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On February 26, 2026, the Senate Special Committee on Aging held a hearing to examine the Food and Drug Administration’s (FDA) review process for rare diseases. The witnesses all shared their frustration that the FDA has tools to allow for flexibility in drug approvals, but that these flexibilities are inconsistently applied across the various subagencies. In their view, this has led to confusion and a lack of transparency in the drug approval process. The committee discussed the current FDA landscape, manufacturing concerns, the impact of delays on patients, and possible policy actions. Possible policy actions discussed included improved clinical trial implementation, use of surrogate endpoints, funding for the Rare Disease Innovation Hub, and greater use of advisory committee meetings.

OPENING STATEMENT

• Chairman Rick Scott (R-FL)

WITNESS TESTIMONY

• Annie Kennedy, Chief Mission Officer, EveryLife Foundation for Rare Diseases – Testimony
• Dr. Jeremy Schmahmann, MD, Director, Massachusetts General Hospital Ataxia Center – Testimony
• Bradley Campbell, President and CEO, Amicus Therapeutics – Testimony
• Dr. Cara O’Neill, MD, FAAP, Chief Science Officer, Co-Founder, Cure Sanfilippo Foundation – Testimony

MEMBER DISCUSSION

Current FDA Landscape

Senators on both sides of the aisle expressed interest in how the FDA’s current FDA landscape impacts the review process for rare disease treatments. Sen. Ron Johnson (R-WI) was interested in the witness’s experiences interacting with the FDA. Dr. Schmahmann shared that he felt a lack of compassion from FDA personnel during meetings and that they did not engage in open dialogue about the science and patient experiences. Dr. O’Neill has had different experiences, sharing that her interactions with regulators have been kind, with them listening to patient experiences. However, Dr. O’Neill has not seen these experiences translate into regulatory action.

Sen. Angela Alsobrooks (D-MD) asked how changes in FDA personnel and guidance have affected trends in the rare disease drug approval process. Ms. Kennedy answered that she is increasingly concerned about application reversals and reports that the FDA has changed requests to sponsors at the last minute. Ms. Kennedy is most concerned about the lack of advisory committee meetings taking place now as compared to a few years ago, as these meetings allow for dialogue between FDA personnel, drug sponsors, and patients.

Sen. Andy Kim (D-NJ) was curious about the manufacturing process for rare-disease treatments. Mr. Campbell explained that many small and mid-sized companies rely on foreign manufacturing due to the time and costs required to build domestic manufacturing facilities. According to Mr. Campbell, it takes an average of 3-5 years to build a facility and an additional 1-2 years to pass FDA inspections before manufacturing can begin. So, while Mr. Campbell is supportive of onshoring drug manufacturing, he noted that the current regulatory environment makes it very difficult.

Impact on Patients

Chairman Rick Scott (R-FL) requested that witnesses share the effects of delayed treatment on patients as well as the ethical implications of stopping compassionate use for drugs. Dr. Schmahmann stated very clearly that delayed treatments lead to increased symptoms and decreased lifespan for patients while also reducing the quality of life for them and their families. Dr. Schmahmann was a strong supporter of compassionate use, sharing that, in his opinion, preventing compassionate use or suggesting trials that would force patients off helpful drugs is very unethical. Dr. O’Neill shared her frustration that the FDA is taking steps to reduce the use of animal models in drug testing due to ethical concerns, while not considering the ethical considerations of not allowing working treatments to be given to children with rare diseases.

Possible Policy Actions

Multiple Senators, including Sen. Dave McCormick (R-PA), Chairman Scott, and Ranking Member Gillibrand, were greatly interested in possible policy actions to better support the drug approval process. Mr. Campbell expressed support for the use of centralized Independent Review Boards (IRBs) and speeding up clinical trial implementation. Mr. Campbell also suggested providing more funding and attention to the Rare Disease Innovation Hub, which could allow for rare disease experts to be trained and deployed across the FDA. Ms. Kennedy felt that allowing greater use of surrogate endpoints and biomarkers would be extremely helpful in centering on patient experience and enabling the approval of treatments for medically complex diseases.

