not be generalizable to patients here. For example, these studies may use a suboptimal control group that does not reflect standard of care in the U.S. The entire biomedical enterprise suffers when clinical research moves to sites with inferior standards for data integrity, safety and ethical protection of participants. As this subcommittee looks forward to upcoming reauthorization of user fee agreements, I want to share three policy proposals that could support our nation's leadership. First, improved transparency can accelerate early stage clinical testing and ensure efficient regulatory review. This helps sponsors avoid similar mistakes and learn from past development failures. The FTA has recently launched an initiative provide more information on why new drugs are not approved, including publicly releasing complete response letters. Congress should provide explicit statutory backing for the FTA's transparency initiative, and extend it to include reasons for halted early stage trials, and a summary of the steps that are taken to resolve these suspensions. Second, Congress should explore ways to increase global standards for clinical research that protects both patients and US competitiveness. The FTA remains the global model of regulatory approval of new therapies, and the US is the most lucrative market for these products, so the agency's priorities shape the trajectory of development programs around the world Congress could examine requiring that a minimum percentage of non-US trial sites undergo FTA inspection third and finally as part of the FTA's operation trial blazer the FTA could pilot coordinate submission and review of investigational new drug and an investigational device exemption applications with international partners which allow new drugs and devices to begin clinical testing Ultimately, any durable effort to promote US clinical development requires investments in public support for foundational research and adequate staffing at the FDA. Thank you for the invitation. I welcome any questions.

Thank you, and now recognize uh, Mister Boyke for his five minute opening statement.

Chairman Griffith, ranking member to get, distinguished members of the subcommittee, thank you for this opportunity to testify today. I'm Tom Boecke, the Bloomberg Chair in Global Health at the Council on Foreign Relations. I recently co-led a year-long analysis with bio-pharmaceutical specialists, China scholars, industrial policy experts who identified the true scope of US dependence on China in the bio-pharmaceutical sector, and to recommend policies to reduce it. My remarks today build on that report, focusing on first the consequences for patients, public health and biosecurity if the US cedes its global leadership in drug discovery and development to China. And second, how Congress and FDA can streamline the early stage clinical development process without undermining patients' timely access to safe and effective medicines. Let's start with how drug discovery is shifting to China and the potential consequences. China now offers pathways for conducting first in human trials that are three to five times faster and one third to half the cost of the US investigational new drug process. Chinese trials are supported by a dense ecosystem of contract research, development and manufacturing organizations, hospitals capable of enrolling hundreds of treatment-naive patients at a single site, heavy state subsidies, and a permissive scientific ethics system. The ability to conduct first in human trials quickly and at low cost enables Chinese biotech firms to determine which compounds hit their tar biological targets to refine them leapfrog more slowly moving US competitors, and to attract licensing vital licensing deals. The results speak for themselves. China's share of global clinical trials starts has risen from one percent in two thousand nine to thirty percent in twenty twenty four, rivaling the US share. By one estimate, one third of the pharmaceutical industry's global licensing spending in twenty twenty five, involved assets originating in China. If this trend continues, it threatens to reallocate the early stage bio-pharmaceutical pipeline away from US startups, and hollowing the American innovation ecosystem. The risk of that will be threefold. First, there's a biosecurity risk if China can create strategic and economic leverage by choking off supplies of innovative medicines on which US patients and hospitals rely. China has already weaponized rare earth critical minerals and agricultural products, targeted pharmaceutical supply chains in Japan and India, and threatened to do so more broadly. Second, there is a risk to to public health if the US capacity to do innovative, uh, drug development diminishes, and we cannot rapidly produce effective countermeasures in a crisis as occurred um in uh covid-nineteen. Third, there's a risk to patient safety because FDA cannot effectively oversee the level of clinical trial activity now occurring in China, which means US patients and health care providers may not be able to fully rely on the resulting data. One recent study found that just twenty GCP inspections, less than one percent, of FDA's GCP inspections between twenty sixteen and twenty twenty-three occurred in China while ninety-three percent of those inspections happened in the US. So what should Congress and the FDA do? First, we need to make our own system better by adopting the operational features of other well-regulated nations that have modernized early clinical trial oversight without sacrificing patient safety or scientific integrity. Congress and the FDA should authorize a narrow expedited pathway for well-characterized drug platforms and lower risk for human studies, such as Australia's pioneered. It should combine that with a program that invests in dedicated university and hospital phase one sites as Norway has, that use public-private partnerships to inform trial design, central IRBs, and ensure diverse trial subject populations that draw from rural areas too. Second, FDA should reduce dependence on Chinese contract bio-manufacturers by making it easier for US firms to adopt advanced manufacturing technologies and AI-driven process innovations, and by being better prepared to authorize temporary importation of essential medicines should China seek to cut off supplies to them. Third, Congress needs to continue to invest in the enabling environment that makes the US biomedical industry thrive. including our federal agencies and especially FDA and NIH. Re-hiring is occurring, but FDA staffing is down from twenty twenty five. The agency has lost real experience and expertise. Adding a user feed to enhance the number and quality of GCP inspections for IND submissions with foreign clinical trial data would help boost FDA's inspection capacity. In conclusion, the same dynamic that hollowed out US generic drug manufacturing is now playing out in biotechnology. but at an earlier stage when FTA and congressional intervention would be effective and is thus more urgent. The time to act is now. I thank the subcommittee the subcommittee for the opportunity to testify and stand ready to answer any questions.

I thank you and all the witnesses. We will now begin questioning. I'd ask that members not begin a new question. Maybe underline that, not begin a new question with about ten or fifteen seconds to go. Because that would hopefully move us along a little quicker. I would encourage members to submit written questions for the rec to for the record. And I and I will tell everybody, I've already leaned back and told my team a couple of questions for the record, cuz I know I can't get to them all that I wanna get to, but you all presented a lot of interesting ideas for us to consider. I now recognize myself for five minutes and I'm gonna start with Doctor Verst. Doctor Verst, the uh appreciate you talking about the uh IND and the FDA has recently

I I do think that it will take the collective uh ecosystem to be leaning in to ensure the success, but I think it is a a terrific beginning in recognizing the problems and the solutioning that will be required in order to achieve that.

All right, now one of the things you mentioned that I've been toying with in my own mind, and you just touched on it tangentially, so maybe I heard what I wanted to hear, was you mentioned risk. And it has bothered me that when you have low risk, sometimes FDA wants everything done, you know, according to their standard process. But if you have low risk, should we be creating a second standard that says, you know, it's not a it d- we don't know if it works for what it says it's gonna work for, but the risk is so low, if you wanna try it,

Uh, I I do believe that is a terrific question. And I think it's really more risk-based in our approaches. And we've got many examples of that within the clinical development arena, supported by the FDA, for instance, risk-based quality monitoring. And I think having the ability to be more, if you will, uh weighted, And of course that is adaptive with regard to our approaches, and and being very focused on that risk-based profile will enable us to speed uh along processes and reviews, but not jeopardizing patient safety nor data integrity along the way.

A- and and that's our concern, or my concern as well. But do they need a nudge from Congress? I'll get an answer from you later. And I'll do a written question on that one. Ms. Winkler? You highlighted how trial capacity uh is currently unevenly distributed across the country with many patients living a long way from the trial site well that's that you welcome to my district.

Absolutely.

I'm a I'm a long way from any place that does that. I mean people say, well why don't you just go to Charlottesville? Well, in parts of my district it's not Charlottesville you'd be looking at, you'd be looking at Cleveland, cuz you're closer to Cleveland than you are to Charlottesville, even though you're in the state of Virginia. Um so what can we do to get more sites involved in the clinical trials and I'm specifically wanna look at community health centers, particularly federal qualified ones. What's the

One of, yeah, one of the ways to um en- enhance access and so to get into areas where they aren't today is to um make better use of and clarify the rules for what's called a hub and a spoke model right so that you have the really important um but uh paperwork heavy um responsibilities of the hub thinking about this is how we run trial sites and they hold some of that responsibility for the so that in communities you could have clinical trials available through the clinicians in the community and they'll operate according to the rules and regulations, you know, and the oversight of the hub, but it it helps um structure the the mechanism so that you aren't trying to recreate in uh a local area where you might have just a couple of physicians who who could do some things let's empower them to do those some things in collaboration with with a hub.

And I would be correct that telemedicine will help with that. Yes?

Yes, there are definitely some opportunities in remote monitoring and telehealth.

Absolutely. Thanks. Doctor Wang, your thoughts on how we can get more sites like community health centers involved in these um early stage clinical trials.

Thank you for the question. Um, what we know from the Australian experience and from um uh from other countries, is that public investment in our health hospitals and health systems um is critical to uh in improving the capacity that they have in running clinical trials and having a centralized network um of these institutions that could be centers of excellence could speak to kind of your concerns today.

All right, if you can do it in thirty s- seconds, Ms. Winkler, uh how can using real-world evidence or natural history studies and trial designs help new treatments for rare diseases?

Uh it could be very helpful in helping to structure the trials as well as after perhaps a product is available to do um follow-up and make sure that we have the performance that was predicted in the initial piece. So that that data both before um submission and approval and after approval, we need to make better use of all of that information.

I appreciate it.

It's rare data.

You did great. I now yield uh back and recognize Ms. DeGette, ranking member of the subcommittee for her five minutes.

Thank you so much, Mr. Chairman. Um, and thanks to everybody for their testimony. Um, really it's it's very rare that we see people from all aspects of the uh industry sort of rowing together in the same direction, and I think I think we know what we need to do. Um, the federal government uh and and the FDA in particular really do have an immense impact on biomedical research. and with grants and other collaborations. But, um, as all of you know, our resources are not unlimited, so we have to fund high-quality basic research and impactful public health projects. Um, Doctor Kowalski, I wonder if you can talk to us about the importance of merit-based peer review process.

Well, merit-based peer review is fundamental to making good decisions that are unbiased, that have input from expert scientists and, um, and are aligned with the unmet needs in science to drive towards clinical benefits. So it's fundamental to um everything we do in science.

So OMB recently proposed a rule that dramatically changes how the federal government would evaluate federal funding and how grantees can use the federal dollars. What would the impact of that rule on the peer uh review process be?

i i think uh any scientist uh would tell you that we need to keep politics out of science. Uh it's critical that uh the science is evaluated on the merits, on the evidence, and that we drive forward um without the influence of um um politics.

And and um uh so you think that the proposed rule would really r- adversely impact the peer review process?

Uh, I think so, and we've uh signed on with a coalition to uh lodge our um feelings about this issue.

Okay. Um, now, it international research collaborations have made important contributions to the science of type one diabetes. Isn't that correct?

Uh, absolutely.

And how would that proposal treat those international collaborations?

Well, we've seen already in a number of groups that we fund, uh, uh, barriers be put up to some of these collaborations. For example, I just came from a meeting where we funded an international study on pregnancy and diabetes. Those investigators often rely on uh agreements through NIH multi-center uh uh study grants. Uh, and those barriers we absolutely do not want to see happen.

Okay. Um, can you just briefly tell us about the TEDdie study?

So this is a an amazing study, it's one of the largest databases currently at NI NIH that's aiming to identify the causation of type one diabetes. Type one diabetes has a genetic component, but even on identical twin who gets type one, it's only about a fifty percent risk the other one will get it, which says there's something in the environment that is causing type one. When we look at the use of AI in the future, I really believe combination of genetics and this environmental data from TEDdy will really help us understand what's causing temporary abuse.

