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Hearing Hearings to examine FDA bureaucracy, focusing on regulator to roadblock.
senate Aging (Special) Committee · Feb 26, 2026 · Transcript · 65 transcript matches
ANNIE KENNEDY "Thank you, Chairman Scott, Ranking Member Gillibrand, and distinguished members of the committee for convening this critical hearing. I am Annie Kennedy, Chief Mission Officer for the Every Life Foundation for Rare Diseases. I'm honored to be here alongside the hundreds of advocates who've joined us for Rare Disease Week on Capitol Hill. Collectively, we are representing the more than 30 million Americans living with rare diseases. Today, there are more than 10,000 known rare diseases, about 70 percent of which start in childhood. For small patient communities facing progressive diseases,..."
Hearing Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role in Advancing U.S. Drug Development.
house Health Subcommittee · Jul 15, 2026 · Transcript · 20 transcript matches
BALDERSON, TROY "My next question is for Dr. Furst. 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 actions should Congress take to ensure that rare disease patients can access innovation research regardless of where they"
CRS Report
The Orphan Drug Act: Legal Overview and Policy Considerations
Resources · Mar 5, 2024
Just under half of all Food and Drug Administration (FDA) drug approvals between 2017 and 2021 were for orphan drugs, which are drugs used to treat rare diseases or conditions. Historically, orphan...
CRS Report
The Orphan Drug Act and Catalyst Pharmaceuticals, Inc., v. Becerra
Reports · Aug 17, 2023
Orphan drugs are used to treat rare diseases and conditions, but they can be costly to produce and are generally used to treat fewer than 200,000 people in the United States. In 1983, Congress...
Testimony ANNIE KENNEDY
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"Thank you, Chairman Scott, Ranking Member Gillibrand, and distinguished members of the committee for convening this critical hearing. I am Annie Kennedy, Chief Mission Officer for the Every Life Foundation for Rare Diseases. I'm honored to be here alongside the hundreds of advocates who've joined us for Rare Disease Week on Capitol Hill. Collectively, we are representing the more than 30 million Americans living with rare diseases. Today, there are more than 10,000 known rare diseases, about 70..."
Testimony GILLIBRAND, KIRSTEN E.
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"Thank you, Chairman Scott. I really appreciate you calling today's hearing. And thank you for our witnesses and the advocates who are here for Rare Disease Day on the Hill. It makes a big difference that you come together to make sure your loved ones are being heard and that the challenges and struggles that you go through as families and supporters are being understood by lawmakers. Every one of us in this room today knows that when we have a friend or a loved one or a family member who has a..."
Testimony GILLIBRAND, KIRSTEN E.
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"like to China. This is bad for business, and it's bad for patients. If we want the U.S. to remain the global leader in biotech, and we want American patients to have access to these novel treatments, things need to change. Congress must hold the FDA accountable. We must make sure FDA fixes its inconsistent and unpredictable application of regulatory flexibility. It's essential for supporting innovation while upholding the highest standard for safety and efficacy in the approval of these rare..."
Testimony ANNIE KENNEDY
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"We saw, actually, the agency deploy this during the pandemic, the COVID pandemic, where we saw real rapid, real-time resolution to a crisis in our country, and the agency was able to interact with sponsors, get questions answered in real time, and then we saw that CBER tried to operationalize that in other places and spaces. We would really, as a rare disease community, like to see that operationalized within rare disease because we believe that our rare disease community has the urgency and..."
Testimony SPEAKER_29
Lowering Health Care Costs for All Americans: An Examination of the Pr...
house Energy and Commerce Committee · Feb 11, 2026
"FDA practices work. So thank you for affirming what I suspected. I appreciate the answers. It's important that the federal government meet the needs of patients by ensuring the FDA is a reliable partner for drug review and approval. Another area I want to touch on is rare diseases. Despite nearly 1 in 10 Americans having a rare disease, only about 5% of the thousands of rare diseases have an FDA-approved treatment. Given how small some of the patient populations are for these diseases,..."
Testimony SCOTT, RICK
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"in the crowd that are here because rare disease drugs are very important to them, and I want to thank everybody for being here. Now I'd like to recognize Ranking Member Gillibrand for her opening statement. Thank you, Ranking Member. I'd like to welcome our witnesses, experts who are here to talk about how serious this issue is and the steps we can take to ensure patients with rare diseases are not left behind by regulatory delay. First, I'd like to recognize Annie Kennedy, Chief Mission..."