Ranking Member Gillibrand suggested greater use of advisory committee meetings as a path forward. Ms. Kennedy shared that advisory committee meetings are extremely impactful for allowing nuance in regulatory decisions, as they allow for outside experts to be included in decision-making. Dr. O’Neill continued that they allow for dialogue with patients and sponsors.

On February 25, 2026, the Senate Health, Education, Labor, and Pensions (HELP) Committee held a hearing to consider the nomination of Dr. Casey Means to be Surgeon General of the Public Health Service. The committee covered the topics of vaccines, nutrition, mental health, substance use, and women’s health. The next step in the confirmation process is for the Senate HELP Committee to hold an Executive Session to vote on the nomination. Assuming the Committee advances her nomination, it would next go to the Senate floor for a vote on confirmation.

OPENING STATEMENTS

• Chairman Bill Cassidy (R-LA)

WITNESS TESTIMONY

• Dr. Casey Means, Nominee to be US Surgeon General – Testimony

MEMBER DISCUSSION

Vaccines

The most prominent topic of conversation was Dr. Means’ views on vaccines. There was bipartisan questioning of whether Dr. Means views vaccines, such as those for the flu, measles, mumps and rubella (MMR), respiratory syncytial virus (RSV) and Hepatitis B (Hep B), as safe and effective for children. Dr. Means stated multiple times that she believes vaccines are key for public health. She also expressed support for the practice of shared clinical decision making and gaining informed consent from patients before administering vaccines. When pressed by Chairman Bill Cassidy (R-LA) about what additional steps pediatricians should take to obtain informed consent, Dr. Means did not have a concrete answer.

Nutrition

One of Dr. Means’ stated priorities is to improve nutrition and reduce consumption of ultra-processed foods. Many senators shared their support, including Ranking Member Bernie Sanders (I-VT), and Sens. Tommy Tuberville (R-AL), Roger Marshall (R-KS), and Ed Markey (D-MA). Sen. Marshall was curious about how Dr. Means expected to accomplish this goal, as many Americans do not have access to nutritious food options. Dr. Means acknowledged that if whole foods are not a sustainable choice, then many Americans will not change their behaviors. To help with this, Dr. Means highlighted the recent change in dietary guidelines encouraging procurement of food sources and her desire to encourage more federal research about food additives and pesticides.

Sen. Jon Husted (R-OH) shared concerns that changing views on pesticides could be detrimental for American farmers. Dr. Means responded that she greatly respects farmers but wants to change agricultural practices so that they are not as dependent on toxic chemicals.

Mental Health and Substance Use

Sens. Husted and Marshall were interested in understanding Dr. Means’ thoughts on the link that smartphones and social media have to adolescent mental health. Dr. Means shared that she is also concerned about the effects of technology on mental health and is committed to working with various public and private sectors to protect children from the harms of smartphones and social media.

Sen. Susan Collins (R-ME) raised concerns about Dr. Means past statements promoting illicit drug use, given the rise in substance use disorders. Dr. Means reassured the senator that she would not make similar statements while in office, as the science is still emerging about the use of various drugs to treat health conditions.

Women’s Health

Chairman Cassidy and Sen. Patty Murray (D-WA) asked about the use of oral contraception, such as Mifepristone pills, and both hormonal and non-hormonal birth control options. Sen. Murray questioned whether Dr. Means supports their approval by the Food and Drug Administration (FDA) due to past comments about their “horrifying health risks for women.” Dr. Means expressed the view that these medications should only be prescribed after an in-depth conversation with a physician and after gaining informed consent about possible risks and side effects. Chairman Cassidy asked if these medications should only be prescribed after an in-person doctor’s visit to which Dr. Means stated that such a requirement is outside of the purview of the Surgeon General’s office.

Sens. Jim Banks (R-IN), Tim Kaine (D-VA), and John Hickenlooper (D-CO) were curious about Dr. Means’ approach to In-Vitro Fertilization (IVF). Dr. Means shared that she is supportive of access to IVF treatments but wants to emphasize the positive effects of lifestyle changes in supporting increased fertility, especially for Polycystic Ovary Syndrome (PCOS).