So so one of the per provisions of the proposed rule would let political appointees terminate grant awards. So that would effect effectively kill projects at any time for any reason. What would happen if the TEDdy, this huge TEDdy uh collaboration that you're talking about were cut off halfway through because maybe a political appointee didn't like the word word environmental.

Uh, the the it would be a a a travesty.

It would it would totally destroy the results of that study.

We w- I I mean we have a we we don't n- We've certainly benefited from Teddy uh with a number of seminal learnings, but the ultimate goal of understanding the genesis of type one diabetes is not there yet,

Right.

and that is one of the most important studies that we would have let's get there.

Right. Doctor Hoang, I wanna ask you quickly, what kind of work does FDA fund externally?

The FTA has a lot of important grant mechanisms, um, specifically in the Office of Orphan Products. FTA grants to rare disease clinical trials, including those for ALS, have supported new product approvals, and all of that research, um, is at risk.

And in your view, if this rule I've been talking about were implemented, would that undercut that external work?

I would have concerns that I would.

Doctor Ho- uh, Kowalski and Hoang, do you think, and and I'm just gonna ask you, Uh, do you believe the proposed rule should be withdrawn? I'll start with you, Doctor Quinn.

Uh, I'm still reviewing the proposed rule, but as written, I have serious concerns before it's implemented.

And Doctor Kowalski.

I think we share that those concerns.

Okay, thanks. Thank you so much, Mister Chairman. I yield back.

General, he yields back now, recognizes the Chairman of the full committee, Mister Guthrie of Kentucky, for his five minutes of questions.

Thank you very much. Thank you. So, so Miss Winkler, the Reagan-Udall Foundation has extensive experience convening stakeholders to identify opportunities to accelerate

Alright. Okay.

drug development, particularly in areas where there are few treatments such as uh psychiatric substance use disorders particularly psychedelics uh who we're referring to. As the administration takes action on accelerating development and access to therapies for these types of conditions, where do you see the greatest opportunity for FDA and external stakeholders to work together to bring safe and effective treatments?

Uh, so there's a few opportunities, um, but in particular assuring that that um that the endpoints that we're pursuing, so what the treatments what the treatments intend to uh to affect, match what patients are looking for. So that's one of the components. And then there's also uh the important conversation between the product developers and the regulator for what is, what should be measured and how um do those clinical trials need to be conducted. Uh, there certainly have been a number of those conversations that have taken place. And now, uh, as that work continues, it'll be important to share the learnings as some products move through that process, what can other developers learn?

Cuz one of the issues like with psychedelics, you can't really do a placebo trial, cuz obviously you, if you've taken, it's placebo psychedelic, you know you're taking the placebo psychedelic.

Right. That is one of the challenges. So FDA has a guidance on that to try and and help parse out, you know, how the trial should be done so that they can prepare it.

They just did that in the last couple of days. You think that's gonna be effective, their their guidance? You like the guidance they put out the last couple of days on that?

Uh, so we have had the privilege to convene some of the conversations that contributed to that. So we don't take a position on the guidance, but we I'm aware that there were constructive conversations that helped, um, uh, make sure that it was reflecting the needs of the community.

Cuz we have a we have a a number of colleagues, uh, in Congress who were combat veterans. and have had this this treatment and have lots of friends and they've talked of this treatment and feel it's really effective and we need to figure hopefully we can figure out a pathway if it is effective to find it's effective and give access if so. So thank thanks. So uh Doctor Kowalski in Breakthrough T one D uh you've worked closely with FTA and product developers events, all the innovative clinical designs and trials uh with endpoint development for therapies and ultimately the benefit patients would uh, type one diabetes. Can you describe how early collaboration between the FDA sponsors and patient organization contributed to that success, and what lessons can be applied more broadly uh to accelerate development?

Yeah, thank you, Mister Chairman. This is such an important piece, I think, of the progress we've seen in type one diabetes. It has been a colla- a collaboration between regulators and patient advocacy groups like ours. You know, there's a hesitancy often in medicine to take risk. But I think one of the uh uh uh under underappreciated things is living with type one diabetes is risky. And we certainly saw uh significant progress made back out when I worked uh quite a bit on diabetes devices, for example, with collaboration, understanding risk benefit, safety, and efficacy, and driving together to doing the right trials and having the right pathways to come through FDA.

Okay, thank you. And so, Doctor Verst, we've heard increasing concerns that other countries are reducing the time it takes to activate clinical trials and role patients, making them more attractive locations for early stage development. What are the potential risk if we as Americans fail to remain competitive with clinical trials? And what, particularly what, so the risk and what we can do to have the greatest impact on ensuring America remains the preferred location.

Mister Chairman, that that's a terrific question. I think the largest risk is not getting on the other side of approvability. better medications to our patients faster, the largest risk. I think what we can do is actually putting more innovation at the top of the funnel. And that is what is truly at risk here, uh from a China uh perspective. And that is more translational, if you will, medicine and science with more assets, drug discovery occurring, you know, early in the process. And then as I mentioned in my uh remarks, simplifying and importantly accelerating and doing more of that adaptive approach, more of the risk ap- approach to actually hasten the process. And then finally, clarification. Especially with biotech pharma companies, they need that early engagement that you alluded to earlier, uh, much more preemptively, even in the pre-IND uh phase, and being more predictive and reliable, especially because of the backing of investors. They really encourage that FDA feedback and insights.

We need to have the risk approach because uh, I mean, if you're just trying to cure the sniffles, you wanna make sure you wanna a lot of tests to make sure there's no side effects. But that's what the present is uh for then the first term, and now push right to try. Some people are in a situation where this is their only hope, and this is something that's gonna cost them their lives. And so We and then with uh or suicide, that's what our some of my colleagues talk about with some of the treatments that I mentioned earlier. So we are focused on it and we look forward to working with you and I yield back.

Okay, the gentleman yields back and I now recognize the ranking member of the full committee, Mister Polon, for his five minutes of questions.

Thank you, Chairwoman. I mentioned in my opening statement we need to ensure there's a robust research infrastructure in the US, something that has been seriously threatened by the Trump administration. In May, the Trump administration took another harmful step to decimate our public research, when OMB overhauled the federal grant rules and gave political leadership final authority over discretionary award decisions. This politicized the science and threatens the independence of scientific research in the United States. On Monday, a group of fifty-seven organizations representing patients, caregivers, and families that rely on drug discovery wrote to congressional leadership, warning of, I quote, " profound implications on biomedical research" and the future of treatments and cures in the united states and i'd like to uh ask unanimous consent madam chair to uh uh introduce this into the record if you will

yeah without objection so

jim thank you so every democrat on this subcommittee wrote to the omb director earlier this week to rescind this uh politicizing rule i wanted to ask doctor hoang your testimony speaks to the vital role of us public investment in biomedical innovation why is it important to have an ecosystem that is supportive of this investment, including on foundational research? Your testimony notes that the US has historically been the engine for biomedical innovation however your research now shows the growth in Beijing China's action in this space. So, basically let me ask you first about the the vital role and then I have a second question, doctor.

So, multiple studies have shown that virtually every new drug that enters the

Well, thank you. But what evidence have you seen regarding the rising

Mm.

Beijing Chinese contributions to early stage clinical development?

So what our study has shown in in collaboration and in um uh knowledge of other studies that are going on as well is that um it's not declining US productivity in science science but rather increasing Chinese investments in early stage research and in the trial networks that we're lacking here and what our study showed is that in the important studies that underlie US cancer drug approvals now a significant portion of them are being done in china and being transported here

it's also part of this brain drain you know we keep reading about um scientists that would normally um, you know, either come to the United States or even American scientists that are leaving the country uh to go elsewhere, China, uh Western Europe, elsewhere. How eh how is that contributing to the eh to the drug development or or lactate over slowing here in the US?

I think Congress has an important role in making the US continue to be a leading destination for the world's best scientists, and I call upon this committee to help that.

Alright, thank you. I'm also concerned that we're undermining the impact of uh or underestimating I should say the impact of our dependence on Beijing. Uh so let me ask Mister Boyki, in the report you published that you note that we need quote " a combination of measures to improve US innovative capacity in the biotech space combined with sensible security restrictions on Chinese firms." So what does your research show as a reason for clinical trial activity from promising interventions shifting away from the united states to to beijing china

thank you for the uh excellent question so uh part of it is a decline in us competitiveness not just vis-a-vis china for early stage clinical trials but also australia and other markets as well it is a cumbersome burdensome process under the ind uh uh approach and that has um helped encourage activity go abroad But make no mistake, uh, China's rise in this area is not just simply a matter of market forces. It is decades of coordinated state intervention and investment. And while it's critical, as we the many of the witnesses have discussed here, to invest in making the US more competitive, we need to acknowledge that, uh, FDA acceptance of foreign clinical trial data depends on its, uh, compliance with, uh, ch- good clinical practices, that it's conducted under uh independent ethics review, and the ability of fda to inspect, and we cannot maintain that standard uh for the amount of activity in china. uh We we have to have increased scrutiny over those trials and increased resources for fda to do that.

Thank you, thank you very much. Thank you, Madam Chair.

Thank you. This gentleman yields back, and I recognize myself for five minutes. Um, You know, we've got two pharmacists on the panel and as a pharmacist, I've spent uh my career helping patients, you know, benefit from innovative medicines and uh but innovation only matters if patients can safely access it. So I'll start with you, Doctor Verst. Uh, your testimony notes that Chinese companies overwhelmingly conduct their phase one uh work inside China. Should Congress view this primarily as a regulatory challenge or as industrial competitiveness challenge?

I think it could be both, uh in terms of the regulatory challenges and and ensuring for instance, um at the end of the day we talked about representativeness, so increasingly we're seeing more global uh uh

Yep.

trials being executed outside of China by Chinese companies.

Mm-hmm.

And and accordingly, you know, we pay very close attention provisioning services um to help them understand the the regulatory implications at the fda level. For instance, representativeness, which is being so critical in the data application.

Yeah.

You know, and and I also think, you know, from just a competitiveness uh perspective, uh that this phase one is quite candidly close on the heels is phase two. Right now the uh comparison of uh phase two trials in China as compared to the US

Yeah.

is increasingly uh in becoming comparable. So I I think both uh threats.

Yeah. OK. Uh, Ms. Winkler, stakeholders often tell us that one FDA review division may expect something entirely different from another so how important is it uh to get greater consistency across the FDA review divisions for encouraging companies to keep early stage development here in the United States?

So not only is the consistency important,

But when sometimes there's a rationale for it in that the review division is looking at a a different organ system,

Mm-hmm.

a different impact.

Yeah.

And so what's important there is for the agency to share the scientific rationale for the differences, not only with that sponsor, but could that information be available more broadly so that the um inconsistencies are explained and provided in cont in context.

I got you. Doctor Wang. You found that China's role in early stage oncology trials has grown substantially. I'm a compounding pharmacist, so when we can't I've said this for forty years. There's a problem with APIs when you have them do what they're doing with everything, drop the price corner of the market, and then we're the ones that pay the price when you can't get those drugs. Should Congress be more concerned about losing scientific leadership or about relying on foreign generated clinical data that FDA may have limited

Thank you. I think both of those should be the focus of this congress. Um and specifically on the latter, um as we heard from other members of this panel, the FTA does not inspect um a significant number of trial sites in China. And what we do know from the literature is that many of these sites have inferior standards uh for data integrity and for safety.

Yeah.

And so we should empower the FTA and and and substantially appropriate funds to do so, uh to do to do those inspections.

