Insights

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.