Vision for the Role

Dr. Means shared that her overall vision as US Surgeon General would be to improve health outcomes through better nutrition and the consumption of whole food and encouraging the healthcare system to focus on the root causes of disease, rather than addressing symptoms and providing sick care. Dr. Means also plans to champion research on environmental exposures that cause disease. Dr. Means believes that her interdisciplinary background allows her to approach the position in a unique way. She hopes to be a messenger and champion solutions to reduce healthcare spending in the United States.

On March 5, 2026, the Senate Health, Education, Labor, and Pensions (HELP) Committee held a hearing on how to transform health care with data and improve patient outcomes. The Committee discussion focused on the topics of prior authorization, cybersecurity, interoperability of electronic health systems, and use of artificial intelligence.

OPENING STATEMENTS

• Chairman Bill Cassidy (R-LA)

WITNESSES

• Dr. Thomas Keane, MD, MBA, Assistant Secretary for Technology Policy and National Coordinator for Health Information Technology, US Department of Health and Human Services – Testimony

MEMBER DISCUSSION

Prior Authorization

Sen. Roger Marshall (R-KS) requested an update on how the office is improving the prior authorization process. Dr. Keane highlighted that they have finalized the HTI 4 rule, which sets standards that allow electronic prior authorization requests to be processed in real time by ensuring that electronic health records (EHRs) can be shared between insurance companies and providers. He also shared that ASTP is working with the Centers for Medicare and Medicaid Services (CMS) to meet the goal of 80% of prior authorizations processed in real-time by January 1, 2027. Sen. Marshall asked about current barriers to prior authorization reforms. Dr. Keane shared that the largest barrier is information sharing between insurance companies and providers, but insurance companies are working with him to identify the choke points and potential solutions. EHR companies have also committed to reducing barriers. Sen. Marshall also advocated for the Senate to advance the H.R. 3514, the Improving Seniors Timely Access to Care Act.

Cybersecurity

Senators also expressed concerns about cybersecurity in the health care space. For example, Sen. Josh Hawley (R-MO) expressed concerns about cybersecurity protections for rural hospitals. Dr. Keane acknowledged that cyber-attacks can be devastating for rural hospitals, and he is committed to ensuring patient data is protected and hospital systems are secure. Dr. Keane highlighted that the HTI 5 rule updates certification criteria to encourage systems to adopt the most modern standards for cybersecurity. Chairman Bill Cassidy (R-LA) questioned whether the new certifications and removal of ones previously in place would lead to lower security and fewer privacy protections. Dr. Keane said that the certifications removed were outdated, redundant, or not widely adopted by industry, and therefore the remove of the old standards had no impact on the market.

Interoperability

Senators on both sides of the aisle expressed interest in health care interoperability. For example, Sen. Tim Kaine (D-VA) focused his questioning on how to expand EHR use to other sectors of the health care system, like behavioral health and long-term care providers. Dr. Keane shared that they have been working with the Substance Abuse and Mental Health Services Administration (SAMHSA) on pilot programs that improve interoperability between providers and social service departments, as well as more generally looking for new, modernized solutions to improve interoperability for all providers, including long-term care. Sen. Marshall asked about the current barriers to interoperability, to which Dr. Keane responded that there need to be clear standards, tools to evaluate them, and ways to ensure that organizations are not willfully engaged in information blocking. Dr. Keane expanded that he works with the Health and Human Services Office of the Inspector General when allegations of information blocking are not addressed after warnings of non-conformity. Sen. Lisa Murkowski (R-AK) appreciated the conversation about interoperability but highlighted that many rural and tribal health systems lack the infrastructure and capacity to benefit from many technological advances in healthcare, including EHRs. Sen. Murkowski questioned if agencies such as the Department of Veterans Affairs (VA) and the Indian Health Service (IHS) have been engaged with the development of interoperability standards to ensure that they are not inadvertently creating barriers. Dr. Keane assured the Committee that he has been working with IHS, which was the first organization to join the Trusted Exchange Framework and Common Agreement (TEFCA), through monthly meetings and aiding in technology vendor and contract conversations.