Hearing Lowering Health Care Costs for All Americans: An Examination of the Prescription Drug Supply Chain
house Health Subcommittee · Feb 11, 2026 · Transcript · 3 transcript matches
SPEAKER_29 "FDA practices work. So thank you for affirming what I suspected. I appreciate the answers. It's important that the federal government meet the needs of patients by ensuring the FDA is a reliable partner for drug review and approval. Another area I want to touch on is rare diseases. Despite nearly 1 in 10 Americans having a rare disease, only about 5% of the thousands of rare diseases have an FDA-approved treatment. Given how small some of the patient populations are for these diseases, developing therapies requires innovative thinking and policies to ensure patients have solutions that they..."
Hearing FY27 BIS Budget: the AI Arms Race and the ICTS Office
house Foreign Affairs Committee · Jul 14, 2026 · Transcript · 3 transcript matches
KEAN, THOMAS H. "Could you provide an update on when the Department of Commerce expects to publish the Federal Register Notice clarifying the implementation of the Section 3D zero percent tariff treatment for pharmaceutical products? Thank you. And will you commit to working with me and the Congress to ensure that the implementation of the pharmaceutical tariffs reflect the unique consideration of rare disease supply chains or to protect rare disease patients from unintended consequences? Thank"
CRS Report
Frequently Asked Questions About Prescription Drug Pricing and Policy
Reports · May 6, 2021
Prescription drugs play an important role in the U.S. health care system. Innovative, breakthrough drugs are providing cures for diseases such as hepatitis C and helping individuals with chronic...
Hearing Full Committee Markup of 16 Bills
house Energy and Commerce Committee · May 21, 2026 · Transcript · 2 transcript matches
MATSUI, DORIS O. "in providing transplants for pediatric patients and adults with high-risk leukemia. Thanks to the pioneering work of patients involved in the National Cord Blood Inventory program, cord blood stem cells are also being used and researched to unlock treatments for rare diseases, Alzheimer's, stroke, and more. The AIMS makes important updates to ensure public cord blood banks can manage the inventory of banked cord blood"
Testimony KEAN, THOMAS H.
FY27 BIS Budget: the AI Arms Race and the ICTS Office
house Foreign Affairs Committee · Jul 14, 2026
"Could you provide an update on when the Department of Commerce expects to publish the Federal Register Notice clarifying the implementation of the Section 3D zero percent tariff treatment for pharmaceutical products? Thank you. And will you commit to working with me and the Congress to ensure that the implementation of the pharmaceutical tariffs reflect the unique consideration of rare disease supply chains or to protect rare disease patients from unintended consequences? Thank"
Testimony BALDERSON, TROY
Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role ...
house Energy and Commerce Committee · Jul 15, 2026
"My next question is for Dr. Furst. 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 actions should Congress take to ensure..."
Testimony BRADLEY CAMPBELL
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"want to add? For sure. I think what we keep coming back to you, I think you hear the theme, is you have all the tools in place. You have the advisory committees, you have accelerated approval pathways, and I think the frustration is when they're deployed inconsistently and without clarity from the sponsors and from the patient community and from the physicians in terms of how you ended up meeting the expectation but then not coming to a positive resolution. There's one other piece that we..."
Testimony SCOTT, RICK
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"Good morning. The U.S. Senate Special Committee on Aging will now come to order. Today we're here to ask a simple but important question. Is the FDA doing everything Congress intended to do to quickly get safe, effective treatments to patients with rare diseases who cannot afford to wait? For more than 30 million Americans living with a rare disease, making sacrifices every day is just a part of life. But something they cannot afford to give up is time. Time means the ability to walk. Time..."
Testimony JEREMY SCHWALMAN
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"Every one of us has something. We're all patients. It may not be a rare disease, but this applies to us. We're talking about us, whether it's a rare disease or not. And so the approach that can be taken, including what was started at our institution, a thing called a platform trial, where you can have one small group of controllers and a number of other patient cohorts trying different"
Testimony SPEAKER_38
Lowering Health Care Costs for All Americans: An Examination of the Pr...
house Energy and Commerce Committee · Feb 11, 2026
"There are more than 10,000 known rare diseases that collectively affect about 10 percent of Americans, most of them children, often fatal diseases. Only several hundred of them have cures, so we need to focus on two areas. One is innovation. How do we bring more science, more technology for more biotech companies, allow our largest biotech and pharmaceutical companies to work in these rare diseases? We need to reduce the complexity of our clinical trial system. We need to modernize the FDA so..."