Yeah. You know, I've talked to drug companies recently and they, they have been to China, they say their industrial uh facilities are are very top of the line, and they have an equivalent like the FDA, but I'm like, how do we know that if we don't go inspect those? And it's there's lots of questions there. Uh, Mister Boelcke, you argue that dependence on China for drug development creates both economic and national security risk, and I agree. Can you explain why manufacturing domestic early stage clinical research

Thank you for the excellent question. So we have seen uh another space trying to exercise a willingness to leverage dependence on its supply chains and its system to gain economic or strategic advantage over other nations. Uh, this is not a fanciful possibility in the pharmaceutical space. They have done this with Japan. already in terms of restricting access to dual use compounds that uh support pharmaceutical manufacturing they have uh undermined india's attempt to develop supply chain resilience in the active pharmaceutical ingredient

yeah

and key starting material space they easily could do so in this context, so it puts us at real risk.

yeah absolutely agree with everything uh one point that stood out in your testimony sir was the limited number of fda inspections conducted in China and I have eleven seconds left. But if FDA cannot routinely inspect foreign trial sites, how confident should Americans be in relying heavily on that data? It's just I I I don't know, this is a problem, and I I came to Congress to fix this, and we're gonna work on this. A lot of the suggestions you all have had we'll take back to the FDA, so thank you for being here. I go back and now I recognize my friend from California, Dr. Ruiz, for his five minutes.

Thank you. Thank you so much. Um, we must first and foremost ensure the effectiveness and the safety of new innovations during the clinical trial process. That's that's ground zero. Uh, and as an emergency physician, I'm concerned about the potential effects that the lack of participation in clinical trials may have on the effectiveness of new medical treatments and therapies for populations that are underrepresented in these clinical trials. OK? Uh, so many barriers prevent patients from participating in clinical trials, especially individuals living in rural and underserved areas, working class, poor uh, families. There are considerable financial considerations, Can someone afford taking time off of work to participate in the trial? Uh, can they afford the transportation to the trial site and co-pays for the follow-up, uh, uh, in the clinical trial visits and afterwards to the doctor? Uh, and um, there are also basic geographic considerations. Uh, if you're living in a rural community like many of my constituents, you might not be able to drive two hours to the nearest academic medical center to participate in their clinical trials. Or you and your doctor might not even know about the trials and the opportunities that uh many patients may welcome, uh especially for uh end-stage illnesses in the first place. Uh, so these are barriers that we can and should address. Uh, that's why I introduced the bipartisan Clinical Trial Modernization Act, HRS three five two one with Representative Flueger from Texas. Imagine that, a Democrat from California working with a Republican from Texas doing the right thing. Isn't that how this should work? Right. So, um, this bill would improve participation in clinical trials by addressing economic barriers and helping bring trials to underserved areas where the patients are. For the reasons I mentioned earlier, participant enrollment and retention remain one of the biggest challenges in conducting clinical trials. Community health centers are trusted providers to fifty-two million patients in underserved communities rural areas across the nation. Given their broad reach, CHC's can bring research closer to where people live and receive care and greatly expand delivery of clinical research to underserved population. Uh, Miss Winkler, in your written testimony you talk about a patient center approach. So what role could community-based providers play in improving patient recruitment and accelerating research and how would bringing clinical trials into the community care settings change the way patients access innovative therapies?

So one of the challenges in clinical trial recruitment is that that patients m- may not be asked. And they're not asked because their local community provider does not have the information about what trial might be appropriate, might be helpful for them. So, um, re-imagining and having a patient-centric system would in in certain disease areas, uh, you know, deploy against, um, a community-based network, providing them with the information about what trials are available and what are the criteria for the patients so that then you could uh look at the patient in, you know, from your community health center and say here are the options and evaluate those right there rather than needing a referral to somewhere somewhere else. So it's a combination of um thinking about the patient and what trials might be appropriate,

Yeah.

and making sure that that information and the ability to do research is there local with the patient.

Yeah.

You know, currently clinical trials remain concentrated in a small number of academic medical centers, which slows recruitment, narrows the range of data that trial sponsors can generate, and leaves millions of patients particularly those living in rural and underserved communities without access to these uh to these trials to these drugs. Um, Doctor Verst, what can this committee do to encourage greater participation uh by community-based providers such as CHDs and clinical research?

Yeah, it's a ter- terrific question. And I I do think it's another one of these multi-pronged approaches, no silver bullet per se. I think in ensuring that the community physicians uh associated with healthcare systems are being encouraged and supported uh with regard to even referral opportunities. Now in order for that to be accomplished, trying to reduce the burden during this process, and of course incentivizing as as appropriate, and using technologies that actually e-sourcing for instance EMR and EDC systems being interconnected to simplify uh the process and identifying the right patient for the right clinical trial at the right time on his or her patient journey and and of course this all is working across the healthcare ecosystem in order to to be a reality.

And and if I may with just one second point of privilege, uh, Doctor Huang, welcome, I I went to Harvard Medical School, did a lot of work at Brigham and Women's Hospital throughout my years of training, including my fellowship with the Harvard Humanitarian Initiative. Welcome on board.

Thanks gentlemen for yielding back and now recognize Doctor Joyce for his five minutes of questioning.

Thank you, Mister Chairman, for holding this important hearing and to our panel for being here today. The United States leads the world in medical innovation. It is critical that the FDA is operating efficiently to ensure that patients in the US have access to the best and again the most innovative treatments that are available. There is now more momentum than ever to innovate in the chronic disease space. Ongoing research has found better ways to screen for, diagnose, and delay the onset of symptoms in diseases like type one diabetes. A delayed diagnosis of T one D drastically increases medical, financial, and emotional burdens. Frequently, patients go undiagnosed until they land in the emergency room with a diagnosis of diabetic ketoacidosis. a life-threatening complication of disease that can lead to lengthy ICU stays and significant long-term health consequences. The status quo is bad for patient outcomes and it puts unnecessary burdens and costs on the patient and on our healthcare system. Recently, Adam Schefter visited Washington DC and discussed how his wife's late diagnosis of T one D influenced him to come to Washington to advocate for earlier diagnoses. He shared with us how despite an adult diagnosis and early challenges adapting to the disease his wife was now able to successfully manage the disease for over twenty years with the help of an insulin pump and with the help of continuous glucose monitoring systems to address these issues and to help people more readily identify and manage the disease before it becomes life-threatening, I am proud to partner with my colleague and my friend, Doctor Kim Schrier, on the Screen for Type One Diabetes Act. This bill would direct CDC to develop a national education campaign and provide evidence-based screening resources to schools, to pediatric practitioners, and to community health centers. Doctor Kowalski, what is needed to prevent patients from reaching deadly symptoms prior to ever being diagnosed with type one diabetes?

Thank you for that amazing question, Representative Joyce. This is such an incredible priority for us at Breakthrough T one D and thank you for your leadership. Uh, I just came from a meeting in Colorado where data was presented that, uh, people who aren't screened for risk, who don't know they're at risk for type one diabetes, have a approximately fifty percent chance of being admitted in DKA upon diagnosis which is diabetic ketoacidosis. This is a very dead dangerous and potentially deadly complication of high blood sugar and lack of insulin. Uh, and in fact, in my home state of New Jersey, we lost a young girl a couple years ago who was misdiagnosed as having the flu and died because she had DKA. Uh, this is preventable. When screened, that risk goes from about fifty percent to less than four percent, saving significant money, potential lives, and we can screen. So we are doing um significant work to make sure that screening happens, that people are aware, and ideally that every American is screened for risk.

Doctor Kowalski, earlier in your testimony you said that we in the United States have the opportunity to become the first country to approve and manufacture a scalable curative therapy for T one D what would it take from the federal government to increase screening infrastructure and ensure that right now that we seize on that opportunity?

I think this is a another example. We keep hearing about the investment in China. We need to maintain our investment here in the United States. Continue to invest in screening, continue to invest in NIH, continue to support the uh uh a fully functional and robust FDA. These therapies are not a matter of um if, they're a matter of when. And we want them to be when soonest here in the United States.

In your opinion, the legislation that I'm working with Doctor Kim Schrier, the Screen for Type One Diabetes Act. Would that help advance screening practices and promote the early diagnosis of all the goals that we have discussed here this morning?

A hundred percent. Thank you.

Mister Chairman, the screen for type one diabetes is a strong piece of bipartisan legislation that will have impact on American lives, which will allow for the early diagnosis and the successful treatment of individuals with type one diabetes. I thank you all for being here today. Mister Chairman, I r yield the remainder of my time.

I thank the gentleman and now recognize the gentlelady from Illinois, Miss Kelly, for her five minutes.

Thank you, Chair Griffith and Ranking Member DeGette for convening this hearing. First, I'd like to acknowledge that this hearing is being hosted in the wake of the one-year anniversary of the passage of in my opinion the big ugly bill, which has left already millions of Americans without access to critical health care. Patients and families across the country count on our scientific infrastructure to develop lifesaving treatments, which is why it's so important to invest in biomedical research, while simultaneously supporting access for those who need it most. In twenty twenty two, along with my colleague, Representative Eshoo, I pushed forward clinical trial diversity standards at the FDA, passed through the Food and Drug Omnibus Reform Act. These standards raised the bar the new FDA applications to submit a plan to improve the enrollment of clinically relevant participant participants from historically underrepresented populations the FDA FDA issued proposed guidance for diversity action plans in June twenty twenty four and January twenty twenty five this administration removed the guidance in their harmful attacks on DEI while the guidance has been reposted under court order It has yet to be finalized, leaving researchers, providers, and patients in limbo. Doctor, first, um, IQ via report, uh, advancing diversity in clinical development through cross-stakeholder commitment and action, uh, highlights, and I quote, failure to include adequate representation from key sub-populations risk failing to identify potential differences in therapy, efficacy, or safety. Can you tell us about the role of diversity in clinical trials and the safety and efficacy of biomedical treatment development?

Thank you. That's a terrific question. And diversity and represent representativity is critical, uh, for good science. And as you mentioned, diversity action plans are absolutely critical for not only, uh, ensuring efficacy and safety of diverse populations, and of course, increasing probability of regulatory success. It's it's obviously very important too to be concentrating on protocol design, so starting with the end in mind uh for that good science. And that also equates to operationalization of clinical trials, understanding for instance uh where are these individuals' patients that are eligible for a specific protocol, diverse populations, where are they being treated, and identifying the right investigators that have the right patient eligible populations, and even being considerate of some of the uh if you will nuances associated with even the eligibility criterion and uh clearly how they are um treated uh through the schedule of events et cetera. So it's uh absolutely critical and in fact it is good science.

Thank you so much. Doctor Kowalski, can you discuss the importance of clinical trial diversity for participants with type one diabetes.

Oh, uh, uh, it doesn't uh type one affects all races and socioeconomic classes, and w- I I I'm really proud it uh a number of questions have come up on this. We mandate in our grant agreements that uh trials are representative of the broad population. Chairman Griffin, you asked about uh uh rural areas, we've seen in uh federally qualified health systems. Uh, we have a program at, uh, University Hospital in Newark, for example, uh, that are having some of the best recruitment for trials. It can be done.

Mm.

And I think there are mechanisms, especially from funders and from government, to help push to make sure that everybody's represented. Um, I I completely agree with my panelists here that, um, we have to look at all populations to fully understand the impact of new drugs and new therapies. Um, often they don't work in every population, and we have to study uh the broad population to get that information.