Artificial Intelligence (AI)

Another bipartisan area of interest was AI in health care. For example, Sen. Angela Alsobrooks (D-MD) asked what the role ASTP should play in establishing guardrails on AI use. Dr. Keane responded that ASTP is committed to ensuring patient safety and privacy are protected, and through the recent Health and Human Services RFI on AI use in health care, ASTP can evaluate where gaps in the federal framework are. Chairman Cassidy asked how to best protect patients who upload their health information onto an AI platform, and how the Health Insurance Portability and Accountability Act (HIPAA) could be applied. Dr. Keane clarified that generally, if an individual chooses to download their health information and upload it to an AI tool, that is the individual’s choice and falls outside of the purview of HIPAA. Chairman Cassidy understood but suggested additional consumer safeguards, like a pop-up that would warn an individual that their health data is not protected once uploaded. Dr. Keane responded that any action that would improve security and patient privacy is worth considering.

On February 11, 2026, the Senate Special Committee on Aging held a hearing to examine how regulatory challenges for physicians can create undue burdens. There was bipartisan support about the need to address the issues of physician burnout, administrative burdens, and prior authorization reforms.

OPENING STATEMENTS

• Chairman Rick Scott (R-FL)

WITNESSES

• Alma Littles, MD, Dean and Chief Academic Officer, Florida State University College of Medicine – Testimony

• Lee Gross, MD, Founder, Epiphany Health Direct Primary Care – Testimony

• Jeffrey Smith, CPA, MBA, FACMPE, CGMA, Incoming Board Chair of Medical Group Management Association (MGMA) and Chief Executive Officer Piedmont HealthCare, PA – Testimony

• Corey Fiest, JD, MBA, Co-founder and Chief Executive Officer, Lorna Breen Heroes’ Foundation – Testimony

MEMBER DISCUSSION

Sen. Ashley Moody (R-FL) expressed concerns that the exodus of providers from traditional practices to direct primary care will lead to care not being accessible to all patients. Sen. Moody asked what Dr. Gross would recommend to ensure that patients served by traditional practices continue to have access to quality and affordable care. Dr. Gross responded that, in his view, a physician who leaves practice completely would be seeing zero patients, but a physician who moves to a direct primary care model is still seeing patients.

Ranking Member Gillibrand (D-NY) requested that Mr. Fiest explain how the wellbeing guide developed by his organization reduces physician burnout. Mr. Fiest shared that clinicians who practice in systems that have implemented the guide have experienced a decrease in time spent outside of direct patient care and increased levels of wellbeing, both of which have decreased burnout symptoms. Ranking Member Gillibrand also asked what the expected impact of the Wasteful and Inappropriate Service Reduction (WISeR) Model will be on administrative burden and burnout. Mr. Smith expressed concerns that the model will increase the need for prior authorizations and lead to more patients seeing delayed or denied care. Mr. Smith continued that these effects would lead to sicker patients and contribute to physician burnout. Ranking Member Gillibrand was also interested in how a mismatch in medical training and actual practice can drive burnout. Dr. Gross argued that current medical education does not teach practice management skills, which are crucial for physicians who want to practice outside of a large hospital system.

Sen. Raphael Warnock (D-GA) questioned what the effect of additional workforce shortages will have on health professionals. Mr. Fiest shared that reductions in staff will impact access, quality, and cost of health care as physicians need to take on greater amounts of administrative work. Sen. Warnock wondered if increasing the number of residency spots, through bills such as S.2439, could be part of a solution. Mr. Smith said that yes, any increase in slots would help, but they also need to incentivize residents to practice general primary care. Sen. Warnock questioned if the costs associated with medical school are dissuading students from entering the profession. Dr. Littles shared that costs are a large factor for students as they must support themselves through schooling. Dr. Littles also stated that students will make decisions on what type of medicine to practice based on salaries for different specialties.

