Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role in Advancing U.S. Drug Development.
Legende
Markierungen im Transkript
- Text Aktuell abgespielter Satz
- Text Suchergebnis
- Text Redebeitrag, auf den der geöffnete Link verweist
Klicken Sie auf einen Satz, um das Video ab dieser Stelle abzuspielen. Markieren Sie Text, um ihn mit Redner, Zeitstempel und Link zu zitieren.
Weitere Optionen
House Energy and Commerce Subcommittee on Health U.S. House of Representatives
Ihren Sitzungen folgenDiana DeGette
FDA reversed direction, demanding a new trial for the product, even though advocates and scientists believe the new trial to be unethical. Luckily, with the evidence that McCary 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 McCary 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 products to market.
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 FDA policy on these issues. I'd like to enter both my letter and the agency's response in the record. I'd like to ask unanimous consent, Mr. Chairman.
Without objection, so ordered.
Diana DeGette
I hope this is 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, as our capabilities evolve, so must our regulators.
And so needless to say, Mr. 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 21st century, and I yield back.
H. Griffith
Gentleman yields back, now recognize the chairman of the full committee, Chairman Guthrie, for five minutes for his opening statement.
Brett Guthrie
Good morning, and thank you, Chairman Griffith, for holding this hearing on this important topic, and the ranking member DeGette, thank you for doing this. Additionally, thank you to all of our witnesses for being here. 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, and 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 biotechnology companies, their clinical trials, and domestic manufacturing. Our ability to develop and manufacture life-saving medicines, vaccines, and other medical products here in the US depends on a strong domestic biotechnology sector and a regulatory environment that supports innovation while preserving FDA standards for safety and effectiveness. Bringing new therapies to patients requires clinical trials that are 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 FDA user fee authorizations, this committee has worked in a bipartisan way to modernize FDA'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 reauthorization cycle. Thank you, Mr. Chairman, I appreciate our witnesses for being here, and I yield back.
Frank Pallone
Chairman yields back, now recognize the ranking member of the full committee, Representative Pallone, 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 collaboration between our public health agencies, namely the Food and Drug Administration and the National Institutes of Health, and industry partners to ensure that our gold 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've 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 life-saving cures. The Trump administration has slashed billions of dollars in critical research funding.
This funding was already congressionally approved, but Trump wasted no time in abruptly and arbitrarily cutting the funding from research institutions and dismantling federal scientific agencies. Experts warn that these funding cuts destabilize 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 FDA for approval. Without this initial public investment into NIH, the pipeline of drugs will slow. In fact, the Congressional Budget Office estimated that a 10% cut to NIH's budget would result in 20 fewer life-saving drugs approved over 10 years.
But the administration's threats to advancing drug development do 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, and I've asked FDA 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 staff, reports 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 glad we're 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 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 Griffith, and I yield back.
H. Griffith
Gentleman 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 and willfully making materially false statements to the legislative branch is against the law under Title 18, Section 1001 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 Dr. Cynthia Verst, who is the president of Design and Delivery Innovation R&D Solutions at IQVIA, Ms. Susan Winkler, 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.
Diana DeGette
Thank you. Thank you, Mr. Chairman. I'm so happy to introduce my friend Dr. Aaron Kowalski, who's the chief executive officer of Breakthrough T1D, which is the leading global organization driving research, advocacy, and cures for type 1 diabetes.
Many people on this committee know my daughter Francesca, who was diagnosed with type 1 diabetes very early in my congressional career, and also Dr. Kowalski and his brother both have it. They, frankly, when they were all diagnosed, 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 Dr. Kowalski's 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. And I know that under his leadership, Breakthrough T1D continues to bring researchers, industry, regulators, and advocates together.
And I just want to say while I have the floor, thank you to Dr. Kowalski, but thank you to all of our witnesses here today and everybody in the room. We are on the cusp of exciting, exciting discoveries, and this committee needs to partner with all of you in how we do it. Okay, I yield back.
H. Griffith
And I appreciate it, and I'd also appreciate all the witnesses being here today. The next witness to be introduced is Dr. Thomas Wang. Did I get it right? All right, he says I got it right. And is an assistant professor of medicine at Brigham and Women's Hospital and Harvard Medical School.
And last but not least, Dr. Thomas Bolly is the Bloomberg Chair in Global Health, Senior Fellow for International Economics, Law, and Development, and Director of the Global Health Program, Council on Foreign Relations. 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, Dr. Cynthia Verst, for her five-minute opening statement.
Cynthia Verst
Chairman Griffith, Ranking Member DeGette, and members of the subcommittee, thank you for the invitation to present to you today. I'm Dr. Cindy Verst, President of R&D Design and Delivery Innovation for IQVIA, a leading clinical research service and commercial insight provider to the life science industry. Our 90,000 employees work every day 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, 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 data points and recommendations. Firstly, the United States remains the most sought-after market for new medicines and thus is a critical destination for clinical trials. And although our data does confirm that China surpassed the US share of Phase 1 innovative industry trial starts in 2021, at the end of 2025, China accounted for 35% and the US 27% of Phase 1 trial share. And looking more closely at the data, I'd like to share other insights.
China's Phase 1 growth is fueled by Chinese-headquartered sponsors, that is, by China, for China, and where their homegrown information relative to innovation is translating at scale from bench to the clinic.
In fact, 89% of phase one trial country uses by Chinese companies in 2025 were actually in China, whereas China's inclusion as a country for US and EU headquartered companies in phase one trials were only about 5% 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 ex-US first-in-human starts. Consistently our sponsors, particularly emerging biopharma companies who drive over 75% 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 ex-US 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 Trailblazer. Congress should consider encouraging the definition and use of fit-for-first-in-human minimum datasets 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 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 data integrity that make the FDA the global gold standard. And of course IQVIA's continuing to be committed to help the Congress, FDA, HHS, and of course other stakeholders to enhance this clinical research efficiency and most importantly, helping our patients receive medicines faster.
Thank you.
Redner 4
The lady yields back and I appreciate that. Now recognize Ms. Winkler for her five-minute opening statement.
Susan Winkler
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 many vantage points, including as FDA's Chief of Staff under both Republican and Democrat commissioners. Part of my role there involved negotiating product safety agreements with China. The Foundation is a nonprofit created by Congress in 2007 to advance regulatory science and support FDA's mission. So thank you for creating the organization.
The insights I offer today 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 Trailblazer. This is encouraging.
When the people who run trials, treat patients, and generate evidence arrive on their own 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 Dr. Verse mentioned, and triaging the review can accelerate routine INDs and focus limited FDA resources to higher-risk INDs, 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. 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 reimagine the screening mechanisms for clinical trial participation. Reimagine 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 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 will continue to support this ecosystem, and we stand ready to help.
Redner 8
Thank you very much, and I now recognize Dr. Kowalski for his five-minute opening statement.
Aaron Kowalski
Good morning, Chairman Guthrie, Ranking Member Pallone, Chairman Griffith, and Ranking Member DeGette and members of the subcommittee, and thank you, Ranking Member DeGette, for your kind introduction and for all you've done for your daughter and our community. Thank you for the opportunity to 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 Dr. Aaron Kowalski, Chief Executive Officer of Breakthrough T1D, formerly JDRF, the leading global research and advocacy not-for-profit organization for type 1 diabetes, or as we say, T1D. Our purpose is to make everyday life with type 1 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 T1D community.
I'm a scientist by training, but I also live with type 1 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 20 years, and I can say with pride that my family and our broad type 1 diabetes community have 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 T1D community needs.
Type 1 diabetes is an autoimmune disease that can affect anyone at any age. Once you have type 1 diabetes, you rely upon insulin for the rest of your life. Without insulin, type 1 diabetes is fatal. Insulin, though, is not a cure for type 1 diabetes. The disease is unrelenting to manage, even with the amazing progress that we've made over the last number of years.
It's 24/7, 365, 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 T1D has invested over two and a half billion dollars in type 1 diabetes research to try to solve these problems. But we can't cure type 1 diabetes alone.
We're so grateful for the bipartisan leadership of Representatives DeGette and Bilirakis and the strong support of this committee for continued support of T1D research and the Special Diabetes Program. America has a long and proud history of leading T1D research, innovation, and treatments. But the rest of the world is catching up. I saw this firsthand during recent visits to China in December and Australia in April. That said, our global leadership in T1D innovation is ours to lose.