Thank you so much. And actually my district, um, I represent the Chicagoland area, but my district is urban, suburban, and rural. So the south side is Chicago and then four thousand five hundred farms as you go further south so uh rural is very, very important to me also. Uh, proper representation in clinical trials ensures that everyone can be confident that treatments are safe and effective. As I continue to fight for critical diversity standards to the NIH clinical trial, integrity act, I urge the FDA to finalize the diversity action plan guidance to the standard mandated by the Food and Drug Omnibus Reform Act of twenty twenty two. And, Mr. Chairman, I yield back.

And the lady yields back now. Recognized gentleman from Georgia, Mister Carter, for his time.

Thank you, Mister Chairman, and thank each of you for being here, this is extremely important and I I appreciate all of your expertise here. You know, I've been around you know for twelve years, so I've seen a lot of what has happened here in the drug industry and uh a lot of the policy that has has shaped the drug industry and in real time and it's um, some of it for better and some of it unfortunately for worse, but you know if you go back a couple of decades we understand that Europe was really the leader in biopharmaceutical innovation and So you ask yourself what happened, what happened in Europe and and why did the United States all of a sudden uh take over that lead. And one of the things that happened was the bureaucratic burden and the price controls that that Europe imposed. And we need to learn some important lessons from that, not to let that happen here in America. I often say that I I practiced pharmacy for over forty years and I saw nothing short of miracles as a result of of research and development, and and we wanna continue with that, we wanna continue to to make sure that um companies are that we're letting the markets work and that we're letting capital flow to risk and that we're letting science lead that is extremely important I do understand and appreciate the point that has been made here about um China and about them taking the lead but we can't we can't um beat China by copying China we China does not operate under the same rules and regulations that we do. We all understand that. We're gonna beat them the same way that um that we beat Europe, and that is by doubling down on what actually made America the the leader in the first place entrepreneurial spirit um access to capital free markets and the best science in the world and I still say we have the best scientists in the world, right here in the United States of America. Doctor Verst, I wanna ask you first um From where you sit, what are are the one or two pro-innovation policies that Congress should be prioritizing right now to make sure that the US holds our lead instead of policies that risk ceding our our leadership to China?

Terrific question. I think that uh most uh recently, of course, trial blazer, the operation trial blazer, those components I think are critical insomuch that it does uh ensure the coalescence or if you will, all of the HHS divisions coming together. We talked here today uh about of course the FDA and - and I know it's not within the uh scope but that adjacency will be critical especially for instance, we talked about today community uh based practitioners becoming uh more integral into clinical trials here in the US and I'll just give a - a few um data points. Oncology. Oncology is the world's largest clinical trial uh market. Over forty to fifty percent of the entire global clinical trials resides within oncology. Our c- our c- oncology clinical trial investigator capacity is being taxed, especially as stated, uh, more of the academic medical centers.

Right.

We - we - it's concentrated there. We have to get out into the communities. You know, so that these are, I think, important components that will help us to ready ourselves for the volume of clinical trials and of course the resulting capacity in order to accommodate them.

OK, well let's let's ta- let's do kind of shift gears a little bit and talk about the clinical trials because it we talked about a little bit up here about decentralizing it and that's something that I really do think is um can be beneficial, particularly in using all the healthcare facilities that are available. It was mentioned FQHC's um I would also carry that to be local pharmacies. Um, that's another area we can. Remember, pharmacists are the most accessible healthcare professionals in America. Over ninety percent of all Americans live within five miles of a pharmacy, so we need to take advantage of those type of things. Um, FTA's own guidance recognizes that fully decentralized approaches aren't right for every trial and I understand that. Um, but, Miss Wainclere, I want to ask you, what's standing in the way of scaling decentralizing trials in the US today and what can the FDA and Congress do to remove those barriers?

So, um, we've talked about some of the barriers and some of it is is training those local investigators. It some of it is even just asking, not only the patient, but asking those providers who might be willing to participate, so the community pharmacist or the the community health centers. Um, and then there is a basic problem, which is contract and budget negotiation. It wastes time and it's ha- it happens again and again and - and uh it - it may seem um strikingly basic and not worth the time of addressing, but it stops engaging new investigators and it stops patients who want to participate in trials having the opportunity to do so.

Well,

We have to fix it.

that - that's - thank you for mentioning that and - and uh it never occurred to me, I didn't realize that was such a problem. Huh. Okay, well thank you all very much and I owe you a bag.

Gentleman yields the bag now, recognize gentlelady from Michigan, Miss Dingell, for her five minutes of questioning.

Thank you, Mister Chair. Thank you for holding this hearing on the importance of maintaining our leadership in drug development. Few people understand the need for a robust, safe and efficient drug development process, like patients with rare diseases and their families. As a result of the Orphan Drug Act, there have been important advances in research on and treatment for rare diseases. Unfortunately, the Trump administration's actions have risked rolling back that progress. The scientists and researchers at the National Institute of Health, who would have previously received funding to develop innovative life-saving treatments for rare diseases, are losing access to grant funding and heading to other countries with more reliable opportunities. a serious issue I think many people don't understand. These impacts are hitting real people first-hand. At the University of Michigan, faculty, staff, most importantly the students are telling me, well, we're gonna lose the next generation of researchers. There are kids that I say, can't you wait? It'll get stable. And they're, we're not gonna be a ping pong ball. We have to have a reliable income. And I think that's dangerous for this country that we're gonna lose the next generation of researchers, and that this instability is impacting their work towards real life-saving innovative and breakthrough discoveries. Cuts to the FDA workforce have added to this chaos, slowing review timelines and making it more difficult for drug makers and patients to get answers about the approval process for treatments that could bring hope to the rare disease community. Doctor Kowalski. I've heard from several companies and patient groups that the turmoil at FDA has impacted the drug approval process. Have you heard that companies are concerned with the instability of that agency, and that they are indicating that they have to think about contingency planning?

Uh, yes, we have, I uh, this is a concern for companies that are working in diabetes, American companies who have what we think will be curative therapies. They are proceeding at FDA, but we have heard

Dr. Kolsky, how will drastic cuts to federally funded health research, including this recent proposed rule that I'll behave and not totally go forth on, to devalue peer review and politicize the federal grant making process affect our ability to advance new therapies including for rare disease treatment.

I often say it takes a village to move a life-changing breakthrough to a person and have their life be changed. I think sometimes we think that this is all the uh responsibility of private companies. Virtually every advancement in in type one diabetes originated with a special diabetes program. Including pivotal FDA trials. Teplizumab, a disease modifying therapy, closed-loop systems, The NIH funded pivotal trials. Companies then drove these therapies to the market. People have done better. So the public-private partnership is incredibly important important here in the United States. And I'm proud of the role, uh, some of our funding has played. It takes a lot to get, but cutting funding would be incredibly short-sighted.

Thank you. Mister Boyke, what, this is what really worries me too. What will these funding cuts mean for US competitiveness with other countries and our adversaries, including China? Could we see more researchers leave the US for more stable funding environments abroad? And I am told I you know, after World War Two, Germany led, we led. I'm told now we are number two in research on versus China. Could you comment?

Absolutely. Great uh question. We are already seeing a flow of scientists um out of the US. Uh, that's a product of the um difficulty of the US funding environment, as well as the rise or emergence of China's biopharmaceutical industry. So this is a real risk. It is also a risk at the agency level. The reality is, some of why it is hard uh to go through the IND process,

I have fifteen seconds. Do you think this is a national security issue?

It is absolutely a national security issue.

Thank you. I'll yield back, Mister Chair, and thank you to all the witnesses.

Gentlelady yields back now, recognizes gentlelady from Florida, Miss Kammick, for her five minutes of questioning.

Thank you, Mister Chairman, thank you to our witnesses for being here today. So, obviously there's a little bit of a dispute in who's leading, but I don't think at this point we're gonna argue about that, but talk about the importance of this leadership in maintaining dominance in this space. That means that we not only accelerate the development of new therapies, but ensuring that we have a regulatory framework that inspires confidence, that strengthens our domestic research ecosystem and expands opportunities for Americans participate in clinical trials. And as the representative, uh, covering the Sid Martin Biotech Hub, uh, a world-renowned facility, this is critically important, not just to our national security, but to folks for me back home. So, I'm gonna start with you, Mister Boyke. Did I say that right? Yes, all right. So, clinical development is increasingly global, has been highlighted here today. But the United States can remain a leader in biomedical innovation by strengthening our domestic ecosystem while continuing to build on those trusted partnerships with countries that share our regulatory standards and commitment to high quality science. The same time, FDA has made it clear that it can rely on foreign clinical data only when it has confidence in the integrity of that data. As more early stage development takes place around the world, that confidence defen- depends on FDA having the resources to inspect the foreign trial sites and verify compliance with good clinical practice standards. something I don't have the confidence in right now. As Congress looks f- toward next user fee uh reauthorization, should we be thinking about those objectives together, continuing to build on trusted partnerships while strengthening FDA's foreign inspection capacity and creating incentives to keep more of that early stage drug development here in the United States? And specifically and quickly, what reforms would you recommend?

Uh, terrific question. Thank you for it. Um, The FDA adopts a risk-based standard to conducting its good clinical practice inspections it's hard to square a risk-based approach

Mm-hmm.

with where those inspections are happening currently. Uh, China, the number of trial or inspections we're conducting in China, not only is dwarfed by the number of inspections we do in the US, it's also true for Japan and Europe. China is is has a number of fewer inspections in that environment. congress should consider uh instituting a user fee for inds that involve foreign clinical trial data so that uh fta can have more resources to be able to pursue these uh inspections we saw something similar in the manufacturing space around generic drug manufacturing it helped uncover concerns with good manufacturing practices the same would be true for good clinical practices

and you would agree that we need to continue to develop the incentives

Absolutely. It really does need to be a a combination of making the US competitive and also having better oversight over uh the activity occurring in China.

A thousand percent agree. Thank you. Um, Doctor Winkler, I'd like to shift to another opportunity for reform, where are you? There you are. Um, so women have historically been underrepresented in many clinical trials, if not most. Particularly where there are therapies that are ultimately intended for both men and women. So while we've seen some progress, it's not near enough. I want to look towards how the next user fee reauthorization and and how Congress could specifically be thinking about increasing enrollment, but ensuring that sponsors are designing trials from the outset to generate meaningful sex-specific safety and efficacy data. Can you speak to that?

Yeah, so there are a couple of opportunities. Um, first it's making sure that that the um sponsors are thinking about what are the endpoints that are relevant to women um or or,

Mm-hmm.

uh shall we say, are there endpoints that are different by sex, uh and making sure that that voice of the individual is heard in designing it. And then in looking at recruiting for trials and engaging in in the research, what are the barriers and what sex-based differences need to be explored not only scientifically, but practically in uh getting then the right mix of participants in the clinical trial. So it requires um intentionality um and then also looking at things, are there opportunities to explore some of these post-market,

Mm-hmm.

right, what needs to be done pre-marketing and then what what might be explored and better understood after a product is already uh on the market, so using real-world data to generate real-world evidence. to help us tease out sex-based differences.

Well, and I know I only have thirteen seconds, but how do you do that without creating additional burdens on the sponsors?

So I think this is part of the upfront and being clear, um,

Mm-hmm.

where you have particular sponsor burden is when it comes in late, or there there was a lack of clarity, or sometimes, um, sponsors don't always listen perhaps as clearly as they should have, uh, upfront.

I appreciate that. Thank you,

General Lady.

and I will submit the rest of my questions for the record.