Sen. Angela Alsobrooks (D-MD) asked what impactful, preventive system reforms could address the root causes of burn out. Mr. Fiest noted that reducing administrative burden, improving nursing safety, and preventing acts of violence would make the workload more manageable for providers. Sen. Alsobrooks then asked how the current prior authorization system contributes to physicians’ burdens and impacts the delivery of care. Mr. Smith explained that prior authorizations are delaying care, with more patients seeking care in urgent care or emergency rooms. Mr. Smith also highlighted that many administrative workers are experiencing high levels of burnout due to the workload and addressing administrative burdens would reduce turnover.

Chairman Rick Scott (R-FL) questioned how increased documentation requirements may reduce the desire to work in underserved areas. Dr. Littles shared that rural communities often do not have access to many electronic health record systems due to the high costs, and for systems that do have them, the complexity and lack of interoperability have a negative impact on the practice experience. Chairman Scott wanted to understand the general structure of Dr. Gross’s day. Dr. Gross shared that he has a greater capacity for same-day patients, which reduces his need to refer patients to the emergency room or specialists. Dr. Gross also has more time to manage his practice.

On February 11, 2026, the House Energy and Commerce Health Subcommittee held a hearing to consider how to reduce health care costs by examining the prescription drug supply chain. This hearing was the Subcommittee’s second on health care affordability, and reports indicate there will be additional hearings focusing on other parts of the health care sector. Overall, Republicans and Democrats both agreed that prescription drug costs are too high and that there is too much consolidation in the prescription drug industry, especially among pharmacy benefit managers (PBMs). However, Republicans and Democrats on the Committee also focused on familiar partisan arguments regarding the impact of the Inflation Reduction Act and recent actions by the Trump administration, including TrumpRx, on prescription drug costs. Unsurprisingly, witnesses from different parts of the pharmaceutical industry sought to highlight actions they have taken to reduce costs and to place blame on other stakeholders, including hospitals and insurance companies, as well as on programs such as the 340B program.

OPENING STATEMENTS

• Health Subcommittee Chair Morgan Griffith (R-VA-9)

• Full Committee Ranking Member Frank Pallone (D-NJ-6)

WITNESS TESTIMONY

• Lori M. Reilly, Esq., Chief Operating Officer, PhRMA – Testimony

• John F. Crowley, President and CEO, Biotechnology Innovation Organization – Testimony

• John Murphy, President and CEO, Association for Accessible Medicines – Testimony

• David Marin, President and CEO, Pharmaceutical Care Management Association – Testimony

• Angie Boliver, President and CEO, Healthcare Supply Chain Association – Testimony

• Chester “Chip” Davis, Jr., President and CEO, Healthcare Distribution Alliance – Testimony

• James Gelfand, President and CEO, The ERISA Industry Committee – Testimony

• Douglas Hoey, CEO, National Community Pharmacists Association – Testimony

• Rachel E. Sachs, Professor of Law, Washington University in St. Louis – Testimony

MEMBER DISCUSSION

PBM Consolidation and Vertical Integration

Multiple Health Subcommittee members on both sides of the aisle expressed concerns about reports that the 3 largest PBMs control 80% of the market and about PBMs’ frequent vertical integration. Members expressed skepticism about Mr. David Marin’s arguments that there is competition within the industry and that this competition leads to lower prices for patients. Other witnesses who did not represent PBMs were also highly critical of certain PBM practices. A number of Subcommittee members praised the inclusion of PBM reforms in the recently passed appropriations legislation, but others said more needed to be done.

Medicare Negotiation Vs. Most-Favored Nation Deals and TrumpRx

The biggest partisan divide centered on whether Medicare negotiation, as passed under the Inflation Reduction Act, or the most-favored-nation deals negotiated by the Trump administration and presented on TrumpRx, represented the better way to address concerns about drug costs. Republicans, including Health Subcommittee Chair Morgan Griffith (R-VA-9) and Rep. Michael Rulli (R-OH-6), praised the Trump administration for its efforts to secure lower drug prices for the American people through TrumpRx. Republicans, including full Committee Chair Brett Guthrie (R-KY-2), criticized the Inflation Reduction Act for disrupting the Medicare Part D marketplace.