America is well-equipped to continue to lead. NIH research funding and FDA regulatory approvals are the lynchpins 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 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 trials are another challenge. C-peptide, for example, is the best, simplest measure of your body's insulin production. Yet in clinical trials, FDA hasn't granted full approval for using C-peptide as an endpoint.
Antiquated requirements needlessly prolong and complicate clinical trials and can deter innovation and investment. I commend the recently announced Trialblazer pilot aimed at modernizing clinical trials. Breakthrough T1D has also stepped in to help by publishing roadmaps for T1D 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 leadership in biopharmaceutical innovation and manufacturing. An FDA with these qualities will be ready for the next wave of T1D 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 T1D. My message is simple. We have the capacity to end type 1 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.
Thomas Wang
Chairman Griffith, Ranking Member DeGette, 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 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 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 1 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 2018, 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, again in 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 FDA 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-US 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 US. 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 FDA has recently launched an initiative to provide more information on why new drugs are not approved, including publicly releasing complete response letters. Congress should provide explicit statutory backing for the FDA'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 FDA remains the global model for 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 FDA inspection. Third and finally, as part of the FDA's Operation Trialblazer, the FDA could pilot coordinated 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 investment in public support for foundational research and adequate staffing at the FDA.
Thank you for the invitation, I welcome any questions.
Tom Boyky
Chairman Griffith, Ranking Member DeGette, distinguished members of the subcommittee, thank you for this opportunity to testify today. I'm Tom Boyky, the Bloomberg Chair in Global Health at the Council on Foreign Relations. I recently co-led a year-long analysis with biopharmaceutical specialists, China scholars, and industrial policy experts to identify the true scope of US dependence on China in the biopharmaceutical 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 biological targets, to refine them, leapfrog more slowly moving US competitors, and to attract vital licensing deals. The results speak for themselves. China's share of global clinical trials starts has risen from 1% in 2009 to 30% in 2024, rivaling the US share.
By one estimate, one-third of the pharmaceutical industry's global licensing spending in 2025 involved assets originating in China. If this trend continues, it threatens to reallocate the early-stage biopharmaceutical 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's a risk to public health if the US capacity to do innovative drug development diminishes and we cannot rapidly produce effective countermeasures in a crisis, as occurred in COVID-19. 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 healthcare providers may not be able to fully rely on the resulting data. One recent study found that just 20 GCP inspections, less than 1% of FDA's GCP inspections between 2016 and 2023, occurred in China, while 93% 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 first-in-human studies, such as Australia has 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 biomanufacturers 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. Rehiring is occurring, but FDA staffing is down from 2025.
The agency has lost real experience and expertise.
Adding a user fee 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 FDA and congressional intervention would be effective and is thus more urgent. The time to act is now. I thank the committee the subcommittee for the opportunity to testify and stand ready to answer any questions.
H. Griffith
I thank you and all the witnesses. We will now begin questioning. I'd ask that members not begin a new question, let me underline that, not begin a new question with about 10 or 15 seconds to go because that would hopefully move us along a little quicker. I would encourage members to submit written questions for the record to for the record and and I will tell everybody I've already leaned back and told my team a couple of questions for the record because I know I can't get to them all that I want to 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 going to start with Dr. Verst.
Dr. Verst, the appreciate you talking about the IND and the FDA has recently put out a proposed expedited investigational new drug program to accelerate clinical research and development. Do you think their plan will work or do we need to do more?
Cynthia Verst
I I do think that it will take the collective 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.
H. Griffith
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 it we don't know if it works for what it says it's going to work for, but the risk is so low if you want to try it you can and create a new standard. Should we do that? because that would then give us the data to whether or not it works. Your thoughts.
Cynthia Verst
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, 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 along processes and reviews, but not jeopardizing patient safety nor data integrity along the way.
H. Griffith
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, I'll do a written question on that one. Ms. Winkler, you highlighted how trial capacity is currently unevenly distributed across the country with many patients living a long way from the trial site. Well, that's that welcome to my district. I'm 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 because you're closer to Cleveland than you are to Charlottesville even though you're in the state of Virginia. So what can we do to get more sites involved in the clinical trials and I specifically want to look at community health centers, particularly federally qualified ones. What say you?
Susan Winkler
One of the ways to enhance access and so to get into areas where they aren't today is to make better use of and clarify the rules for what's called a hub and spoke model, right? So that you have the really important but paperwork-heavy responsibilities of the hub, thinking about this is how we run trial sites, and they hold some of that responsibility for the spoke 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 and the oversight of the hub, but it helps structure the mechanisms so that you aren't trying to recreate in a local area where you might have just a couple of physicians who could do some things. Let's empower them to do those some things in collaboration with a hub.
Susan Winkler
Yes, there are definitely some opportunities in remote monitoring and telehealth.
H. Griffith
Thanks. Dr. Wang, your thoughts on how we can get more sites like community health centers involved in these early-stage clinical trials.
Thomas Wang
Thank you for the question. What we know from the Australian experience and from other countries is that public investment in our hospitals and health systems is critical to improving the capacity that they have in running clinical trials, and having a centralized network of these institutions that could be centers of excellence could speak to kind of your concerns today.
H. Griffith
All right, if you can do it in 30 seconds, Ms. Winkler, how can using real-world evidence or natural history studies in trial designs help new treatments for rare diseases?
Susan Winkler
It could be very helpful in helping to structure the trials as well as to after perhaps a product is available to do follow-up and make sure that we have the performance that was predicted in the initial piece. So that data both before submission and approval and after approval, we need to make better use of all of that information, this rare data.
Diana DeGette
I appreciate it. You did great. I now yield back and recognize Ms. DeGette, ranking member of the subcommittee, for her five minutes.
Thank you so much, Mr. Chairman. And thanks to everybody for their testimony. Really, it's very rare that we see people from all aspects of the industry sort of rowing together in the same direction, and I think we know what we need to do. The federal government and the FDA in particular really do have an immense impact on biomedical research and with grants and other collaborations. But as all of you know, our resources are not unlimited, so we have to fund high-quality basic research and impactful public health projects.
Dr. Kowalski, I wonder if you can talk to us about the importance of merit-based peer review process.
Aaron Kowalski
Well, merit-based peer review is fundamental to making good decisions that are unbiased, that have input from expert scientists and are aligned with the unmet needs in science to drive towards clinical benefit. So it's fundamental to everything we do in science.
Diana DeGette
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 review process be?
Aaron Kowalski
I think any scientist would tell you that we need to keep politics out of science. It's critical that the science is evaluated on the merits, on the evidence, and that we drive forward without the influence of politics.
Diana DeGette
And so you think that the proposed rule would really adversely impact the peer review process?
Aaron Kowalski
I think so and we've signed on with a coalition to lodge our feelings about this issue.
Diana DeGette
Okay. Now international research collaborations have made important contributions to the science of type 1 diabetes, isn't that correct?
Absolutely.
Diana DeGette
And how would that proposed rule treat those international collaborations?
Aaron Kowalski
Well we've seen already in a number of groups that we fund, 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 agreements through NIH multi-center study grants and those barriers we absolutely do not want to see happen.
Okay. Can you just briefly tell us about the TEDDY study?
Aaron Kowalski
So this is an amazing study, it's one of the largest databases currently at NIH that's aiming to identify the causation of type 1 diabetes. Type 1 diabetes has a genetic component, but even an identical twin who gets type 1, it's only about a 50% risk the other one will get it, which says there's something in the environment that is causing type 1. When we look at the use of AI in the future, I really believe the combination of genetics and this environmental data from TEDDY will really help us understand what's causing type 1 diabetes.
Diana DeGette
So one of the provisions of the proposed rule would let political appointees terminate grant awards, so that would effectively kill projects at any time for any reason. What would happen if the TEDDY, this huge TEDDY collaboration that you're talking about, were cut off halfway through because maybe a political appointee didn't like the word environmental?
It would be a travesty.
It would totally destroy the results of that study.
Aaron Kowalski
I mean we haven't, we don't, we've certainly benefited from TEDDY with a number of seminal learnings, but the ultimate goal of understanding the genesis of type 1 diabetes is not there yet and that is one of the most important studies that would help us get there.