General Lady yields back,

I yield.

and now recognize the General Lady from California, Miss Baragon.

Uh, thank you, Mr. Chairman, Mr. Boykin. I wanna start with you. Um, I have this chart that I used in uh July of twenty twenty five. It's an FTA organizational chart. I don't know um if you could see it from where you are are, but basically what it is is it has uh the different um sections of FTA And when I use this actually, this is now gone, we've got four more gone. Um, this is now gone, the, we've got four total. But one of them that's been gone, I wanna point out is, is the uh, the food oversight. And why am I bringing up the food oversight? Cuz I'm getting calls in my congressional office about this concern right now, about this explosive diarrhea causing illness, calls called cyclosporiosis, I don't know if I've got that right. which like going through thousands, uh it's going through number of states and thousands of people are dealing with it. Um, do you think that the elimination of these offices within the FDA um is a good thing?

For both the conversation we're having around clinical trial oversight and attracting more activity as well as ensuring public health in the US we need a robust FDA, appropriately staffed.

Okay, so um, you know, right around the same time that I was using this chart, you tweeted this article called inside the collapse of the FDA from the New York Times. W would you agree that it's important that we have experienced uh voices and institutional knowledge at the FDA?

If we're going to compete with other countries, especially China in biomedical innovation, we need to have the expertise and the experience to do the best regulatory science at the fta

uh and do you agree that we need to protect the us system of biomedical innovation

absolutely it's a priority for biosecurity for public health and for patients

and will we help achieve that goal if we're making cuts to the national institutes of health

one of the foundations to the us biomedical innovation system is robust funding for basic research from the nih

So it would not help if we're cutting NIH.

Uh, cutting NIH funding does not advance our ability to have that basic research that has informed so much of US drug development.

OK, and so if we're trying to protect the US system of biomedical innovation, uh, does it help if we're having and continue to have hostility to international students from abroad?

We need to have an environment that attracts the best scientists, both domestically and internationally to be able to compete in the current global environment.

OK, so it doesn't help that that that's happening. And what about uh does it help us be the leaders in bio uh medical innovation if if the United States is attacking research institutions here?

Again, the foundation for US uh biomedical innovation has really been the universities in our basic research. We need to support them.

Right, so it's not helping. And and mister uh Boyki, I think you know what I'm talking about. You wrote an article, um, you wrote an article in which you specifically said, and now I'm gonna just quote from it, um, if I could find the sentence about "The US has led the pharmaceutical industry because of collaboration among American universities." And you go on to say more things. "Yet this resource is not inexhaustible. The presidents risk wandering it through his cuts in funding for the National Institutes of Health." Hostility to international students and attacks on research universities. And that's why I'm asking you, because you wrote about this specific thing. Let me ask you another thing about uh uh because this hearing's about biomedical innovation and it's interesting that it says the role of the FDA when we're gutting the FDA and the FDA is collapsing. Um How about placing blanket tariffs on pharmaceuticals? Is that gonna help the US uh be less dependent on foreign drugs

tariffs if deployed need to target where that dependence arises and that's really on upstream inputs uh particularly from china so if we're going to use tariffs they need to appropriately target that level of ingredients and they need to exempt uh allied and well-regulated diversified sources

and yet again this administration is doing that and the republican congress is totally supporting that. I'm citing your article of July twenty second, twenty twenty five, called America's Pill Problem, and its subheading is, tariffs won't fix the country's reliance on foreign medicines. So here we have all these actions being taken by this administration, all these actions supported by this Republican Congress, and we're having a hearing about how to actually be leaders, yet they're not speaking out against these particular actions. Mr. Boyki, thank you, and I go back.

Ten lady yields back. Now recognize gentlemen from New Jersey, Mister Kane.

Thank you, uh, Mister Chairman. And I appreciate the committee beginning the important preparation to reauthorize the prescription drug user fee amendments next year. New Jersey boasts a thriving biotechnology industry and not only provides jobs locally, but provides the world with therapies that improve and extend life. Uh, Miss Verst and Miss Winkler, my district is home to numerous small uh rare disease companies. These constituents and other companies have frequently raised concerns about inconsistency and even within FTA review divisions. This is a significant source of uncertainty that translates to an unpredictable regulatory environment, and disincentives uh investment in rare in areas of unique need. What additional steps can the FDA take to improve consistency among FDA reviews of rare disease products while also preserving an adaptive framework that acknowledges the unique challenges of rare disease drug development? Both of you.

Yes, so um thank you, and there are a couple of ways. One, I'll say that the FDA's rare disease innovation hub has actually been quite helpful in bringing together not only the Mm. conversation among the divisions at FDA and explanation where they may diverge. There sometimes are scientific rationales that what is good enough for one division in affecting one symptom um or or uh characteristic of a rare disease is appropriate, and a different review division may have a different standard but it's scientifically justified. If we don't know that scientific justification, it it looks wrong or unfair, Um, a- and, and so learning more and sharing more, so having more of those conversations and sharing the learnings, um, in rare disease I think is one of the, the greatest opportunities we have.

Thank you. Ms. Bursch?

Terrific question. I, I think, uh, several ways the, the agency can help on the rare disease front. Uh, notably, over sixty-five percent of all clinical trials conducted around the globe phase one through three are coming from biotech customers. Biotech customers need more certainty.

Mm-hmm.

Uh, and of course their investors need more certainty to fund their their rare disease clinical trials. Uh, I think that having said that, providing greater clarity and guidance will be very important to try to reduce some of the uncertainty. Um, and I do think that the uh, Operation Trial Blazer addresses many of this, uh, in terms of, for instance, uh, highlighting the use of, you know, for instance, one adequate and well-controlled trial with confirmatory evidence. You know, that is a really important step forward, and that revised, uh, draft guidance will help to provide just that more certainty. Thank you.

Thank you. Um, Ms. Winkler, in your testimony you stressed the importance of doing more with less. which is critical for the development of oncology and rare disease products, where trials are costly and it's difficult to find patients. First, how can the FDA support modernizing clinical trial designs and structures to allow for more patient access to trials?

Mm-hmm. Um, there are a couple of different mechanisms. Part of it is is clarity on the part of the regulator. We've said repeatedly this morning that it's it's easier to follow rules that you've heard about and understand. and and can ask questions. So part of it is the clarity from the regulator and then in particular clarity about this idea of a hub and a spoke model where I may be responsible at a central location for the research oversight um then it makes it easier to extend um the clinical trial, uh expertise and reach.

Okay. And second, how can Congress strengthen FTA's ability in this uh through the user fee or authorization next year?

So as part of the user fee reauthorization process, you know, there will be important um situations emerge where actually revising the statute, uh, would be helpful. And and I think that that will there were some ideas today, um, and others that will emerge. And then tailoring the resources that are needed, um, to implement some of those ideas. A statutory requirement for foreign inspections, for example, without any at least assessment of the resources necessary, is a requirement that's just logically unlikely to be met.

Thank you. Uh, Miss Furst, um, there are unfortunately many barriers to mental health care. And I'm concerned, um, no, excuse me, and I'm committed to eliminating those barriers. One of those barriers is limited development of psychiatric drugs compared to drugs that treat physical health conditions. First, can you speak to the unique barriers f- Faced by psychi- uh psychiatric drug sponsors?

Yeah, another terrific question. I think some of the barriers really is uh patient access. And - and of course um that being hugely important when conducting any clinical trial, but especially psychiatric uh trials, and so prom- promoting more of the community-based practitioners, uh because clearly academic medical centers alone will not help solve this problem. So increasing awareness with uh uh and and within the community, the practicing you know physician community in in particular, and really taking on many of the uh areas of opportunity uh that Susan has has mentioned here today uh would be extremely helpful in accessing and recruiting those patients into clinical trials, and of course understanding their needs and requirements to operationalize the clinical trials with their needs in mind. to retain those patients and of course extracting the uh necessary efficacy and safety data to be uh promulgating new medications for these patients.

Okay, and second, what recommendations would you have for

I hate to I hate to interrupt you, but we'll have to do questions for the record.

Okay.

You're uh way over time.

Okay. Uh, well thank you then, I I yield back and I'll follow up with the follow-up questions regarding what what we can do in Congress.

I appreciate it.

Follow-up.

Thank you.

Thank you, Mr. Chairman.

Now I recognize the gentlelady from New York, Ms. Ocasio-Cortez.

Thank you, Mister Chairman, and uh thank you to all of our witnesses for being here today. Um, I want to talk about one aspect of innovation in health care, uh which is our pharmaceutical patent system. Um, I've served on many different committees in Congress at this point, financial services, um uh, you know, natural resources oversight, and this word innovation uh can include really incredible breakthroughs that save people's lives, and also a lot of scammy behavior that we see across industries, you know, in financial services. Stock buybacks, for example, being an innovation that doesn't really create anything new of value, but is certainly a new way to, um, juice some numbers. Um, and so I think I w- I want to dig in a little bit on our pharmaceutical patent system. Um, because this system is critical to drug development in the United States and ensuring that breakthrough drugs become available to patients here. Um, but it is also a system that is abused by big pharma to prevent long-standing drugs from becoming cheaper and more widely available to everyday Americans. Uh, Doctor Wang, when it comes to pharmaceuticals, the federal government reviews a company's patent application, and determines whether or not it meets specific standards, like creating a brand new drug to treat a specific disease. Is that correct?

That's correct.

And when uh patents, and these patents, when a drug company earns a patent for a new drug, they get a period of protection from competition when no other company can sell that same drug, typically twenty years from the filing. Correct?

Right.

And the logic of this is that if you are a company and you invested millions of dollars in the development of a drug you should have exclusivity once you develop that drug to earn that money back uh and have that return and be rewarded for that innovation, right? That's kind of the the gist of the system. Um, but over the years we've seen some drug companies do some curious things with this. Um, we've actually seen them, I think, abuse this period of protection to create monopolies over the market, and then increase the prices on their drugs. One example of big pharma abusing this system is when they file additional patents. They'll make a new drug, they'll get their twenty years, then they'll file additional patents that involve really minor and often unnecessary changes to their product. So they'll have a great pill and then they'll just change it to a capsule and then refile the patent um and we're seeing that in in quite a few uh treatments, correct?

That's correct. In in a small number of cases, some of these reformulations can help our patients, they can make treatments more available and easier to use, but in the vast majority of cases, these are life cycle extending.

Yeah, in fact, the pharmaceutical company AbbVie used this strategy with their drug Humira, which is used to treat rheumatoid arthritis. By barely changing their products, they obtained over a hundred and thirty patents that gave them continued exclusive market control over rheum over rheumatoid arthritis treatments and during this time the price from Humira from Humira went from five hundred dollars for one syringe to nearly three thousand dollars per syringe. And that l- led to about eighty thousand dollars for a one year supply for a patient. In fact, AbbVie isn't the only one doing this. I have an example right here, um, from AstraZeneca. This is an inhaler pump. Um, it was loaned to me by someone with asthma. And we've seen that AstraZeneca, what they did was that they had this treatment, the actual, uh, medication inside, did not change at all the administration of it um but they filed a new patent to maintain exclusivity of it and can you guess what was the great innovation that was worth this not going generic for

i'm i'm not aware but i think you know the answer congresswoman

yeah it's this little plastic piece right here and it's one of the big features of that new patent this thing to keep the cap from coming off uh was a major part of the new patent that they had filed um and to extend and to prevent this lifesaving drug from going generic and um and bringing down costs for everybody. I was just curious what do you think we do about this?