Testimony ANNIE KENNEDY
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"having a girlfriend, and serving his country. And Congress did respond. In that time, Congress has built a framework that incentivizes rare product development, authorizes regulatory flexibility, creates new pathways to approval, and embeds patient experience into review. While fewer than 5% of rare diseases currently have an FDA-approved treatment, nearly 1,400 orphan-designated therapies are now changing the lives of patients and families. But recently, this momentum has shifted. We are here..."
Testimony ANNIE KENNEDY
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"So, first of all, thank you so much, Senator, for being here, and for all of you being here today, and for this important issue. I think, first, it's important to say that I'm not an expert in vaccines, and I'm here really to focus on rare disease. But I appreciate the question really asking about the trends that we're seeing, and us being here really trying to follow the trends and understand what that means for rare disease. I also agree with Dr. O'Neill that we have seen real intention from..."
Testimony BRADLEY CAMPBELL
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"That means when sponsors and regulators work together, the result was an oral treatment option that has saved thousands of patients years of biweekly infusions. We know rare diseases are biologically complex. We know they're difficult to study. As we sit here today, 95% of the more than 10,000 known rare diseases lack an FDA-approved treatment. I think that's statistics many of us are familiar with. But if you fast-forward that pace of development, it will take us 150 years to only treat half..."
CRS Report
Deep Fakes and National Security
Resources · Apr 17, 2023
“Deep fakes”—a term that first emerged in 2017 to describe realistic photo, audio, video, and other forgeries generated with artificial intelligence (AI) technologies—could present a variety of...
Hearing H.R. 9773, Permanent Trump Secure Border Act and Ratification of Subcommittee Assignments
house Judiciary Committee · Jul 21, 2026 · Transcript
UNKNOWN "ICE released his parents one day before his death. In another case, a U.S. citizen with a rare disease died after ICE detained his father and primary caretaker. ICE also detained a DACA recipient while he was on his way to the NICU to bring breast milk to his infant daughter, who was born nearly six weeks"
Hearing Business meeting to consider the nominations of Brett Matsumoto, of Maryland, to be Commissioner of Labor Statistics, Department of Labor, James...
senate Health, Education, Labor, and Pensions Committee · Jul 15, 2026 · Transcript
SPEAKER_05 "Senator, thank you for the question and thank you for meeting with me earlier. Whether it's CJD or CWD, certainly if I am confirmed as a CDC director, I would work with your team to understand what are the capacities of the CDC to look into these types of very rare diseases. I know their resource constraints are, you know, something that is a reality, whether it's with the CDC or any other institution. So I certainly want to make sure that I was not aware of what you just described, but I want to make sure if I am confirmed that I work with your team and the career scientists at the CDC to..."
Hearing Hearings to examine the nominations of Sean Kaufman, of Georgia, to be Assistant Secretary for Preparedness and Response, and Erica Schwartz, of...
senate Health, Education, Labor, and Pensions Committee · Jul 15, 2026 · Transcript
SPEAKER_05 "certainly if I am confirmed as a CDC director, I would work with your team to understand what are the capacities of the CDC to look into these types of very rare diseases. I know the resource constraints are something that is a"
Hearing Fiscal Year 2027 Labor, Health and Human Services, Education, and Related Agencies Bill
house Departments of Labor, Health and Human Services, Education, and Related Agencies Subcommittee · Jun 5, 2026 · Transcript
DELAURO, ROSA L. "of daily necessities, while hollowing out health, education, nutrition, and housing programs that help families make ends meet. Despite these objections, and I would just say very honestly, there are some bright spots in this bill that deserve recognition. First, I am glad to see the overall funding level for the National Institutes of Health is increased under this bill, especially targeted increases for research on cancer, Alzheimer's, ALS, diabetes, women's health, and rare diseases. Second, during our subcommittee hearing on the science of reading earlier this year, I called for this..."