Diana DeGette
Dr. Hwang, I want to ask you quickly, what kind of work does FDA fund externally?
Thomas Wang
The FDA has a lot of important grant mechanisms, specifically in the Office of Orphan Products. FDA grants to rare disease clinical trials, including those for ALS, have supported new product approvals and all of that research is at risk.
Diana DeGette
And in your view, if this rule I've been talking about were implemented, would that undercut that external work?
Redner 20
Dr. Kowalski and Hwang, do you think, and I'm just going to ask you, do you believe the proposed rule should be withdrawn? I'll start with you Dr. Hwang.
Redner 17
I'm still reviewing the proposed rule, but as written I've serious concerns before it's implemented.
And Dr. Kowalski?
I think we share those concerns.
Okay, thanks. Thank you so much Mr. Chairman, I yield back.
Brett Guthrie
Gentlelady yields back, now recognize the chairman of the full committee, Mr. Guthrie of Kentucky, for his five minutes of questions.
Thank you very much. Thank you to all. So Ms. Winkler, the Reagan-Udall Foundation has extensive experience convening stakeholders to identify opportunities to accelerate drug development, particularly in areas where there are few treatments such as psychiatric substance disorders, particularly psychedelics, what 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?
Susan Winkler
So there's a few opportunities, but in particular assuring that the endpoints that we're pursuing, so what the treatments intend to affect, match what patients are looking for.
So that's one of the components. And then there's also the important conversation between the product developers and the regulator for what should be measured and how do those clinical trials need to be conducted. There certainly have been a number of those conversations that have taken place and now, as that work continues, it'll be important to share the learnings as some products move through that process, what can other developers... Because one of the issues with psychedelics is you can't really do a placebo trial because obviously if you've taken a placebo psychedelic, you know you're taking the placebo psychedelic.
Redner 16
Right, that is one of the challenges. So FDA has a guidance on that to try and help parse out, you know, how the trial should be done so... They just did that in the last couple of days. You like the guidance they put out in the last couple of days on that? You think that's going to be effective, their guidance?
Susan Winkler
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 am aware that there were constructive conversations that helped make sure that it was reflecting the needs of the community.
Brett Guthrie
So we have a number of colleagues in Congress who were combat veterans and have had this treatment and have lots of friends who have talked of this treatment and feel it's really effective and we need to, hopefully we can figure out a pathway if it is effective to find it's effective and give access if so. So thanks. So Dr. Kowalski, in Breakthrough T1D, you've worked closely with FDA and product developers to advance all the innovative clinical designs and trials with endpoint development for therapies and ultimately to benefit patients with type 1 diabetes. Can you describe how early collaboration between the FDA, sponsors, and patient organizations contributed to that success and what lessons can be applied more broadly to accelerate development?
Aaron Kowalski
Thank you, Mr. Chairman. This is such an important piece I think of the progress we've seen in type 1 diabetes has been a collaboration between regulators and patient advocacy groups like ours. There's a hesitancy often in medicine to take risk, but I think one of the underappreciated things is living with type 1 diabetes is risky. And we certainly saw significant progress made back when I worked 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.
Brett Guthrie
Okay, thank you. And so Dr. Verst, we've heard increasing concerns that other countries are reducing the time it takes to activate clinical trials and enroll patients, making them more attractive locations for early-stage development. What are the potential risks if we as Americans fail to remain competitive at clinical trials and what particularly, so the risk and what we can do to have the greatest impact on ensuring America remains the preferred location?
Cynthia Verst
Mr. Chairman, 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 from a China perspective and that is more translational, if you will, medicine and science with more assets, drug discovery occurring early in the process. And then as I mentioned in my remarks, simplifying and importantly accelerating and doing more of that adaptive approach, more of the risk 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 much more preemptively, even in the pre-IND phase and being more, if you will, predictive and reliable, especially because of the backing of investors, they really encourage that FDA feedback and insights.
Brett Guthrie
We need to have the risk approach because, I mean, if you're just trying to cure the sniffles, you want a lot of tests to make sure there's no side effects. But that's what the President is for in the first term and now pushed right to try. Some people are in a situation where this is their only hope, and this is something that's going to cost them their lives. And so we, and then with our suicide, that's what 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.
The gentleman yields back, and I'll now recognize the ranking member of the full committee, Mr. Pallone, for his five minutes of questions.
Frank Pallone
Thank you, Chairwoman. I mentioned in my opening statement we need to ensure there's a robust research infrastructure in the U.S., 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 politicizes the science and threatens the independence of scientific research in the United States. On Monday, a group of 57 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 ask unanimous consent, Madam Chair, to introduce this into the record, if you will.
Without objection, so ordered.
Frank Pallone
Thank you. So every Democrat on this subcommittee wrote to the OMB director earlier this week to rescind this politicizing rule. I wanted to ask Dr. Hwang, your testimony speaks to the vital role of U.S. 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 U.S. 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 vital role, and then I have a second question, Doctor.
Thomas Wang
So multiple studies have shown that virtually every new drug that enters the U.S. market is supported by NIH science. Nearly a third of all federal patents are based on federal research. And we can't hope to increase early-stage clinical development without sustainable investments in our science.
Frank Pallone
Well, thank you. But what evidence have you seen regarding the rising Beijing Chinese contributions to early-stage clinical development?
Thomas Wang
So what our study has shown, in collaboration and in knowledge of other studies that are going on as well, is that it's not declining U.S. productivity in 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 U.S. cancer drug approvals, now a significant portion of them are being done in China and being transported here.
Frank Pallone
Is also part of this brain drain, you know, we keep reading about scientists that would normally, you know, either come to the United States or even American scientists that are leaving the country to go elsewhere, China, Western Europe, elsewhere. How is that contributing to the drug development or lack thereof or slowing here in the U.S.?
Thomas Wang
I think Congress has an important role in making the U.S. continue to be a leading destination for the world's best scientists, and I call upon this committee to help that.
Frank Pallone
All right, thank you. I'm also concerned that we're undermining the impact of, or underestimating, I should say, the impact of our dependence on Beijing. So let me ask Mr. Boyki, in the report you published that you note that we need, quote, a combination of measures to improve U.S. 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 for promising interventions shifting away from the United States to Beijing, China?
Tom Boyky
Thank you for the excellent question. So, part of it is a decline in US competitiveness, not just vis-à-vis China for early stage clinical trials, but also Australia and other markets as well. It is a cumbersome, burdensome process under the IND approach and that has helped encourage activity to go abroad. But make no mistake, 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 many of the witnesses have discussed here, to invest in making the US more competitive, we need to acknowledge that FDA acceptance of foreign clinical trial data depends on its compliance with good clinical practices, that it's conducted under independent ethics review and the ability of FDA to inspect. And we cannot maintain that standard for the amount of activity in China. 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.
Earl Carter
Thank you, the gentleman yields back and I recognize myself for five minutes. You know, we've got two pharmacists on the panel and as a pharmacist, I've spent my career helping patients benefit from innovative medicines. But innovation only matters if patients can safely access it. So I'll start with you, Dr. Verst. Your testimony notes that Chinese companies overwhelmingly conduct their phase one work inside China.
Should Congress view this primarily as a regulatory challenge or as industrial competitiveness challenge?
Cynthia Verst
I think it could be both in terms of the regulatory challenges and in ensuring, for instance, at the end of the day, we talked about representativeness. So increasingly, we're seeing more global trials being executed outside of China by Chinese companies. And accordingly, you know, we pay very close attention, provisioning services to help them understand the regulatory implications at the FDA level. For instance, representativeness, which is being so critical in the data application. And I also think, you know, from just a competitiveness perspective, that this phase one is, quite candidly, close on the heels is phase two.
Right now, the comparison of phase two trials in China as compared to the US is increasingly becoming comparable. So I think both threats.
Susan Winkler
Okay. Ms. Winkler, stakeholders often tell us that one FDA review division may expect something entirely different from another. So how important is it 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 different organ system, a different impact. 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 inconsistencies are explained and provided in context.
Earl Carter
I gotcha. Dr. 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 40 years, there's a problem with APIs when you have them do what they're doing and with everything, drop the price, corner 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 the FDA may have limited ability to inspect?
Thomas Wang
Thank you. I think both of those should be the focus of this Congress. And specifically on the latter, as we heard from other members of this panel, the FDA does not inspect 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 for data integrity and for safety. And so we should empower the FDA and substantially appropriate funds to do so, to do those inspections.