I think very quickly and I'd be happy to submit more for the record but Um, these strategies unfortunately reward the very thing that we should um be avoiding, which is aggressive legal maneuvers instead of the risky basic science that our country should prioritize.

Great. Thank you very much, and I yield back.

Generally, he yields back now, recognizes gentleman from Ohio, Mister Balderson, for his five minutes of questioning.

Thank you, Mister Chairman, um and thank you all for being here. Um, my first question is for uh Mister Calvas, doctor. Kowalski, thank you for being here. Uh, your testimony mentions how the breakthroughs that wearables such as continuous glucose monitoring devices have helped millions of patients manage their diabetes, and that these were made possible due to sustained research, investments, and a strong FDA review process. What ideas do you have to improve clinical trial designs and requirements to spur innovation for these types of wearable technologies to continue to help patients with type one diabetes.

Uh, thank you for that question. That's a real point of pride that I got to work on the project that developed many of these therapies and my brother and I benefit as do many, many people, which is uh amazing. I think what we saw in that process and what we need to continue is clear, and we've heard this from a number of the panel members, clear and consistent regulation. We want sponsors to know what they need to do. I think we need to continue to have the voice of the patient at the table. You know, as we continue to innovate, type one diabetes isn't solved as are uh many diseases. We still have a lot of work to do. And the risk benefit, we need the voice of the patient and the voice of the clinicians at the table. We also need to accelerate uh the use of more biomarkers. You know, as we look at how fast can we move the trials, for example, in some of the m- uh e- even next-gen uh type one diabetes therapies, we've been limited by a an old way to look at type one diabetes that's not even applicable when you're at risk for the disease, which is really slowed down process progress. So better bio um markers. And I just can't stress enough the the risk benefit um barriers. Being risk averse when people are suffering, developing diabetic complications, um living with this uh grueling uh disease, we need to take that into consideration and continue to innovate.

Thank you very much, and I appreciate your passion with that, so. Uh, my next question is for uh, Doctor Furse. Thank you for being here, ma'am. Uh, I often hear that rare disease drug development is limited not by scientific opportunity but by the difficulty of identifying, enrolling, and retaining patients in clinical trials. As we look forward for ways to strengthen America's leadership in biomedical innovation, what specific regulatory or policy barriers still prevent broader participation in rare disease clinical trials? And what actions should Congress take to ensure that rare disease patients can access innovation research regardless of where they live?

Terrific questions. I think initially, uh, patient access and understanding where these patients, their standard of care, the patient journeys, uh, are just critical questions to help us when uh identifying rare disease trial opportunities, trying to access those patients at the right time on their journey. And that can be achieved through real-world evidence and understanding then the natural history of their diseases as as well. And insomuch as using that real-world data a- and and of course ensuring that we are helping with the design of clinical trials that is very simplified and not burdensome, for neither the patient nor their uh investigator. And that I think is uh clearly uh of importance and where you can help, and of course again I refer back to Project uh Operation the the Operation Trial Blazer where there is some uh clear guidance uh that you can help us to uh ensure that draft and revise guidance on for instance uh one randomized, well-controlled trial with confirmatory evidence that helps to reduce the number of patients uh required for the generation of evidence and that being so critical uh to ensure that treatments or getting better treatments uh to our patients faster.

OK, thank you. Um, I I will stay with you. Um, doctor, clinical trials produce plenty of data, but sponsors frequently report that the process of gathering and submitting

Okay, I'm gonna go very fast. Uh,

Mm-hmm.

firstly, I think uh with regard to the use of again real world evidence, life science models, you mentioned digital, digital twins. novel trial designs, which is absolutely critical. In addition, you mentioned wearables, uh, the use of, uh, patient-reported outcomes, digital connected devices, uh, and and reducing, if you will, the burden on, uh, the investigators and patients is is just in- incredibly important. And the good news is that the those digital data sets actually reduce less time, less burden, less cost, in cleaning data, and in fact those data can actually be surfaced for early signal detection, thus accelerating development.

Right. Thank you very much. Mr. Chairman, I'll go back.

Gentleman yields back now. Recognize the gentleman from Louisiana, Mister Carter, for his five minutes.

Thank you, Mr. Chairman and Ranking Member. To our witnesses for joining us today. We're here today to discuss the critical role of FDA and the importance of maintaining America's leadi- leadership in scientific research. and innovation on a global scale. You had Republicans in the Trump administration who spent the last year attacking the federal workforce while dismantling and politicizing the research that drives the development of lifesaving treatments and cures. Now to make matters worse. The Office of Management and Budget is proposing some of the most sweeping and damaging changes to the federal grou- grant-making system in modern history. These reforms will replace long-standing non-partisan merit-based grant-making processes with one ones that is subject to political influence and control. Federally funded research is the infrastructure that powers American innovation, science, and national security. When we cut that funding, we aren't just trimming the budget line. We're shrinking the pipeline of scientists and researchers that this country depends on to remain competitive on a global scale. At a time when our foreign adversaries are strategically making investments in scientific education and emerging technologies, choosing to shrink our own talent pipeline isn't physically responsible. It's a big mistake. The consequence of these actions will be felt for decades and weaken our ability to compete and innovate and lead globally. Doctor Wang, I proudly represent New Orleans, home of world class research institutions like Tulane University, LSU Health and many others. These institutions rely on federal funding to advocate groundbreaking biomedical research innovation and develop discoveries to improve lives, and health outcomes. How do federal research funding cuts affect United States leadership in biomedical research and innovation?

Cuts to that federal investment, as you mentioned Congressman, are tremendously harmful. Um and they um threaten to push back um all the leadership that we've gained over the years.

From your experience as a physician and an academic medical center, what impact can these disruptions have on clinical trials in patient access?

It could add a harmful role um for sure on um the initiation of new trials and and also um creates a freeze over trials that are already in progress. Um and I just want to stress for the record that these views are my own, and don't represent those of my employer.

Fair enough. How do these cuts undermine the global competitiveness, particularly in the competition of countries like China?

Again, I think um a loss of investment in American science does um a big disservice um to our efforts to create new therapies.

Why is maintaining strong workforce at FDA important to advancing new treatments and protecting patients?

As we've heard from other members of this panel, um, FDA has an incredible role in the innovation ecosystem, and um, our leadership over the past two decades relies on a well-functioning and well-staffed FDA.

You've got members of Congress sitting here with you. What should we do? If you now have a magic wand, and you're talking to members of congress who serve on this powerful subcommittee how would you use your magic wand tell us to do better

uh representative first I would start by asking how much time you have um

I have a I have a minute and twelve so you can give me a good answer

yeah

but um in seriousness I think um the first thing that we can do um uh um from the congress's perspective is uh reauthorizing the user fee agreements and funding the NIH Um, and assuming that we have those things in place, I I refer to some of the recommendations that I have in my written statements. Some of those include improving transparency so that sponsors know how to run trials and also learn from past failures. And the second is increasing the reliability of data that's imported and used to support new drug applications here. And part of that involves ensuring that data from non-U. S. trial sites are valid and that they're done with integrity.

How damaging is it to threaten NIH funding And how, what impact does that have on our budding researchers who are looking to study here in America and continue when they see the threat that NIH funding may be put on a political chopping block?

I think it's incredibly damaging. All of the research that I know of points to the incredible work that the NIH does, and the importance of it um for new medicines. Um and I worry that the next generation of world-class talent um will leave this country, because of those cuts.

And we must do everything in our power to ensure that the best and brightest sets here, stay here, the best and brightest that want to come here to study, to find, uh, cures for dreaded diseases, continue to wanna come here and that's why we must maintain our leadership role in funding for NIH. Uh, my time is is ended. I I yield.

Gentlemen, gentlemen, yields back now, recognize the gentleman from New York, Mister Langworthy.

Thank you, Mister Chairman, uh, the United States has long been the world's leader. in biomedical innovation and american researchers, universities and life science companies have developed groundbreaking treatments uh that have improved in many cases saved millions of lives of people, not just here in the united states but around the world. But we can't take that leadership for granted. Uh as other countries work aggressively to attract biomedical research and early stage clinical development, we should be looking for opportunities to modernize outdated processes, embrace new technologies, and remove unnecessary barriers that slow motion. One area where I think we're seeing some of the most promising advances in the early stages of drug development, and and with that uh Doctor Wurst is you may know much of the preclinical drug development process still relies on laboratory and animal models for dosing and toxicity studies. Uh as technologies like AI, uh virtual control groups, and other new approaches uh and methodologies continue to advance, where do you see the greatest opportunities to safely reduce the reliance on animal models? And from IQVIA's global perspective, what scientific or regulatory barriers are preventing a broader adoption of those technologies here in the United States?

Hmm. Terrific questions. I think by far, uh new approach methods or NAM is is the needle mover in terms of preclinical uh, acceleration of timelines. I think, uh, as the agency, the FDA just recently in operation trial, uh, Blaser identified in fact a new quantitative pharmacological modeling on this very front that helps us with dose optimization, which is, as you know, very critical, uh, in animal testing is getting the right dose, the right safe dose. Uh, and I think those are the, uh, methodologies that will significantly enhance the efficiency and acceleration in the preclinical space and i think it's just the continuation of the agency providing uh clarity and the actual guidance uh uh that will be extremely helpful, and especially biotech uh biopharma uh uh sponsors that so need this uh uh very consistent and clear uh guidance as they develop their drugs.

uh i appreciate that and i'm and i'm encouraged by the progress we're seeing uh with the tools like the virtual control groups and other validated alternatives but we shouldn't continue relying on outdated testing models that are unnecessary and lead to the unnecessary slaughtering and and uh untimely deaths of dogs and cats in this country simply because that's the way it's always been done And um, if these approaches can provide the FDA with reliable scientific evidence that it needs I think we have a real opportunity to stop the p process of testing on cats and dogs in this country uh and I believe there's a mandate that's growing from the American people as the curtain's been pulled back on this process uh that they don't wanna see this as the way we're approving our drugs in this country um and it's just common sense. Uh, but that's only one piece of the puzzle. We need to make sure that the United States remains the best place in the world to conduct early stage research in developed medicines because I don't I, I, I wanna lead this country to ban animal testing on dogs and cats. I don't wanna see it just off- offshore to China, where they are going to do it without any humanity whatsoever. Um, so, uh, Miss, Miss Winkler, What best practices should FDA implement to make the United States the most attractive environment for early stage research and clinical development?

Uh, first and foremost, it's a risk proportionate IND requirements. So saying for the risk of this product and for the the um, you know, is it going to healthy volunteers or into patients, what do we need to see in that IND? And also then focusing FDA re- resources on the more novel interventions where we might have more safety questions. Another mechanism is continuing FDA's efforts in the um new alternative methodologies that Doctor Wurst mentioned and in particular not n we shouldn't be looking for methodologies that replace what we learn what that replace the animal models we should be looking for new methodologies that give us the information that we were looking for in the animal models and those are two different questions and so we should look be be exploring and sharing what what were what toxicity were we looking for and how could we do that in a different way than using animal models. Also need to strengthen our our phase one um intensiveness, but primarily it is a risk proportionate approach to IND submission and transparency about that process and a rolling application.

Thank you very much. I could ask you questions all day, but I'm out of time, so I'll submit some more for the record and I yield back, Mister Chairman.

Yeah, I'm gonna yield back now, recognize Mister Landsman of Ohio for his five minutes of questioning.