On the other hand, Democratic members, including full Committee Ranking Member Frank Pallone (D-NJ-6) and Health Subcommittee Ranking Member Diana DeGette (D-CO-1), argued that the deals from the Trump administration lacked transparency. As a result, they argued, there is no way to verify how effective they are at lowering drug costs. These members asked Subcommittee Chair Guthrie if he would be willing to work with them to determine the details of these deals. He said he would as long as that didn’t blow up the deals in question. Democrats such as Rep. Marc Veasey (D-TX-33) criticized TrumpRx for listing drugs that could actually be found for cheaper using manufacturer or other coupons. Democratic members such as Reps. Pallone and Jennifer McClellan (D-VA-4) criticized Republicans for including carve-outs for orphan drugs from the Medicare negotiation program in the reconciliation bill and called for expanding Medicare price negotiation to the commercial market.

Federal Government’s Role in Drug Development and Approval

Members on both sides of the aisle expressed interest in the federal government’s role in drug development and approval. Rep. Dan Crenshaw (R-TX-2) asked Ms. Reilly about efforts to modernize the regulatory framework when it comes to the approval of new therapies. She responded that a transparent regulatory environment and strong IP protections are necessary to support the development of innovative therapies, including gene and cell therapies. On the other side of the aisle, Democratic members questioned witnesses about the impact of reductions in force (RIFs) at the Food and Drug Administration (FDA) and concerns about reports of politicalization within the agency. All of the witnesses who were asked about the impact of RIFs and politicization expressed concerns about the negative impact on drug development and approval. Democratic members such as Reps. Pallone and Kim Schrier (D-WA-8) expressed specific concerns about the FDA not reviewing Moderna’s flu vaccine due to vaccine skepticism. Other Democratic members criticized Republicans for not speaking out about cuts to basic research at agencies such as the National Institutes of Health (NIH). Rep. Guthrie countered that the NIH received a half-billion-dollar increase in the appropriations legislation.

Other Topics

• Rep. Griffith asked Mr. Crowley if they should start looking at insurance companies as fiduciaries and hold them liable for harm they cause to patients. Mr. Crowley agreed that this is a good idea to provide more protection for patients.

• Reps. Gus Bilirakis (R-FL-9) and Kevin Mullin (D-CA-15) asked Mr. Crowley about rare disease drug development. In response to a question from Rep. Bilirakis on how we can continue to encourage investment in finding cures for rare diseases without burdening patients, Mr. Crowley said we need to reduce the complexity of the clinical trial system, modernize the FDA, and ensure medications are affordable and accessible.

• Rep. Troy Balderson (R-OH-12) asked Mr. Hoey to elaborate on specific challenges rural pharmacies face when negotiating with PBMs, specifically with take-it-or-leave-it contracts for reimbursements. Mr. Hoey explained that PBMs hold all the leverage, forcing pharmacies to accept contracts that pay them less than it costs to acquire the drug; as a result, 5,000 pharmacies have closed in the last 4 years.

• Rep. Cliff Bentz (R-OR-2) asked Ms. Sachs how AI might help analyze the pharmaceutical space. She noted the FDA’s approval of AI-enabled medical devices and the use of AI to streamline clinical trial enrollment. She also suggested using AI to help the subcommittee look through and summarize the vast amounts of information in the pharmaceutical space. She also warned of an AI arms race between providers and insurers that could deny care through prior authorization.

• Rep. Rick Allen (R-GA-12) asked Mr. Gelfand if his proposed bill, H.R.5509, the Safe Step Act, would help hold insurers accountable for increasing the use of step therapy, leading to nonadherence and access issues. Mr. Gelfand explained that reforming step therapy makes sense and that it should be electronic, timely, and aligned with medical management.