Earl Carter
Yeah, you know, I've talked to drug companies recently and they have been to China. They say their industrial facilities 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. Mr. Bolke, you argued 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 capacity is now becoming a biosecurity issue and not merely an economic issue?
Tom Boyky
Thank you for the excellent question. So we have seen in other space China exercise a willingness to leverage dependence on its supply chains and its system to gain economic or strategic advantage over other nations. 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 support pharmaceutical manufacturing. They have undermined India's attempt to develop supply chain resilience in the active pharmaceutical ingredient and key starting materials space.
They easily could do so in this context, so it puts us at real risk.
Earl Carter
Yeah, absolutely. Agree with everything. One point that stood out in your testimony, sir, was the limited number of FDA inspections conducted in China. And I have 11 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 don't know.
This is a problem and I came to Congress to fix this and we're going to 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 yield back and now I recognize my friend from California, Dr. Ruiz, for his five minutes.
Raul Ruiz
Thank you. Thank you so much. We must first and foremost ensure the effectiveness and the safety of new innovations during the clinical trial process. That's ground zero. 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, okay?
So many barriers prevent patients from participating in clinical trials, especially individuals living in rural and underserved areas, working class, poor families. There are considerable financial considerations, like can someone afford taking time off of work to participate in the trial? Can they afford the transportation to the trial site and copays for the follow-up in the clinical trial visits and afterwards to the doctor? And there are also basic geographic considerations.
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 many patients may welcome, especially for end-stage illnesses in the first place. So these are barriers that we can and should address. That's why I introduced the bipartisan Clinical Trial Modernization Act, H.R. 3521, with Representative Pfluger 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 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 52 million patients in underserved communities, rural areas across the nation.
Given their broad reach, CHCs can bring research closer to where people live and receive care and greatly expand delivery of clinical research to underserved populations. Ms. Winkler, in your written testimony you talk about a patient-centered 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?
Susan Winkler
So one of the challenges in clinical trial recruitment is that patients 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 reimagining and having a patient-centric system would in certain disease areas, you know, deploy against 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 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 thinking about the patient and what trials might be appropriate and making sure that that information and the ability to do research is there local with the patient.
Redner 23
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 to these trials, to these drugs. Dr. Verst, what can this committee do to encourage greater participation by community-based providers such as CHCs in clinical research?
Cynthia Verst
Yes, it's a terrific question, and I do think it's another one of these multi-pronged approaches, no silver bullet per se. I think ensuring that the community physicians associated with healthcare systems are being encouraged and supported with regard to even referral opportunities.
And, and of course this all is working across the healthcare ecosystem in order to be a reality.
Raul Ruiz
And if I may just one second, point of privilege, Dr. 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.
Redner 24
Thank you. Thanks, gentleman, for yielding back and now recognize Dr. Joyce for his five minutes of question.
John Joyce
Thank you, Mr. 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 1 diabetes.
A delayed diagnosis of T1D 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 T1D 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 20 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 Dr. Kim Schrier on the Screen for Type 1 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. Dr. Kowalski, what is needed to prevent patients from reaching deadly symptoms prior to ever being diagnosed with type 1 diabetes?
Aaron Kowalski
Thank you for that amazing question, Representative Joyce. This is such an incredible priority for us at Breakthrough T1D and thank you for your leadership. I just came from a meeting in Colorado where data was presented that people who aren't screened for risk, who don't know they're at risk for type 1 diabetes, have a, approximately 50% 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, 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. This is preventable.
When screened, that risk goes from about 50% to less than 4%, saving significant money, potential lives, and we can screen.
So we are doing significant work to make sure that screening happens, that people are aware, and ideally that every American is screened for risk.
John Joyce
Dr. Krawczyk, 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 T1D. What would it take from the federal government to increase screening infrastructure and ensure that right now that we seize on that opportunity?
Aaron Kowalski
I think this is 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 a fully functional and robust FDA. These therapies are not a matter of if, they're a matter of when, and we want them to be when soonest here in the United States.
John Joyce
In your opinion, the legislation that I'm working with Dr. Kim Schrier, the Screen for Type 1 Diabetes Act, would that help advance screening practices and promote the early diagnosis of all the goals that we have discussed here this morning?
Thank you. Mr. Chairman, the Screen for Type 1 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 1 diabetes. I thank you all for being here today. Mr. Chairman, I yield the remainder of my time.
100%.
John Joyce
I thank the gentleman. And now recognize the gentlelady from Illinois, Ms. Kelly, for her five minutes.
Robin Kelly
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 healthcare. Patients and families across the country count on our scientific infrastructure to develop life-saving treatments, which is why it's so important to invest in biomedical research while simultaneously supporting access for those who need it most. In 2022, 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 raise the bar for new FDA applications to submit a plan to improve the enrollment of clinically relevant participant participants from historically underrepresented populations.
The FDA issued proposed guidance for diversity action plans in June 2024. In January 2025, 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. Dr. Verst, IQVIA report, Advancing Diversity in Clinical Development Through Cross-Stakeholder Commitment and Action, highlights, and I quote, "Failure to include adequate representation from key subpopulations risks 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?
Cynthia Verst
Thank you. That's a terrific question. And diversity and representativity is critical for good science. And as you mentioned, diversity action plans are absolutely critical for not only ensuring efficacy and safety of diverse populations and, of course, increasing probability of regulatory success.
It's obviously very important too to be concentrating on protocol designs, so starting with the end in mind for that good science. And that also equates to operationalization of clinical trials, understanding, for instance, 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, if you will, nuances associated with even the eligibility criterion and clearly how they are treated through the schedule of events, etc. So it's absolutely critical, and in fact, it is good science.
Redner 27
Thank you so much. Dr. Kowalski, can you discuss the importance of clinical trial diversity for participants with type 1 diabetes?
Aaron Kowalski
Oh, it doesn't, type 1 affects all races and socioeconomic classes, and I'm really proud, a number of questions have come up on this. We mandate in our grant agreements that trials are representative of the broad population. Chairman Griffith, you asked about rural areas. We've seen in federally qualified health systems, we have a program at University Hospital in Newark, for example, that are having some of the best recruitment for trials. It can be done.
And I think there are mechanisms, especially from funders and from government, to help push to make sure that everybody's represented. I completely agree with my panelists here that we have to look at all populations to fully understand the impact of new drugs and new therapies. Often they don't work in every population, and we have to study the broad population to get that information.
Redner 28
Thank you so much. And actually, my district, I represent the Chicagoland area, but my district is urban, suburban, and rural, so the south side of Chicago and then 4,500 farms as you go further south. So rural is very, very important to me also. 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 through 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 2022.
And Mr. Chairman, I yield back.
Redner 26
The lady yields back. Now recognize the gentleman from Georgia, Mr. Carter, for his time.
Earl Carter
Thank you, Mr. Chairman, and thank each of you for being here. This is extremely important, and I appreciate all of your expertise here. You know, I've been around now for 12 years, so I've seen a lot of what has happened here in the drug industry and a lot of the policy that has shaped the drug industry in real time. And it's 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 why did the United States all of a sudden take over that lead? And one of the things that happened was the bureaucratic burden and the price controls 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 practiced pharmacy for over 40 years and I saw nothing short of miracles as a result of research and development.
And we want to continue with that. And we want to continue to make sure that 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 China and about them taking a lead. But we can't, we can't beat China by copying China, because China does not operate under the same rules and regulations that we do.
We all understand that.
We're going to beat them the same way that we beat Europe, and that is by doubling down on what actually made America the leader in the first place: entrepreneurial spirit, access to capital, free markets, and the best science in the world. And I still say we have the best science in the world right here in the United States of America. Dr. Vorst, I want to ask you first, from where you sit, what 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 seeding our leadership to China?
Cynthia Verst
Terrific question. I think that most recently, of course, Trailblazer, the Operation Trailblazer, those components, I think, are critical in so much that it does ensure the coalescence, or if you will, all of the HHS divisions coming together. We talked here today about, of course, the FDA and, and I know it's not within the scope, but that adjacency will be critical, especially, for instance, we talked about today community-based practitioners becoming more integral into clinical trials here in the US. And I'll just give a few data points. Oncology, oncology is the world's largest clinical trial market.