Thank you, Mister Chair, and thank you all for for being here, uh I wanna talk about biosimilars and uh get some uh feedback and on on how to get some of these really important drugs to market faster. So as as you all know these are generics and uh you know in Europe if it's approved it's approved and and we move forward, or they move forward, and here uh if it's approved there's additional testing and so on and so forth. And so Uh, it it leads to, you know, it's a very time-consuming costly process, uh, and patients end up paying more, uh, for prescription drugs. So, uh, one of the things that, uh, we have proposed, uh, Representative Flueger and I, is a biosimilar red tape elimination act, which would remove these extra steps and lower the cost for patients. Um, uh, uh, Doctor Winkler Given, Miss Winkler, sorry. Given, promoted you. Sorry, I don't.

I don't have a JAD.

She's got a JAD, exactly. Miss Winkler, you gave that sense. She's gotta be a doctor. Given the FDA's uh current framework, what changes uh at the FDA would best support biosimilars and other generics entering the market? You're very close to this, obviously.

Uh, yes, so um the foundation facilitated a series of conversations with biosimilar developers who uh had not had much interaction with the F the FTA. And what we learned from that project um was at some level not surprising. What the biosimilar developers said is we need more clarity about what's what's required, right, like what exactly do we have to do, and then the rationale for it, as well as um just uh more conversations where the FDA maybe have expertise in the project, the product rather, where the company is trying to develop a biosimilar, how much of that can be shared. So it it is clarity, it's continued um learning and and sharing the the the information. And then we should recognize that some of the challenges in the biosimilars market are actually not with FDA and getting them to the market but their adoption and pull through. So, FDA can certainly do some.

But is it is it not true that when the FDA approves a biosimilar there's still additional testing, whereas say in Europe, if it's approved, it's approved.

Well, so it's approved there th th and there are um some continuing monitoring, but the main challenge that that we have seen is that it uh tends to be payer adoption, that uh, present some of these actually getting to patients.

What does that mean? Sorry.

Um, in that if I am setting a a formulary, I may choose to continue to have one of those products rather than having um Part of the reason the price drops when you have generics or biosimilars, is when you have a number of them entering the market. In order to enter the market successfully, you need to actually reach patients so that you have dollars returning. If the payment structure prohibits that getting to the patient and actually getting those dollars, then you don't have it you biosimilars won't enter the market.

If you could change it to mar- today, I mean, is there any one big thing that that would help expedite this, eh, that you think makes a lot of sense or

I'm always looking for the one big thing and this doesn't have one. Um, but there is I think FDA has made strides in being more clear about what is actually required. And and then sharing that information with the biosimilar community and

I mean, our sense is that there is just too much red tape,

we need that.

uh,

Mm.

you know, when it comes to these things. And so, um, ye- and so I guess the the question, I mean, you know, the clarity obviously helps, but I it it also just seems to me that this is, uh, uh, this has already been tested, it's already been approved, it should just, you know

Yeah. Well, and the structure for our biosimilars is, um, you know, much newer than the structure for generic drugs, but it's not new, and so the it is an opportunity where we have learnings and

Yep.

perhaps applying those learnings and changing the standards, it may be time.

Um, Doctor Kowalski, could you speak to the importance of bringing more of these biosimilars, uh uh generics to market for patients? Um, especially, you know, we we talk about we've talked a lot about the capping the cost of insulin for seniors and how this could increase competition and and lower costs.

Oh, so we uh strongly support uh the bipartisan uh insulin act. Insulin, as I said in my opening statement, is mandatory for life with people with type one diabetes. And we've seen people rationing insulin and even dying for rationing of insulin. So any mechanism that will drive costs to be affordable and effective, so people aren't choosing uh between rent or a car payment and their insulin,

Yep.

I I've cut you off, so I'm sorry I went over.

Mister.

I um but I appreciate that and I I agree. Thank you. I yield back.

Gentleman yields back. Now recognize the uh gentlelady from Washington, Doctor Schreier, for her five minutes of questioning.

Thank you, Mister Chairman, and thank you for that answer about insulin. Uh, anything we can do to bring down the cost of a medication that uh people like me would die without within days, uh I think. I'm not gonna test it, is really important. Um, so thank you to all of our witnesses. Uh, I want to talk first, um, about biosecurity and research and global competition. Seventeen years ago, the share of clinical trials started by Chinese companies was only one percent. Uh, last year, China headquartered companies accounted for thirty-nine percent of global oncology clinical trial starts and thirty-six percent of vaccine trial starts. and that is massive growth, uh and they are quintupling on their investment in research and development. Um there are really practical implications to this, I I would just point to COVID as an example, the first Chinese developed COVID vaccine showed significantly lower efficacy um than the one in the US uh ranging from fifty point four percent to about sixty percent. Um by comparison the first American made uh mRNA vaccines were about ninety-five percent effective. And American innovation of various standards, supported by a fully-staffed FDA, uh, saved millions of lives during the pandemic. Uh, I'm thinking about the next pandemic. Um, and if we yield our biomedical leadership to adversaries, the consequences for our country could be catastrophic, in- including whether our adversaries would even share those vaccines with us. Um, Ms. Winkler, when early stage cancer and vaccines trial trials m migrate to China because the regulatory pipeline is faster and cheaper, what does that mean for the safety of Americans? And in a world where China, say, leads, for example, in drug research and development, how much longer would it take American patients to get those breakthrough medications and treatments? So part of the the

challenge here too is right when we have the innovation here we're learning here and we're we're having the the local um and and better understanding of the product and we can innovate more quickly when that innovation is not here we need to learn from it and so the i think the the challenge here is how do we better improve the us system so that folks will want to be here um because there is a risk of um others there there is the risk of what we do not control being weaponized against the united states and particularly in biomedical innovation and simply access to biomedical products or food or or you know these things that are essential to life uh we should be positioning the us for greatest success so that we are less susceptible to such challenges

uh i completely agree all we have to do is reflect on where we were with regard to masks and gowns five years ago and that really paints the picture of what it means when you get into more um more complicated uh and scientific uh elements. Um I wanted to pivot a little bit to vaccines because there's been a lot of discussion information and a lot of misinformation about vaccine safety um and and I wanted to just point out first um long before vaccine ever reaches a clinic the FDA does really thorough vetting and the determination of safety and efficacy through gold standard clinical trials and w- once it is licensed, it then goes to the CDC's advisory committee on immunization practices, or ACIP, um, where they look at FDA data, pore over these thousands of pages, and then recommend exactly for whom and at what age on a- and on what schedule a vaccine should be administered. um to best protect our country. Um the ACIP recommendation process has saved millions of lives, but its uh authority really depends on uh scientific credibility. And that's why Secretary Kennedy's overhaul of ACIP has been uh so concerning. Um when we inject or when he injected politics into a committee meant for objective medical decision making, we threatened the hard-earned trust that parents and physicians uh like myself rely on every day um to any of the panelists who wanna comment just in twenty five seconds here in light of the recent political overhauls of advisory committees like ACIP how can congress ensure that fda's clinical trial evaluations remain insulated from politics and stick with science

so i'll just make one observation in and to remind us that when FDA approves a product, whether it's a vaccine or a drug or a biologic or a device, their job is not over. FDA conducts and monitors, and the product sponsors do as well, post-market surveillance, so the learning continues. So I would just underscore that it's um we sometimes think about the review and approval process as ending. It actually begins the next stage of scientific understanding, which is continuing to gather information about how that product performs in continuing to monitor for negative effects, which is an important part of the entire uh medical product regulation system.

Thank you. I wanna double-click on that because there are some things that happen in one in two million people and you don't know that until it's rolled out.

Yeah.

Thank you for that continued spiel.

And the gentlelady yields back and now recognize

Yield back.

the gentlelady from Massachusetts, Miss Trahan.

Thank you, Mr. Chair. I'm glad we're holding this hearing about biomedical

innovation which is absolutely critical in my home state of Massachusetts, and to every American who's desperately waiting on a cure. This hearing is based on a real threat, that we're losing our edge to China. But I wanna be clear, you know, under this administration, we're not being outpaced, we're forfeiting before any promising new therapy even reaches the FDA. There's discovery, there's preclinical work, there's first in human testing, and almost all of it traces back to NIH. Nearly every drug the FDA approves is linked to NIH funded research. So talking about late stage clinical trials when the entire pipeline has been under siege feels like we're rearranging the deck chairs on the Titanic. Doctor Verst, your testimony notes that emerging pre-commercial biopharma companies drive more than three quarters of new phase one trial starts. Is it fair to say that these small biotechs are some of the most vital for the innovation

Indeed. I think that the these are critical um customers or sponsors rather, in terms of um fueling and promulgating our innovation, as you rightly highlight. In fact, um the seventy-five percent being phase one in orientation

Mm-hmm.

in terms of the predominance of the biotech uh sector. But in addition, sixty-five percent of phase one through three clinical trials globally are actually conducted and invested by this biotech community.

And and these are the most sensitive to capital constraints. Um, Doctor Boicki, can you elaborate on what happens when one of those small companies loses its funding in the early stages? Does that science wait patiently for them to c- recover? Does it go somewhere else or does it die? It's enlighten us.

It uh, the science doesn't wait patiently. So um, uh small biotech firms in china are taking advantage of the opportunity to do regulatory arbitrage by going through a pre ind process of being able to

yeah

uh conduct first in human trials much quicker uh to be able to fast follow the innovations that occur in the us and be able to seek licensing deals for that research

the district i represent is home to companies just like that i'd i'd like to share one of them uh versatope therapeutics is a small biotech. Just a couple dozen employees that grew out of UMass Lowell's um incubator. They were developing two critical innovations, a malaria vaccine and a universal flu vaccine, one shot lifetime protection, both NIH funded. But last year NIH terminated Versatope's flu vaccine grant for convenience. Their malaria grant renewal was frozen for months. And although it was ultimately reinstated, the damage had already been done. ten scientists were furloughed, it's possible that the products they were working on will never see a pivotal clinical trial. And as if that wasn't bad enough, it gets worse. OMB just proposed a rule that takes that exact mechanism, termination for convenience, and makes it the law for every federal grant in our country. That same rule replaces the scientific review process with political appointees, upending years of stability that has made the United States the global gold standard when it comes to biomedical research. Doctor Kowalski, Breakthrough T one D submitted comments on the rule earlier this week. Can you explain what it means for early stage researchers to have this codified in federal regulation and how will it change what recourse a company has when its grant is simply terminated?

Well, Breakthrough T one D did sign on to a United for Cures coalition letter letter to the both the House and the Senate, uh, requested that proposal be withdrawn. We, uh, feel very strongly that letting political appointees override scientific peer review, uh, puts decis real breakthrough therapies at risk, as you uh have described. And uh having grants terminated for convenience threatens long time studies and significant progress made it making that we're making towards breakthrough therapies.

I agree. You know, that's why I led more than a hundred members warning OMB about the potentially disastrous consequence on this rule, specifically on our biomedical research infrastructure. I'd like to enter into the record the response we received from director Vogt.

And the date?

And I'd no- uh, it's dated June thirtieth.

Okay, of this year.

Yes.

All right, without objection.

It doesn't seem to me like

Objection so ordered.

Thank you. Doesn't seem to me like they're taking full and fair consideration of the almost three hundred and fifty thousand public comments they have received. In fact, this response doubles down on the rule, calling NIH grants too woke. and recycling old conspiracy uh theories. It's just deeply unserious.

Yeah.

This administration says America should be the best place in the world to develop medicines. And all of us here agree with that. But you can't be that place while signing innovative companies' death warrants and calling it convenience. Thank you, Mr. Chairman. I yield back.