Hearing Hearings to examine the President's proposed budget request for fiscal year 2027 for the Department of Health and Human Services.
senate Health, Education, Labor, and Pensions Committee · Apr 22, 2026 · Transcript
SPEAKER_02 "Through President Trump's Great American Recovery Initiative, HHS is matching compassion with action to help Americans break the cycle of addiction. At HHS, we are prioritizing patients with ultra-rare diseases and their families and driving faster access to life-saving treatments. We're restoring gold standard science and integrity across the"
Hearing “Defending Main Street: Combating CCP Threats to America’s Small Businesses.”
house Small Business Committee · Mar 25, 2026 · Transcript
MR. SEAN MURPHY "honoring the promise that no veteran dies alone. My advocacy for my son and veterans centers on palliative care and access to cannabinoids, rescheduling marijuana from Schedule 1 to Schedule 3 to unlock research and healing, especially for rare diseases. I'm also an advocate against child trafficking, and I implore you to stand against it at home and"
Hearing Member Day to examine S.297, to amend title XXVII of the Public Health Service Act to require group health plans and health insurance issuers...
senate Health, Education, Labor, and Pensions Committee · Mar 19, 2026 · Transcript
JOHNSON, RON "Congress has repeatedly given the FDA, the agencies, the flexibility to allow, particularly patients with rare diseases, and we're going through this right now, whether it's Duchenne Filippio syndrome, we've held some good hearings in the aging committee on these things,"
CRS Report
Drug Pricing and Intellectual Property: The Legislative Landscape for the 117th Congress
Reports · Mar 31, 2021
Intellectual property (IP) rights play an important role in the development and pricing of pharmaceuticals, such as prescription drugs and biological products (biologics). To provide incentives for...
Testimony NEILL
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"Congress has given FDA the tools of flexibility it needs to accelerate approvals for these devastating diseases, but sadly, flexibility and speed are not actually what most rare disease patients are witnessing. Transformative therapies are at FDA's doorstep, and so with great respect, families are pleading for FDA to unlock the door and move with urgency so that our children can have a chance at the life they"
CRS Report
FDA Regulation of Laboratory-Developed Tests (LDTs)
InFocus · Dec 19, 2025
Laboratory-developed tests (LDTs) are a class of in vitro diagnostic (IVD) device that is designed, manufactured, and used within a single laboratory. LDTs may be used in the context of identifying...
CRS Report
Congressional Court Watcher: Recent Appellate Decisions of Interest to Lawmakers (June 24–June 30, 2024), Part 2
Posts · Jul 1, 2024
The federal courts issue hundreds of decisions every week in cases involving diverse legal disputes. This Sidebar series selects decisions from the past week that may be of particular interest to...
Testimony ANNIE KENNEDY
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"We have more than 250 products that have been approved through the accelerated approval pathway, but fewer than 20 percent of those are for the non-oncologic, non-infectious rare diseases, which means there is a distinction between what's happening in the rare community that's in this room and the broader rare community. So it's really important when we look at those statistics that we sort of look at what the different medical product centers are comfortable with and are"
Testimony JEREMY SCHWALMAN
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"But since starting Troruluzol a year ago, he has not changed. Mary, in her late 40s, has Type 2. After six and a half years on Troruluzol, she has not changed. These inherited neurodegenerative diseases worsen inexorably. And in this business, staying the same is success. Like other rare diseases, Ataxia is difficult to study, deteriorates slowly, and manifests differently, even within families. There are 15,000 Ataxia patients in America. Some affect hundreds of people, some just a few. Now..."
CRS Report
Prescription Drug User Fee Act (PDUFA): 2017 Reauthorization as PDUFA VI
Reports · Mar 16, 2018
The Prescription Drug User Fee Act (PDUFA, now called PDUFA I) was reauthorized as PDUFA VI by the Food and Drug Administration Reauthorization Act of 2017 (FDARA, P.L. 115-52). First passed by...
CRS Report
How FDA Approves Drugs and Regulates Their Safety and Effectiveness
Reports · May 8, 2018
The Food and Drug Administration (FDA), a regulatory agency within the Department of Health and Human Services, regulates the safety and effectiveness of drugs sold in the United States. FDA divides...
Testimony KEAN, THOMAS H.
Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role ...
house Energy and Commerce Committee · Jul 15, 2026
"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 and even within FDA review divisions. This is a significant source of uncertainty that translates to an unpredictable regulatory environment and disincentives investment in rare in areas of..."