Over 40 to 50 percent of the entire global clinical trials resides within oncology. Our oncology clinical trial investigator capacity is being taxed, especially as stated, more of the academic medical centers. We have to get out into the communities. You know, so 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.
Earl Carter
Okay, well let's, let's do kind of shift gears a little bit and talk about the clinical trials because we talked about it a little bit up here about decentralizing it. And that's something that I really do think is, can be beneficial, particularly in using all the healthcare facilities that are available. It was mentioned FQHCs. I would also carry that to be local pharmacies. That's another area we can, remember, pharmacists are the most accessible healthcare professionals in America.
Over 90 percent of all Americans live within five miles of a pharmacy, so we need to take advantage of those type of things. FDA's own guidance recognizes that fully decentralized approaches aren't right for every trial, and I understand that. But Ms. Winkler, I want to ask you, what's standing in the way of scaling decentralized trials in the US today, and what can FDA and Congress do to remove those barriers?
Susan Winkler
So, we've talked about some of the barriers, and some of it is training those local investigators. 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 community health centers. And then there is a basic problem, which is contract and budget negotiation. It wastes time, and it happens again and again. And it may seem 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.
We have to fix it.
Debbie Dingell
I didn't realize that was such a problem. Huh. Okay. Well, thank y'all very much and I yield back.
Chairman yields back, now recognize gentlelady from Michigan, Ms. Dingell, for her five minutes of questioning.
Thank you, Mr. 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 firsthand. At the University of Michigan, faculty, staff, most importantly, the students are telling me, well, we're going to 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 going to be a ping-pong ball.
We have to have a reliable income. And I think that's dangerous for this country, that we're going to 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 drugmakers and patients to get answers about the approval process for treatments that could bring hope to the rare disease community. Dr. 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?
Aaron Kowalski
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 they're making contingency plans. This includes Canada, this includes Europe, Australia, and that's concerning to us.
Debbie Dingell
Dr. Kowalski, 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?
Aaron Kowalski
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 responsibility of private companies. Virtually every advancement in type 1 diabetes originated with the 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 here in the United States and I'm proud of the role some of our funding has played. It takes a lot to get, but cutting funding would be incredibly short-sighted.
Debbie Dingell
Thank you. Mr. Boyki, 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 II, Germany led, we led. I'm told now we are number two in research versus China. Could you comment?
Tom Boyky
Absolutely. Great question. We are already seeing a flow of scientists out of the US. That's a product of the 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 to go through the IND process is due to unpredictability and inconsistency from FDA staff. We've lost staffers, we've lost a lot of expertise and experience and that's affecting our competitiveness as well.
Redner 32
Thank you. I'll yield back, Mr. Chair, and thank you to all the witnesses.
Redner 34
Gentlelady yields back. Now recognizes gentlelady from Florida, Ms. Cammack, for her five minutes of questioning.
Kat Cammack
Thank you, Mr. 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 going to argue about that, but talk about the importance of this leadership and 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 to participate in clinical trials. And as the representative covering the Sid Martin Biotech Hub, a world-renowned facility, this is critically important not just to our national security, but to folks for me back home.
So I'm going to start with you, Mr. Boyke. Did I say that right? Yes. All right. So clinical development is increasingly global, as has been highlighted here today, but the United States can remain a leader in biomedical innovation by strengthening our domestic research ecosystem while continuing to build on those trusted partnerships with countries that share our regulatory standards and commitment to high-quality science.
At the same time, FDA's 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 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 toward next user fee 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?
Tom Boyky
Terrific question. Thank you for it. The FDA adopts a risk-based standard to conducting its good clinical practice inspections. It's hard to square a risk-based approach with where those inspections are happening currently. China, the number of trials 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, has a number of fewer inspections in that environment. Congress should consider instituting a user fee for INDs that involve foreign clinical trial data so that FDA can have more resources to be able to pursue these 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 practice.
Redner 33
And you would agree that we need to continue to develop the incentives for more early-stage drug development here domestically.
Tom Boyky
Absolutely. It really does need to be a combination of making the US competitive and also having better oversight over the activity occurring in China.
Kat Cammack
A thousand percent agree. Thank you. Dr. Winkler, I'd like to shift to another opportunity for reform. Where are you? There you are.
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 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?
Susan Winkler
Yes, so there are a couple of opportunities. First, it's making sure that the sponsors are thinking about what are the endpoints that are relevant to women, or shall we say, are there endpoints that are different by sex, 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 the research, what are the barriers and what sex-based differences need to be explored not only scientifically, but practically in getting then the right mix of participants in the clinical trial. So it requires intentionality and then also looking at things, are there opportunities to explore some of these post-market, right, what needs to be done pre-marketing and then what might be explored and better understood after a product is already on the market, so using real-world data to generate real-world evidence to help us tease out sex-based differences.
Redner 39
Well, and I know I only have 13 seconds, but how do you do that without creating additional burdens on the sponsors?
Susan Winkler
So I think this is part of the upfront and being clear. Where you have particular sponsor burden is when it comes in late, or there was a lack of clarity, or sometimes sponsors don't always listen perhaps as clearly as they should have upfront.
Redner 36
I appreciate that. Thank you. And I will submit the rest of my questions for the record. I yield.
Nanette Barragán
Gentlelady yields back. I now recognize the gentlelady from California, Ms. Barragán.
Thank you, Mr. Chairman. Mr. Boyki, I want to start with you. I have this chart that I used in July of 2025. It's an FDA organizational chart. I don't know if you could see it from where you are, but basically what it is is it has the different sections of FDA.
And when I used this, actually, this is now gone. We've got four more gone. This is now gone. We've got four total. But one of them that's been gone I want to point out is the food oversight.
And why am I bringing up the food oversight? Because I'm getting calls in my congressional office about this concern right now about this explosive diarrhea-causing illness called cyclospora-usis, I don't know if I've got that right, which like going through thousands, it's going through number of states and thousands of people are dealing with it. Do you think that the elimination of these offices within the FDA is a good thing?
Tom Boyky
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 to do its job.
Nanette Barragán
Okay. So you know, right around the same time that I was using this chart, you retweeted this article called Inside the Collapse of the FDA from the New York Times. Would you agree that it's important that we have experienced voices and institutional knowledge at the FDA?
Tom Boyky
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 FDA.
Redner 38
And do you agree that we need to protect the US system of biomedical innovation?
Tom Boyky
Absolutely, it's a priority for biosecurity, for public health, and for patients.
Nanette Barragán
And would we help achieve that goal if we're making cuts to the National Institutes of Health?
Tom Boyky
One of the foundations to the US biomedical innovation system is robust funding for basic research from the NIH.
Redner 46
Cutting NIH funding does not advance our ability to have that basic research that has informed so much of US drug development.
Tom Boyky
Okay, and so if we're trying to protect the US system of biomedical innovation, does it help if we're having and continue to have hostility to international students from abroad?
Nanette Barragán
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.
Okay, so it doesn't help that that's happening. And what about, does it help us be the leaders in biomedical innovation if the United States is attacking research institutions here?
Tom Boyky
Again, the foundation for US biomedical innovation has really been the universities and our basic research. We need to support them.
Nanette Barragán
Right, so it's not helping. And Mr. Boyki, I think you know what I'm talking about. You wrote an article, you wrote an article in which you specifically said, and now I'm going to just quote from it, 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. Yeah, this resource is not inexhaustible. The president's risk squandering 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, 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's collapsing. How about placing blanket tariffs on pharmaceuticals? Is that going to help the US be less dependent on foreign drugs?
Tom Boyky
Tariffs, if deployed, need to target where that dependence arises, and that's really on upstream inputs, 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 allied and well-regulated diversified sources.
Nanette Barragán
And yet again, this administration is doing that, and the Republican Congress is totally supporting that. I'm citing your article of July 22nd, 2025, 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 yield back.
Redner 45
Gentlelady yields back. Now recognize gentleman from New Jersey, Mr. Kean.
Thomas Kean
Thank you, Mr. 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 that not only provides jobs locally but provides the world with therapies that improve and extend life. Ms. Verst and Ms. Winkler, my district is home to numerous small rare disease companies. These constituents and other companies have frequently raised concerns about inconsistency even within FDA review divisions. This is a significant source of uncertainty that translates to an unpredictable regulatory environment and disincentivizes investment 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.