And, Mr. Ahan, if you will make sure you get a copy of that, so we can have it for the record, and now recognize Gentleman from Texas, Mister Vesey, for his five minutes of questioning.

Uh, thank you, Mister Chairman. I know that much of today's discussion has focused on how Congress can modernize clinical trials uh and make them faster and more efficient. And I think those goals are very important, uh, particularly as advances in science and technology create new opportunities to bring innovative therapies to patients more quickly. Uh, at the same time, I think especially we learned a lot of this lesson around COVID-19, uh, making sure that we ensure that clinical research uh reflects the diversity of the population that we have here and who's ultimately want to use these therapies is going to be very critical to generating evidence that is both scientifically robust uh and applicable doctor uh given your experience designing and implementing these these clinical trials across a wide uh array um how can congress insure that efforts to modernize clinical trials also expand participation amongst uh historically underrepresented communities again, particularly when you take into consideration a lot of the noise that had we have to go through during COVID-nineteen to get people to just to take that that vaccine.

I- it's a terrific question and certainly, you know, a challenge on the clinical trial um uh front. I think that first and foremost, understanding how to recruit and incentivize community physicians to participate in clinical trials. And of course, it's incumbent upon us as an industry to ensure that we reduce the burden um in participation. And beginning as uh Doctor Winkler, I give you honorary degree,

Thank you.

uh mentioned in terms of the academic medical centers are critically important, but so are the hub and spoke referral methodologies. If we're able to uh put the the right technology systems in place within health care systems, EMR uh technology that can enter uh be interoperable with electronic data capture associated with clinical trials. One would then be able to identify the right patient at the right time on their clinical journey to be eligible for certain uh clinical trials. That's just one example of how to access those community uh

Mm-hmm.

physicians and of course their respective patient population to help encourage more participation in clinical trials. And we call that clinical research, uh, really as a treatment option.

Yeah, yeah. Wow, wow. That's there. I'd also like to touch on the FDA's approval process, which has long relied on evidence demonstrating that new therapies are both safe and effective. And as advances in science create opportunities to use novel endpoints and biomarkers in clinical development, we are considering how these tools can complement traditional And I know that this the challenge is ensuring the gr- that greater flexibility strengthens the clinical research enterprise while preserving the, uh, high evidentiary standards, uh, that providers rely on. And, uh, Doctor Kowalski, your testimony supports modernizing clinical requirements, including increased use of novel endpoints and biomarkers. Uh, Doctor Kowalski, what safeguards are going to be needed to ensure that these new approaches continue to generate reliable evidence and maintain public confidence?

Sure, I think uh having a robust FDA that is staffed uh uh appropriately and following clear and consistent regulatory um expectations will help us lead with safety and then follow with efficacy I think in our patient community, we've seen uh uh um the pace not meet the sense of urgency and that again the opportunity cost as you're living with a dangerous disease every day where you're dosing a life-threatening drug in our patient community.

Yeah.

So I think that risk-benefit analysis has to be clear, and then we need the right staff to then follow up on executing against the regulations. Um, our community is desperate for innovation and we're seeing it and we're on the cusp of it. And um, I think if we lead with safety, uh, you'll see innovative therapies reaching the population and driving benefit.

Yeah, no, absolutely. Thank you. Mrs. Sherman, I yield back. Thank you. The gentleman has yielded back and we have Mr. Alkencloss on the way. I would ask the witnesses, do you all have time to wait a minute or so? Now if he's twenty minutes away, we won't wait. But if he shows up in the next minute or two, we'll we'll hold if you all are okay with that.

That's great.

All right, thank you. Alrighty, we had turned the clock on. We were gonna give you another five minutes. You're done. You still had four minutes and thirty seconds, but I'm glad you didn't wait to Then Bush is to the test. I now recognize the gentleman from Master Ch- Massachusetts Mr. Alkin Claus for his five minutes of questioning.

Thank you, Chairman, and thank you in particular for your ongoing partnership as we work to modernize clinical trials in the United States with bipartisan legislation, currently in discussion draft form, which we've gotten uh substantive feedback on and hopefully soon in uh legislative form. Um, I wanna discuss that uh with the witnesses here and i've uh appreciated the written testimony. My first question is for you, Miss Winkler. Um uh

Kevin.

We have seen many areas of overlap between the research that Reagan Udall has done on clinical trials modernization, uh operation trial blazer from the administration, and then our own um legislative draft, including risk-based non-clinical testing, patient-centric patient mash matching, increased FDA communication and transparency, shared learning to the agency's application of flexible evidentiary strategies and adaptive trial designs, and better use of existing data, such as natural history studies and real-world evidence. Um, what are key considerations for Congress to support FDA consistency and transparency in trial design accepted endpoints and natural history studies while maintaining a flexible regulatory environment?

So, um, key is part of the what what you just talked about where it's a consistent message from what congress is uh creating and what stakeholders are saying and what HHS is saying that um seeing the same threads uh in a common direction is incredibly helpful to the FTA staff in saying yes this is how we pursue it um and then

yep

continuing with that is the clear question of um is there clarity in the authorizing statute then the agency has the opera opportunity to provide the detail and regulations and the resources and staff to implement it.

Part of it, Congress can legislate, and and we should, and and our bill really focuses on the things that only Congress can do, particularly creating a a point of care clinical trial enrollment process. Cuz only, you know, maybe five to seven percent of eligible US patients are enrolled at any given time. And if we want to widen the throughput of US clinical trials, we just have to make it easier to recruit and enroll, right, and I think Congress has a special role there. A lot of it can be done by FDA leadership though, in terms of that that trial design, accepted endpoints, consistency. FDA's had a a hurly-burly last eighteen months, um, and now has an opportunity for a reset after regrettably hemorrhaging a lot of competence and credibility under the leadership uh uh uh the previous leadership. What advice would you give to an incoming FTA commissioner about how to restore a culture of high standards for safety and efficacy and also the kind of consistency and early engagement that creates certainty for investors and investigators?

Mm-hmm. So, um, it obviously that that job is an incredibly challenging one, but the the, uh, a way to approach it is thinking about the stability for the staff. So what is it that the agency is pursuing, what are the priorities, and what are the what are the expectations of them? And that's that they discharge their work as strong, competent scientists and and lawyers and all of the other staff who are experts in the s- the the law and the regulatory structure. And in order to do their job well, they have to interact with the experts outside the FDA to help with the emerging science and the emerging products. So, underscoring that stability within the agency as well as the um in the really the essential communication and interaction with external stakeholders in order to keep pace with the science. To be a science-based regulator,

Which

you have to keep pace with the science.

Which, you know, of course, is what Reagan Udall has been designed to do, right? Um, and I I do think for that for those FDA regulators that human t- one of the things I hear from investigators all the time importance of that human to human interaction early on in the process. Just sit across from the table with each other and just go through point by point, what are we looking for for endpoints, what are we looking for for trial trial design. My sense has been that that highly iterative interactive process has degraded over time. And not just, I I wouldn't say not just be under the previous leader, but maybe really over the last five to ten years. And I think the next FDA leadership has to just inculcate that culture again of you know really engage deeply with Your investigator.

Exactly. Um, some, I think wrongly, call interaction between the regulator and regulated industry as industry capture. I I think to be a functional regulator you have to interact with the industry that you're regulating so that you understand your their challenges.

Specifically early on.

The regulator still makes the decision.

Yep. Specifically early on. Um, well, much more, but I will yield back, uh, to the chairman.

I thank the gentleman, now recognized gentleman from California, Mister Mullin, for his five minutes.

Thank you, Mister Chair. And thank you to our witnesses for being here today. I'm glad we've convened this important hearing to discuss the US leadership uh and the FDA's role uh in drug development. I am very proud to represent California's fifteenth congressional district, otherwise known as the birthplace of biotechnology. Life-saving therapies and cures are research and developed every day in my hometown of South San Francisco, pushing the boundaries of what we thought was scientifically possible. and expanding our ability to keep our loved ones healthy. Before discussing ways the FDA can advance drug development and the review process, we must first address the unforced errors coming from this administration that is ultimately slowing down drug development and potentially preventing the next groundbreaking therapy from making it to patients. Attacks on the NIH, research cuts, and recent OMB efforts to politicize independent scientific research are all pushing innovators to study and work abroad. We're going to lose a generation of the leading scientific minds, and President Trump and the majority continue to undermine American innovation. On top of that, preventable upheaval at the FDA is hurting domestic drug development. I recently sent a letter to the FDA outlining how the agency's staffing cuts, leadership turnover, and inconsistent priorities is adding uncertainty to the system and making it increasingly difficult for manufacturers to work. with the FDA. And those un- ultimately hurt by the FDA's delayed communications and changing guidance will be patients who must go even longer without the therapy that could change their life for the better. So, Doctor Kowalski, uh, thank you for being here. From the patient perspec- uh, advocate perspective, could you please outline what the impacts would be if the next therapy or cure and development for type one diabetes for example was delayed due to preventable factors?

I mean, I think the answer is quite obvious, it would be devastating. I mean, our patient community, we've seen amazing advancements in my lifetime. My brother started on urine glucose testing. Um, and here we are now wearing automated insulin delivery systems, thanks to NIH support, partnership with FDA, partnership with companies. And now here we are on the cusp of curing the disease and preventing the disease. And to lose that would just be absolutely devastating.

Thank you for that. We cannot lose the momentum uh in in so many ways. And in addition to pushing back on ways this administration has slowed US drug development, we must also be forward thinking about ways for the FDA to modernize processes as the science continues to advance. So uh, Ms. Winkler, your testimony highlighted that current FDA communication with drug sponsors leads to guesswork and overly conservative assumptions that ultimately slows down the process. So, what more does the FDA and Congress need to do uh to streamline these interactions and ensure responses are clear and prompt?

Mm-hmm. Part of it is uh simply having the opportunity for more interaction, a particular early stage, smaller companies, to be able to have that that conversation. Um and and then the that there are risk um proportionate or uh requirements. So what is the risk of the IND and are you having phase appropriate requirements for that? Uh, and then it is um the clarity and speed of communication is is essential. And then we have to tackle the other part too, where sometimes sponsors assume because someone else has done something, they must do it as well. So they the data packages get bigger and bigger because they saw someone else do it. Um, we have to clarify that in fact that's not the approach that that may be needed.

Thank you for that. So um, Uh, let me just pardon me, conclude by saying it's vital that we meet the scientific and medical needs to, today by continuing to modernize and streamline FDA's drug review process. I look forward to continuing this work to get therapies to patients faster. Thank you all for being here. Thank you, Mister Chair, for allowing me to wave on, and with that I'll yield back.

Gentleman yields back. That brings us to the end. I would like to thank all of our witnesses, uh, again for being here. I was accused to get uh having more people in the back like a clown car and bringing more out, but uh she told me she only had five more and apparently they didn't make it before I closed. Uh, I'll remind members uh w- excuse me, thank you all for being here. Members will have additional questions uh for you all subsequent to the hearing. I'll remind members they have ten business days to submit their questions for the record, and I ask the witnesses to respond to the questions prop- promptly. Members should submit their questions by the close of business on Wednesday, July twenty ninth. And uh we let's see, I must I must have missed a page. We approve all the other items that were uh on the documents for the re there we go. I ask unanimous consent to insert into the record the documents included on the staff hearings document list, along with the other ones that we uh accepted during the hearing, and without objection so ordered. That being said, and without objection, subcommittee is adjourned. Thank you all.