Testimony SCOTT, RICK
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"patients, there are serious national security consequences that come with the FDA's inaction and delays. Our adversaries have been accelerating their drug development and approval, attracting investment, talent, and clinical trials. FDA inaction here at home creates an economic and national competitive issue. Let me be clear. This hearing is not about weakening safety standards. Safety must always come first. But safety and speed are not mutually exclusive. A system can protect patients while..."
Testimony GILLIBRAND, KIRSTEN E.
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"to use authorized flexibilities, limited communication with drug sponsors, failure to incorporate patient experience and real world evidence reviews, and FDA shifting its regulatory position on trial design at the last minute, rejecting drug applications, and requiring new clinical trials the sponsor may be unable to perform. This is heartbreaking for patients, and it's why we can't afford delays and disruptions in treatment. Rare diseases can progress rapidly, cause irreversible harm, and in..."
Testimony GILLIBRAND, KIRSTEN E.
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"Thank you, Mr. Chairman. I want to introduce our next witness, Bradley Campbell. Mr. Campbell is the President and CEO of Amicus Therapeutics, a biotechnology company focused on discovering and developing new medicines for people living with rare diseases. During his tenure at Amicus, he led the global commercialization of Galifold, a medication used to treat Fabry disease in adults, which was approved by the U.S. Food and Drug Administration on an accelerated"
Testimony BRADLEY CAMPBELL
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"Thank you very much, Chairman Scott, Ranking Member Gillibrand, the rest of the senators on the committee. It's my privilege to be here today to speak to you about our experience in developing drugs for people living with rare diseases. I'm also honored to be here alongside my fellow panelists. I have had the privilege to work at Amicus for the last 20 years and have dedicated most of my professional career to developing new treatments for people living with rare"
Testimony BRADLEY CAMPBELL
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"We can't ask patients to wait for years before approved treatments come when the difference between life and death can be that single breath. I think the rare disease innovation ecosystem in the United States has made enormous progress over the last 20 years, but it must now again adapt in speed, agility, and flexibility to keep the pace of innovation. One of our own development experiences at Amicus, I think, sheds light on how regulatory flexibility and working together with sponsors and..."
Testimony BRADLEY CAMPBELL
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"Fast forward 15 years, Fabry disease is now a treatable disease, advancement in treatments have changed that trajectory and for the first time he's thinking about a future he thought he would never have. I look forward to working together with the members of this committee and indeed with the regulators, the broad community here focused on rare diseases to find ways to ensure that we have a flexible, adaptable, agile regulatory system that can keep pace with modern innovation. Thank you, Mr...."
Testimony NEILL
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"Thank you, Chairman Scott, Ranking Member Gillibrand, and members of the committee. On behalf of 15 million children with rare diseases in this country, I thank you truly for your concern. I'm Cara O'Neill, chief science officer at Cure San Felipo Foundation, a"
Testimony ALSOBROOKS, ANGELA D.
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"family members. I've heard from many Marylanders living with rare diseases and from their families, including dozens who have visited my office this week to share their priorities and their concerns. As I underscore with the NIH director, a few weeks ago, patients suffering from devastating diseases do not have time to wait for needless delays to critical cures. For Marylanders with rare diseases, delayed access is not just an"
Testimony ALSOBROOKS, ANGELA D.
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"The agency first declined even to review the application, then reversed course just one week later after a revised regulatory approach. The abrupt change raised serious concerns about transparency, predictability, and political influence, and what should be a scientific process, an episode many have described as regulatory whiplash. What does that kind of volatility signal about how the FDA is functioning right now, and why does that matter for rare disease reviews that depend on regulatory..."
Testimony ANNIE KENNEDY
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"But what we're concerned about is, to your question, what seems to be reversals in decisions where there had been previous agreement, previous work with sponsors that was directing sponsors to move in one direction, and then regulatory decisions seem to be yielding different decisions at this point. So, as I stated in my testimony, we have seen recently 23 complete response letters issued in rare diseases that are delaying access to the patient community and having devastating consequences to..."
Testimony MCCORMICK, DAVID
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"and thank you all so much for being here today to help bring some of these critical issues to Americans and Pennsylvanians to light. Ms. Kenney, more than 30 million Americans live with rare diseases, yet fewer than 5 percent have an approved treatment. Since – as you mentioned, since early 2025, the FDA has issued at least 23 complete response letters for rare disease therapies, while advisory committees' use has declined dramatically. Since – as you mentioned, since early 2025, the FDA has..."