Susan Winkler
Yes, so 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 different product centers at FDA, so Center for Biologics, Center for Drugs, and Center for Devices, and stimulating the conversations so that they can have a better understanding of what endpoint might be relevant in a rare disease and how to use that endpoint. But there are further opportunities. It comes from greater collaboration and conversation with the patient communities to make sure that the endpoints that are being pursued are patient relevant, but then also 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 or 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 looks wrong or unfair, and so learning more and sharing more, so having more of those conversations and sharing the learnings in rare disease, I think is one of the greatest opportunities we have.
Cynthia Verst
Thank you. Terrific question. I think several ways the agency can help on the rare disease front. Notably, over 65% of all clinical trials conducted around the globe, phase one through three, are coming from biotech customers. Biotech customers need more certainty, and of course their investors need more certainty to fund their rare disease clinical trials.
I think that having said that, providing greater clarity and guidance will be very important to try to reduce some of the uncertainty. And I do think that the Operation Trial Blazer addresses many of this in terms of, for instance, 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 draft guidance will help to provide just that more certainty. Thank you.
Thomas Kean
Thank you. 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?
Susan Winkler
There are a couple of different mechanisms. Part of it is clarity on the part of the regulator that we've said repeatedly this morning that it's easier to follow rules that you've heard about and understand and can ask questions. So part of it is the clarity from the regulator in 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, then it makes it easier to extend the clinical trial expertise and reach.
Thomas Kean
And second, how can Congress strengthen FDA's ability in this through the user fee reauthorization next year?
Susan Winkler
So as part of the user fee reauthorization process, you know, there will be important situations emerge where actually revising the statute would be helpful. And and I think that that will there were some ideas today and others that will emerge. And then tailoring the resources that are needed to implement some of those ideas. A statutory requirement for 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.
Thomas Kean
Okay. Thank you. Ms. Furst, there are unfortunately many barriers to mental health care and I'm concerned, excuse me, and I'm committed to eliminating those barriers. One of those barriers is limited development of psychiatric drugs compared to drugs to treat physical health conditions. First, can you speak to the unique barriers faced by psychiatric drug sponsors?
Cynthia Verst
Another terrific question. I think some of the barriers really is patient access and and of course that being hugely important when conducting any clinical trial, but especially psychiatric trials. And so promoting more of the community-based practitioners because clearly academic medical centers alone will not help solve this problem. So increasing awareness with an and within the community, the practicing, you know, physician community in particular and really taking on many of the areas of opportunity that Susan has has mentioned here today would be extremely helpful in accessing and recruiting those patients in the 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 necessary efficacy and safety data to be promulgated new medications for these patients.
Okay. And second, what recommendations would you have?
Redner 43
I hate to I hate to interrupt you, but we'll have to do questions for the record you're way over time.
Redner 41
All right. Well, thank you then. I yield back and I'll follow up with a follow-up question in regard to what we can do in Congress to follow up. Thank you, Mr. Chairman.
Alexandria Ocasio-Cortez
I appreciate it. Thank you. Now recognize the gentlelady from New York, Ms. Ocasio-Cortez.
Thank you, Mr. Chairman, and thanks to all of our witnesses for being here today. I want to talk about one aspect of innovation in health care, which is our pharmaceutical patent system. I've served on many different committees in Congress at this point, Financial Services, you know, Natural Resources, Oversight, and this word innovation 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 juice some numbers. And so I think I want to dig in a little bit on our pharmaceutical patent system because this system is critical to drug development in the United States and ensuring that breakthrough drugs become available to patients here, 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.
Dr. 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.
Alexandria Ocasio-Cortez
And when 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 20 years from the filing, correct?
Right.
Alexandria Ocasio-Cortez
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 and have that return and be rewarded for that innovation, right? That's kind of the gist of the system. But over the years, we've seen some drug companies do some curious things with this. 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 20 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. And we're seeing that in quite a few treatments, correct?
Redner 48
That's correct. 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.
Alexandria Ocasio-Cortez
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 130 patents that gave them continued exclusive market control over rheumatoid arthritis treatments. And during this time, the price for Humira went from $500 for one syringe to nearly $3,000 per syringe. And that led to about $80,000 for a one-year supply for a patient. In fact, AbbVie isn't the only one doing this.
I have an example right here from AstraZeneca. This is an inhaler pump, 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 medication inside did not change at all, the administration of it. 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?
Alexandria Ocasio-Cortez
Yeah. It's this little plastic piece right here. It's one of the big features of that new patent. This thing to keep the cap from coming off was a major part of the new patent that they had filed and to extend and to prevent this life-saving drug from going generic and bringing down costs for everybody.
Thomas Wang
I think very quickly, and I'd be happy to submit more for the record, but these strategies unfortunately reward the very thing that we should be avoiding, which is aggressive legal maneuvers instead of the risky basic science that our country should prioritize.
Troy Balderson
Gentlelady yields back, and I now recognize the gentleman from Ohio, Mr. Balderson, for his five minutes of questioning.
Thank you, Mr. Chairman, and thank you all for being here. My first question is for Dr. Kowalski. Thank you for being here. 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 1 diabetes?
Aaron Kowalski
Thank you for that question. It'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 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 1 diabetes isn't solved, as are 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 the use of more biomarkers. You know, as we look at how fast can we move trials, for example, in some of the even next-gen type 1 diabetes therapies, we've been limited by an old way to look at type 1 diabetes that's not even applicable when you're at risk for the disease, which has really slowed down progress. So, better biomarkers, and I just can't stress enough the risk-benefit barriers. Being risk-averse when people are suffering, developing diabetic complications, living with this grueling disease, we need to take that into consideration and continue to innovate.
Troy Balderson
Thank you very much, and I appreciate your passion with that. My next question is for Dr. Verst. Thank you for being here, ma'am. 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 action should Congress take to ensure that rare disease patients can access innovation research regardless of where they live?
Cynthia Verst
Terrific questions. I think initially, patient access and understanding where these patients, their standard of care, the patient journeys are just critical questions to help us when 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 the natural history of their diseases as well. And in so much as using that real-world data 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 investigator.
And that I think is clearly of importance. And where you can help, and of course again I refer back to Project Operation, the Operation Trailblazer, where there is some clear guidance that you can help us to ensure that draft and revised guidance on, for instance, one randomized well-controlled trial with confirmatory evidence, that helps to reduce the number of patients required for the generation of evidence, and that being so critical to ensure that treatments are getting better treatments to our patients faster.
Troy Balderson
Okay, thank you. I will stay with you. Dr. Clinical trials produce plenty of data, but sponsors frequently report that the process of gathering and submitting this information is still unnecessarily complicated. Where are there opportunities for FDA to leverage digital technologies to reduce administrative burden, improve trial efficiencies, and allow researchers to spend more time developing therapies and less time navigating paperwork? You have about 20 seconds, ma'am, I apologize.
Cynthia Verst
Okay, I'm going to go very fast. Firstly, I think 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, the use of patient-reported outcomes, digital connected devices, and reducing, if you will, the burden on the investigators and patients is just incredibly important. And the good news is that those digital datasets 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.
Redner 59
Gentleman yields back, now recognize the gentleman from Louisiana, Mr. Carter, for his five minutes.
Troy Carter
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 leadership in scientific research and innovation on a global scale. Yet Republicans in the Trump administration have spent the last year attacking the federal workforce while dismantling and politicizing the research that drives the development of life-saving 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 grant-making system in modern history.
These reforms would replace long-standing non-partisan merit-based grant-making processes with 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 fiscally 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.
Dr. 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 the United States' leadership in biomedical research and innovation?
Thomas Wang
The cuts to that federal investment, as you mentioned, Congressman, are tremendously harmful, and they threaten to push back all the leadership that we've gained over the years.
Troy Carter
From your experience as a physician and an academic medical center, what impact can these disruptions have on clinical trials and patient access?
Thomas Wang
It could have a harmful role, for sure, on the initiation of new trials and also creates a freeze over trials that are already in progress. And I just want to stress for the record that these views are my own and don't represent those of my employer.
Redner 55
Fair enough. How do these cuts undermine the global competitiveness, particularly competition with countries like China?
Thomas Wang
Again, I think a loss of investment in American science does a big disservice to our efforts to create new therapies.
Redner 56
Why is maintaining a strong workforce at FDA important to advancing new treatments and protecting patients?
Thomas Wang
As we've heard from other members of this panel, the FDA has an incredible role in the innovation ecosystem, and our leadership over the past two decades relies on a well-functioning and well-staffed FDA.
Redner 55
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 to tell us to do better?
Thomas Wang
But in seriousness, I think the first thing that we can do from the Congress's perspective is reauthorizing the user fee agreements and funding the NIH. And assuming that we have those things in place, I refer to some of the recommendations that I have in my written statement. 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-US trial sites are valid and that they're done with integrity.
Troy Carter
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?
Thomas Wang
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 for new medicines. And I worry that the next generation of world-class talent will leave this country because of those cuts.
Troy Carter
And we must do everything in our power to ensure that the best and brightest that's here stays here, the best and brightest that want to come here to study to find cures for dreaded diseases continue to want to come here, and that's why we must maintain our leadership role in funding for NIH. My time is ended. I yield back.
Redner 53
The gentleman yields back. Now recognize the gentleman from New York, Mr. Langworthy.
Nicholas Langworthy
Thank you, Mr. Chairman. 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 that have improved and 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 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 is in the early stages of drug development.
Redner 60
And with that, Dr. Verst, as you may know, much of the preclinical drug development process still relies on laboratory and animal models for dosing and toxicity studies. As technologies like AI, virtual control groups, and other new approaches 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?
Cynthia Verst
Terrific questions. I think by far new approach methods or NAM is the needle mover in terms of preclinical acceleration of timelines. I think as the agency, the FDA, just recently in Operation Trial Blazer 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 in animal testing, is getting the right dose, the right safe dose. And I think those are the 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 clarity in the actual guidance that will be extremely helpful, and especially biotech, biopharma sponsors that so need this very consistent and clear guidance as they develop their drugs.
Redner 60
I appreciate that, and I'm encouraged by the progress we're seeing 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 untimely deaths of dogs and cats in this country simply because that's the way it's always been done. And if these approaches can provide the FDA with reliable scientific evidence that it needs, I think we have a real opportunity to stop the process of testing on cats and dogs in this country. And I believe there's a mandate that's growing from the American people as the curtain's been pulled back on this process, that they don't want to see this as the way we're approving our drugs in this country. And it's just common sense.
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 and develop medicines, because I want to lead this country to ban animal testing on dogs and cats. I don't want to see it just offshored to China, where they are going to do it without any humanity whatsoever. So, Ms. Winkler, what best practices should FDA implement to make the United States the most attractive environment for early-stage research and clinical development?
Susan Winkler
First and foremost, it's a risk-proportionate IND requirements. So saying for the risk of this product and for the, you know, is it going into healthy volunteers or into patients, what do we need to see in that IND? And also then focusing FDA resources on the more novel interventions where we might have more safety questions. Another mechanism is continuing FDA's efforts in the new alternative methodologies that Dr. Verst mentioned. And in particular, we shouldn't be looking for methodologies that replace what we learn, 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, 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, Mr. Chairman.
Redner 62
Gentleman yields back. Now recognize Mr. Landsman of Ohio for his five minutes of questioning.
Greg Landsman
Thank you, Mr. Chairman. And thank you all for, for being here. I want to talk about biosimilars and, uh, get some, uh, feedback 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, 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 we have proposed, uh, Representative Pfluger and I, is a Biosimilar Red Tape Elimination Act, which would remove these extra steps and lower the cost for patients. Um, Dr. Winkler, uh, given, Ms. Winkler, sorry, given, promoted you. I'm sorry. I don't, I don't know.
She's got a JD. She's got a JD, exactly. Ms. Winkler, you gave that sense, the, you know, she's got to be a doctor. Given the FDA's current framework, what changes at the FDA would best support biosimilars and other generics entering the market? You're very close to this, obviously.
Susan Winkler
Uh, yes. So, um, the foundation facilitated a series of conversations with biosimilar developers who, uh, had not had much interaction with the, the FDA. 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 more conversations where the FDA may be 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 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.
Redner 63
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?
Susan Winkler
Well, so it's approved there and there are, um, some continuing monitoring, but the main challenge that, that we have seen is that it, um, tends to be payer adoption that, uh, presents some of these actually getting to patients.
What does that mean? Sorry.
Susan Winkler
Um, in that if I am setting 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, biosimilars won't enter the market.
Redner 63
If you could change it tomorrow today, is there any one big thing that that would help expedite this that you think makes a lot of sense or?
Susan Winkler
I'm always looking for the one big thing and this doesn't have one. 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 with
Greg Landsman
Our sense is that there is just too much red tape when it comes to these things. And so I guess the question, I mean, you know, the clarity obviously helps, but I it also just seems to me that this is a this has already been tested, it's already been approved, it should just, you know.
Susan Winkler
Well, and the structure for biosimilars is, 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 perhaps applying those learnings and changing the standards it may be time.
Greg Landsman
Dr. Kowalski, could you speak to the importance of bringing more of these biosimilars generics to market for patients, especially, you know, we've talked a lot about capping the cost of insulin for seniors and how this could increase competition and lower cost.
Aaron Kowalski
Yeah, so we strongly support the Bipartisan Insulin Act. Insulin, as I said in my opening statement, is mandatory for life with people with type 1 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 between rent or a car payment and their insulin.
I've sorry I went over, but I appreciate that and I agree. Thank you. I yield back.
Greg Landsman
Gentleman yields back. Now recognize the gentlelady from Washington, Dr. Schrier, for her five minutes of questioning.
Kim Schrier
Thank you, Mr. Chairman, and thank you for that answer about insulin. Anything we can do to bring down the cost of a medication that people like me would die without within days, I think, I'm not going to test it, is really important. So thank you to all of our witnesses. I want to talk first about biosecurity and research and global competition. Seventeen years ago, the share of clinical trials started by Chinese companies was only 1%.
Last year, China-headquartered companies accounted for 39% of global oncology clinical trial starts and 36% of vaccine trial starts. And that is massive growth and they are quintupling on their investment in research and development. There are really practical implications to this. I would just point to COVID as an example. The first Chinese-developed COVID vaccine showed significantly lower efficacy than the one in the US, ranging from 50.4% to about 60%.
By comparison, the first American-made mRNA vaccines were about 95% effective. And American innovation and rigorous standards supported by a fully staffed FDA saved millions of lives during the pandemic. I'm thinking about the next pandemic and if we yield our biomedical leadership to adversaries, the consequences for our country could be catastrophic, including whether our adversaries would even share those vaccines with us. Ms. Winkler, when early-stage cancer and vaccine trial trials 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?
Susan Winkler
So part of the challenge here too is right when we have the innovation here, we're learning here and we're having the local 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 challenge here is how do we better improve the US system so that folks will want to be here because there is a risk of others 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 you know these things that are essential to life. We should be positioning the US for greatest success so that we are less susceptible to such challenges.
Kim Schrier
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 more complicated and scientific elements. 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. And I wanted to just point out first, long before a vaccine ever reaches a clinic, the FDA does a really thorough vetting and the determination of safety and efficacy through gold standard clinical trials. And once it is licensed, it then goes to the CDC's Advisory Committee on Immunization Practices or ACIP, where they look at FDA data, pore over these thousands of pages, and then recommend exactly for whom and at what age and on what schedule a vaccine should be administered to best protect our country.
The ACIP recommendation process has saved millions of lives, but its authority really depends on scientific credibility. And that's why Secretary Kennedy's overhaul of ACIP has been so concerning. When we inject or when he injected politics into a committee meant for objective medical decision-making, we threaten the hard-earned trust that parents and physicians like myself rely on every day. To any of the panelists who want to comment just in 25 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?
Susan Winkler
So I'll just make one observation 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 product sponsors do as well post-market surveillance, so the learning continues. So I would just underscore that it's 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 and continuing to monitor for negative effects, which is an important part of the entire medical product regulation system.
Redner 73
Thank you. I want to 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.
Thank you for that.
Lori Trahan
And the gentlelady yields back. And now recognize the gentlelady from Massachusetts, Ms. 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 want to 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. Dr. 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 pipeline?