Testimony MCCORMICK, DAVID
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"Thank you for highlighting that urgency, and I think we feel it, and you're helping us to feel it. So, thank you for that. Mr. Campo, just on the issue of accelerating the process, I was taken by the fact in preparing for this that some countries are moving much faster on rare disease therapies. The European Union's FAST EU program caps multinational clinical trial authorization at 70 days, and Australia allows many trials to begin almost immediately after an ethics approval. So, what's going..."
Testimony BRADLEY CAMPBELL
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"Thank you, Senator. I completely agree. I think, you know, the United States has been the beacon of biotechnology innovation for so many decades, and it's part of why we have more therapies approved today for rare diseases than we did, you know, decades ago, and I would acknowledge legislation like the Orphan Drug Act that led to that, but as we said in our testimony and many of the panelists here today, we've now stopped keeping pace with innovation, and I think one exact point, which I raised..."
Testimony ANNIE KENNEDY
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"We have individuals in this room who have very limited life expectancies, and if we don't address what's happening in rare disease with that same sense of urgency, they would not be here with us if we were to convene this hearing another year from now. Yes, Senator, you absolutely are. And Congress has recognized this urgency in statute and has enabled the use of regulatory flexibilities, and that is what's reflected in the accelerated approval pathway and the use of surrogate outcome measures..."
Testimony ANNIE KENNEDY
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"What we're concerned about is that in many of these complete response letters, what we're the trend that we're detecting backing away from a comfort level or use of the surrogate biomarkers. But what we've seen in rare disease is surrogates work. Surrogate biomarkers do save"
Testimony KIM, ANDY
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"Mr. Campbell, I wanted to bring you in on this because, you know, you've talked about this at length built off of what we just heard. How would expanded use of adaptive trial designs or surrogate endpoints by the FDA lend itself to achieving better outcomes when it comes to rare disease approvals? I just want to jump in here at the end. I mean, the urgency on the trials and moving up forward on the approvals, but Mr. Campbell, I also want to raise another issue, which is just how long it often..."
Testimony BRADLEY CAMPBELL
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"whether you think of that as a security threat or whether you think of that as a threat to getting drugs to patients, in either case, I think Congress can take a leadership role in helping the FDA to speed through that process. Yeah, I think the first step is just use what we have available to us, right? So if you look at oncology, I think in 2024 there are 8,000 different therapeutics in clinical trial design. That dwarfs the number in rare diseases today. And I think a large part of that is..."
Testimony JEREMY SCHWALMAN
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"The physicians there, the residents are there, the nurse, the nurse practitioner, the physical therapist, the patient and the family, it's a conversation, a discussion. This is not what we're hearing from across the board, and certainly from the other rare diseases. And we're here, the few of us, turns out there are 30 million people behind us, as you"
Testimony GILLIBRAND, KIRSTEN E.
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"For each of the witnesses, you can start, Ms. Kennedy. How important is it to the rare disease community for FDA to restore the use of advisory committees? And Dr. Shraman and Dr. O'Neill, congressional actions have advanced how patient experience data is included in the development and evaluation of rare disease"
Testimony ANNIE KENNEDY
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"And what we're concerned about right now is that the agency isn't engaging with the patient community. It's everything. We, yesterday, had one of our communities showcase in front of close to 800 members of the rare disease community how an advisory committee meeting helped inform a key regulatory decision for the Barr syndrome community by showing how an open public hearing enabled members of that community illuminate the nuance of a very complex regulatory decision in a very complicated..."
Testimony ANNIE KENNEDY
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"That's not the point. But the point is, for external experts, including clinicians, including those with statistical expertise and manufacturing expertise that are not always internal to the agency, to be brought to bear on regulatory decisions, because we realize that with 10,000 rare diseases, it is not possible for FDA to always have all of that expertise internal. Those committee hearings must be at the avail of the agency, but also those open public hearings must be available so that..."
Testimony GILLIBRAND, KIRSTEN E.
Hearings to examine FDA bureaucracy, focusing on regulator to roadbloc...
senate Aging (Special) Committee · Feb 26, 2026
"How important is it to the rare disease community for FDA to restore the use of advisory committees? And Dr. Shraman and Dr. O'Neill, congressional actions have advanced how patient experience data is included in the development and evaluation of rare disease therapies. What is the importance of the patient voice in this regulatory process, and what more is needed to ensure FDA includes this information in its decision"
Testimony KEAN, THOMAS H.
FY27 BIS Budget: the AI Arms Race and the ICTS Office
house Foreign Affairs Committee · Jul 14, 2026
"I also appreciate your leadership in strengthening America's pharmaceutical supply chain and advancing domestic manufacturing. My district is home to numerous rare disease drug developers who are typically small biotechs with only a few FDA approved products. Their patients are often children who have few to no treatment options. the President recognized that the supply chain of rare disease therapies would receive a zero percent tariff"
Testimony GRIFFITH, H. MORGAN
Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role ...
house Energy and Commerce Committee · Jul 15, 2026
"how we can get more sites like community health centers involved in these early stage clinical today. All right, if you can do it in 30 seconds, Ms. Winkler, how can using real world evidence or natural history studies and trial designs help new treatments for rare diseases? I now yield back and recognize Ms. DeGette, ranking member of the subcommittee, for"
Testimony DR. THOMAS HWANG
Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role ...
house Energy and Commerce Committee · Jul 15, 2026
"of these institutions that could be centers of excellence could speak to kind of your concerns of work does FDA fund externally? 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 should be withdrawn? I'll start with you, Dr. Hwang. I'm still reviewing the proposed rule, but as written, I have serious concerns before..."
Testimony DINGELL, DEBBIE
Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role ...
house Energy and Commerce Committee · Jul 15, 2026
"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. As a result of the Orphan Drug Act, there have been important advances in research on and treatment for rare diseases. The scientists and researchers at the National Institute of Health, who would have previously received funding to..."
Testimony DINGELL, DEBBIE
Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role ...
house Energy and Commerce Committee · Jul 15, 2026
"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 drug makers 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..."
Testimony DINGELL, DEBBIE
Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role ...
house Energy and Commerce Committee · Jul 15, 2026
"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? 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? Thank"
Testimony MS. SUSAN WINCKLER
Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role ...
house Energy and Commerce Committee · Jul 15, 2026
"comes in late, or there was a lack of clarity, or sometimes sponsors don't always listen perhaps as Both of you. 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..."
Testimony DR. CYNTHIA VERST
Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role ...
house Energy and Commerce Committee · Jul 15, 2026
"volume of clinical trials and, of course, the resulting capacity in order to accommodate them. opportunities we have. Thank you. Terrific question. I think several ways the agency can help on the rare disease front. Notably, over 65 percent 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..."
Testimony KEAN, THOMAS H.
Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role ...
house Energy and Commerce Committee · Jul 15, 2026
"that more certainty. Thank you. 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? There are a couple And second, how can Congress strengthen FDA's ability in this through the user"
Testimony DR. CYNTHIA VERST
Maintaining America’s Leadership in Biomedical Innovation: FDA’s Role ...
house Energy and Commerce Committee · Jul 15, 2026
"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"
Testimony UNKNOWN
H.R. 9773, Permanent Trump Secure Border Act and Ratification of Subco...
house Judiciary Committee · Jul 21, 2026
"ICE released his parents one day before his death. In another case, a U.S. citizen with a rare disease died after ICE detained his father and primary caretaker. ICE also detained a DACA recipient while he was on his way to the NICU to bring breast milk to his infant daughter, who was born nearly six weeks"
Testimony SPEAKER_05
Business meeting to consider the nominations of Brett Matsumoto, of Ma...
senate Health, Education, Labor, and Pensions Committee · Jul 15, 2026
"Senator, thank you for the question and thank you for meeting with me earlier. Whether it's CJD or CWD, certainly if I am confirmed as a CDC director, I would work with your team to understand what are the capacities of the CDC to look into these types of very rare diseases. I know their resource constraints are, you know, something that is a reality, whether it's with the CDC or any other institution. So I certainly want to make sure that I was not aware of what you just described, but I want..."
Testimony SPEAKER_05
Hearings to examine the nominations of Sean Kaufman, of Georgia, to be...
senate Health, Education, Labor, and Pensions Committee · Jul 15, 2026
"certainly if I am confirmed as a CDC director, I would work with your team to understand what are the capacities of the CDC to look into these types of very rare diseases. I know the resource constraints are something that is a"
Testimony DELAURO, ROSA L.