Cynthia Verst
Indeed. I think that these are critical customers or sponsors, rather, in terms of fueling and promulgating our innovation, as you rightly highlight. In fact, the 75% being phase one in orientation in terms of the predominance of the biotech sector. But in addition, 65% of phase one through three clinical trials globally are actually conducted and invested by this biotech community.
Lori Trahan
And these are the most sensitive to capital constraints. Dr. Boyke, 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 recover? Does it go somewhere else or does it die? Enlighten us.
Tom Boyky
It, uh, science doesn't wait patiently. So, um, 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 conduct first-in-human trials much quicker, to be able to fast-follow the innovations that occur in the US and be able to seek licensing deals for that research.
Lori Trahan
The district I represent is home to companies just like that. I'd like to share one of them. Versatope Therapeutics is a small biotech, just a couple dozen employees that grew out of UMass Lowell's 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. Dr. Kowalsi, Breakthrough T1D 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?
Aaron Kowalski
letter to the both the House and the Senate to request that that proposal be withdrawn. We feel very strongly that letting political appointees override scientific peer review puts breakthrough therapies at risk, as you have described. And having grants terminated for convenience threatens long-time studies and significant progress that were making towards breakthrough therapies.
Lori Trahan
I agree. It's why I led more than 100 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 Vought.
It's dated June 30th.
All right, without objection. Objection so ordered.
Lori Trahan
Thank you. Doesn't seem to me like they're taking full and fair consideration of the almost 350,000 public comments they've received. In fact, this response doubles down on the rule, calling NIH grants too woke and recycling old conspiracy theories. It's just deeply unserious. 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.
Redner 72
And Ms. Trahan, if you'll make sure you get a copy of that so we can have it for the record. And now recognize gentleman from Texas, Mr. Veasey, for his five minutes of questioning.
Marc Veasey
Thank you, Mr. Chairman. I know that much of today's discussion has focused on how Congress can modernize clinical trials and make them faster and more efficient. And I think those goals are very important, particularly as advances in science and technology create new opportunities to bring innovative therapies to patients more quickly. At the same time, I think especially we learned a lot of this lesson around COVID-19, making sure that we ensure that clinical research reflects the diversity of the population that we have here and who's ultimately going to use these therapies is going to be very critical to generating evidence that is both scientifically robust and applicable. Dr. Verse, given your experience designing and implementing these clinical trials across a wide array, how can Congress ensure that efforts to modernize clinical trials also expand participation amongst historically underrepresented communities, again, particularly when you take into consideration a lot of the noise that we had to go through during COVID-19 to get people to just to take that vaccine?
Cynthia Verst
It's a terrific question and certainly a challenge on the clinical trial 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 in participation. And beginning as Dr. Winkler, I give you an honorary degree, 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 put the right technology systems in place within healthcare systems, EMR technology that can 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 clinical trials.
That's just one example of how to access those community physicians and of course their respective patient population to help encourage more participation in clinical trials. And we call that clinical research really as a treatment option.
Redner 78
Wow, this is, yeah.
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 approaches. And I know that this the challenge is ensuring that greater flexibility strengthens the clinical research enterprise while preserving the high evidentiary standards that providers rely on. And Dr. Kowalski, your testimony supports modernizing clinical requirements, including increased use of novel endpoints and biomarkers. Dr. Kowalski, what safeguards are going to be needed to ensure that these new approaches continue to generate reliable evidence and maintain public confidence?
Aaron Kowalski
Sure. I think having a robust FDA that is staffed appropriately and following clear and consistent regulatory expectations will help us lead with safety and then follow with efficacy. I think in our patient community, we've seen 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. 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. Our community is desperate for innovation, and we're seeing it, and we're on the cusp of it.
And I think if we lead with safety, you'll see innovative therapies reaching the population and driving benefit.
Redner 75
The gentleman has yielded back, and we have Mr. Auchincloss on the way. I would ask the witnesses, do you all have time to wait a minute or so? Now, if he's 20 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.
Redner 77
All right. Thank you. Okay. All righty. We had turned the clock on.
We were going to give you another five minutes. You're down you still had four minutes and 30 seconds, but I'm glad you didn't wait didn't push us to the test. I now recognize the gentleman from Massachusetts, Mr. Auchincloss, for his five minutes of questioning.
Jake Auchincloss
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 substantive feedback on and hopefully soon in legislative form. I want to discuss that with the witnesses here, and I've appreciated the written testimony. My first question is for you, Ms. Winkler. We have seen many areas of overlap between the research that Reagan-Udall has done on clinical trials modernization, Operation Trialblazer from the administration, and then our own legislative draft, including risk-based non-clinical testing, patient-centric patient 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.
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?
Susan Winkler
So, key is part of the what you just talked about, where it's a consistent message from what Congress is creating and what stakeholders are saying and what HHS is saying. That seeing the same threads in a common direction is incredibly helpful to the FDA staff. It's saying, yes, this is how we pursue it. And then continuing with that is the clear question of is there clarity in the authorizing statute, then the agency has the opportunity to provide the detail in regulations and the resources and staff to implement it.
Jake Auchincloss
Part of it Congress can legislate, and we should, and our bill really focuses on the things that only Congress can do, particularly creating a point-of-care clinical trial enrollment process, because only 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 trial design, accepted endpoints, consistency. FDA's had a hurly-burly last 18 months, and now has an opportunity for a reset after regrettably hemorrhaging a lot of competence and credibility under the previous leadership.
What advice would you give to an incoming FDA 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?
Susan Winkler
So, obviously that job is an incredibly challenging one, but the 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 expectations of them? And that's that they discharge their work as strong, competent scientists and lawyers and all of the other staff who are experts in 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 really essential communication and interaction with external stakeholders in order to keep pace with the science.
To be a science-based regulator, you have to keep pace with the science.
Jake Auchincloss
Which, you know, of course is what Reagan-Udall has been designed to do, right? And I do think for those FDA regulators, one of the things I hear from investigators all the time is the 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 design. My sense has been that that highly iterative interactive process has degraded over time, and not just I wouldn't say not just 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.
Susan Winkler
Exactly. Some, I think wrongly, call interaction between the regulator and regulated industry as industry capture. I think to be a functional regulator, you have to interact with the industry that you're regulating so that you understand their challenges. The regulator still makes the decision.
Redner 85
Yeah, specifically early on. Well, much more, but I will yield back to the chairman.
Redner 86
I thank the gentleman. Now recognize the gentleman from California, Mr. Mullin, for his five minutes of questioning.
Kevin Mullin
Thank you, Mr. 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 and the FDA's role in drug development. I am very proud to represent California's 15th Congressional District, otherwise known as the birthplace of biotechnology. Life-saving therapies and cures are researched 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 are going to lose a generation of the leading scientific minds if 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 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, Dr. Kowalski, thank you for being here from the patient advocate perspective. Could you please outline what the impacts would be if the next therapy or cure in development for type 1 diabetes, for example, was delayed due to preventable factors?
Redner 81
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, 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.
Redner 83
Thank you for that. We cannot lose the momentum 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, 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 to streamline these interactions and ensure responses are clear and prompt?
Redner 82
Part of it is simply having the opportunity for more interaction, particularly early-stage smaller companies to be able to have that conversation. So, what is the risk of the IND and are you having phase-appropriate requirements for that? And then it is the clarity and speed of communication is essential. And then the, that there are risk-proportionate requirements. 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 the data packages get bigger and bigger because they saw someone else do it.
We have to clarify that in fact that's not the approach that may be needed.
Redner 66
Thank you Mr. Chair for allowing me to waive on and with that I'll yield back.
H. Griffith
Gentleman yields back. That brings us to the end. I would like to thank all of our witnesses again for being here. I was accusing Ms. DeGette of having more people in the back like a clown car and bringing more out but she told me she only had five more and apparently they didn't make it before I closed. I'll remind members excuse me thank you all for being here.
Members will have additional questions for you all subsequent to the hearing. I'll remind members they have 10 business days to submit their questions for the record and I ask the witnesses to respond to the questions promptly. Members should submit their questions by the close of business on Wednesday July 29th. And we see I must have missed a page. We approve all the other items that were on the documents for the there we go.
I ask unanimous consent to insert into the record the documents included on the staff hearing's document list along with the other ones that we accepted during the hearing and without objection so ordered. That being said and without objection subcommittee is adjourned. Thank you all.