Fiscal Year 2027 Labor, Health and Human Services, Education, and Rela...
house Appropriations Committee · Jun 5, 2026
"of daily necessities, while hollowing out health, education, nutrition, and housing programs that help families make ends meet. Despite these objections, and I would just say very honestly, there are some bright spots in this bill that deserve recognition. First, I am glad to see the overall funding level for the National Institutes of Health is increased under this bill, especially targeted increases for research on cancer, Alzheimer's, ALS, diabetes, women's health, and rare diseases. Second,..."
Testimony MATSUI, DORIS O.
Full Committee Markup of 16 Bills
house Energy and Commerce Committee · May 21, 2026
"in providing transplants for pediatric patients and adults with high-risk leukemia. Thanks to the pioneering work of patients involved in the National Cord Blood Inventory program, cord blood stem cells are also being used and researched to unlock treatments for rare diseases, Alzheimer's, stroke, and more. The AIMS makes important updates to ensure public cord blood banks can manage the inventory of banked cord blood"
Testimony BILIRAKIS, GUS M.
Full Committee Markup of 16 Bills
house Energy and Commerce Committee · May 21, 2026
"Since then, the program has helped strengthen collaboration between researchers, regulators, manufacturers, and patient advocates working to better understand and treat ALS. This amendment continues that work by extending these programs through 2031, while making targeted improvements developed in coordination with FDA, NIH, HHS, and stakeholder groups to help address challenges unique to rare disease drug development, including clinical trial enrollment, safety reporting, and the use of small..."
Testimony SPEAKER_02
Hearings to examine the President's proposed budget request for fiscal...
senate Health, Education, Labor, and Pensions Committee · Apr 22, 2026
"Through President Trump's Great American Recovery Initiative, HHS is matching compassion with action to help Americans break the cycle of addiction. At HHS, we are prioritizing patients with ultra-rare diseases and their families and driving faster access to life-saving treatments. We're restoring gold standard science and integrity across the"
Testimony MR. SEAN MURPHY
“Defending Main Street: Combating CCP Threats to America’s Small Busin...
house Small Business Committee · Mar 25, 2026
"honoring the promise that no veteran dies alone. My advocacy for my son and veterans centers on palliative care and access to cannabinoids, rescheduling marijuana from Schedule 1 to Schedule 3 to unlock research and healing, especially for rare diseases. I'm also an advocate against child trafficking, and I implore you to stand against it at home and"
Testimony JOHNSON, RON
Member Day to examine legislative priorities.
senate Health, Education, Labor, and Pensions Committee · Mar 19, 2026
"Congress has repeatedly given the FDA, the agencies, the flexibility to allow, particularly patients with rare diseases, and we're going through this right now, whether it's Duchenne Filippio syndrome, we've held some good hearings in the aging committee on these things,"
CRS Report
Medicare Drug Price Negotiation Under the Inflation Reduction Act: Industry Responses and Potential Effects
Reports · Dec 8, 2023
The 2022 budget reconciliation legislation commonly known as the Inflation Reduction Act (IRA; P.L. 117-169) established the Medicare Drug Price Negotiation Program (Program). The Program authorizes...
CRS Report
The Role of Patents and Regulatory Exclusivities in Drug Pricing
Reports · Jan 30, 2024
Intellectual property (IP) rights play an important role in the development and pricing of prescription drugs and biologics. To encourage innovation, IP law grants inventors exclusive rights in a...
CRS Report
Specialty Drugs: Background and Policy Concerns
Reports · Aug 3, 2015
Specialty drugs are one of the fastest-growing areas of health care spending. There is no one set definition of specialty drugs, although insurers and other health care payers often characterize them...
CRS Report
Implementation of the Medicare Drug Price Negotiation Program: Centers for Medicare and Medicaid Guidance and Legal Considerations
Reports · May 16, 2023
This report discusses P.L. 117-169, a budget reconciliation measure often referred to as the Inflation Reduction Act (IRA), and its creation of the Medicare Drug Price Negotiation Program (the...
CRS Report
The Food and Drug Administration (FDA) Budget: Fact Sheet
Report · Jun 10, 2026
The Food and Drug Administration (FDA) regulates the safety of foods (including dietary supplements), cosmetics, and radiation-emitting products; the safety and effectiveness of drugs, biologics...
CRS Report
The National Institutes of Health (NIH): Background and Congressional Issues
Reports · Jan 13, 2025
The National Institutes of Health (NIH), under the Department of Health and Human Services (HHS), is the leading federal agency for biomedical and health research. In FY2024, NIH used its over $47...