The Advocate

March 2025: Volume 5, Issue 3

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In This Issue:

ASGCT Discusses Cell Therapies in HHS Roundtable
NIH Funding Update: Proposed 15% Cap on Facilities & Administration  
FY25 Continuing Resolution Funds Government Through September 
House, Senate Working Toward Budget Reconciliation 
Senate Acts on Nominees to Lead Health Agencies 
Executive Orders and Administrative Actions Impact CGT
CMMI Terminates Demonstration Models; CGT Access Model Will Proceed  
Community Quick Take Tackles Immune Reponses to AAV 
Read Our Regulatory Guidance Comments to FDA
Share Your Insights on Gene Therapy Regulation  
Empowering Patients Summit Available On Demand 
Events
Policy News

ASGCT Advocacy

ASGCT Discusses Cell Therapies in HHS Roundtable

Society leadership recently participated in a roundtable with Secretary for HHS, Robert F. Kennedy Jr. and FDA’s Acting Deputy Commissioner Sarah Brenner. During this meeting, ASGCT's CEO and members of the Board of Directors discussed various aspects of cell and gene therapies, while reinforcing the organization's ongoing commitment to advocating for patient access to safe, efficacious treatments. ASGCT continues its dedicated advocacy for appropriate regulatory pathways and timely access to care across the entire regenerative medicine field. 

NIH Funding Update: Proposed 15% Cap on Facilities & Administration  

ASGCT supports robust funding for the National Institutes of Health (NIH), which supports both direct grant funding and facilities & administration (F&A) reimbursements. NIH’s proposed 15% cap on F&A has sparked concern across the scientific community. This month, ASGCT led an organizational sign-on letter for professional associations, academic institutions, and patient advocacy groups, bringing the collective voice of the biomedical research community to Congress. The letter underscores the critical nature of NIH funding not just for scientific advancement, but as an investment in the American economy.  

As of March 19 the F&A cuts have been halted by a preliminary injunction in federal court, which will remain in place while several lawsuits proceed. The lawsuits are based in part on existing Congressional appropriations language that prevents NIH from unilaterally altering F&A reimbursements; that same language was included in the Continuing Resolution passed by Congress on March 14 (see next section). 

ASGCT also released a public statement in February about the F&A cuts, and members of the ASGCT Board of Directors recently visited Capitol Hill to share their front-line research experiences with key congressional offices. Additionally, organizations including Research!America, the NCATS Alliance, and the American Society of Transplantation & Cellular Therapy (ASTCT) have voiced their concerns. The CGT pipeline has grown exponentially in recent years, but researchers require strong NIH support in order to continue that trajectory. 

FY25 Continuing Resolution Funds Government Through September 

President Trump signed a seven-month continuing resolution (CR) (H.R.1968) on March 15, funding the government through Sept. 30. The CR passed the House 217-213 on a party-line vote, then passed the Senate 54-46 with primarily Republican support. The bill increased defense spending by $6 billion while cutting non-defense spending by $13 billion. The NIH faces a $280 million (0.6%) reduction from FY24 levels due to reductions in 21st Century Cures funding, while the Congressionally Directed Medical Research Program saw a 57% cut.  

The CR did not include an extension of the Rare Pediatric Disease Priority Review Voucher (PRV) program, which expired in December 2024. The PRV program incentivizes the development of therapies for rare pediatric diseases by awarding a voucher to sponsors who develop products for these conditions. This voucher can be used to expedite FDA review of the recipient’s future drug application or sold to another company, potentially worth hundreds of millions of dollars. The program is particularly important for gene and cell therapy development in rare diseases, as it provides a significant financial incentive for companies to invest in these often challenging and costly areas of research. Many rare pediatric diseases lack effective treatments, and the PRV program has encouraged innovation in this space. ASGCT will continue to advocate for its reauthorization this Congress. 

House, Senate Working Toward Budget Reconciliation 

On Feb. 25, the House of Representatives narrowly passed a budget resolution for fiscal year 2026 (FY26) with a vote of 217-215. This resolution allows for $4.5 trillion in tax cuts and aims for $2 trillion in spending cuts over 10 years. Meanwhile, the Senate had previously approved its own "skinny" budget resolution on Feb. 20 with a 52-48 vote, focusing on border security, military funding, and energy policies. The two chambers now face the challenge of reconciling their bills, with key differences in approach and scope:​ 

• The House's plan aligns more closely with President Trump's agenda, proposing a comprehensive "one big, beautiful bill" approach. ​  

• The Senate's version postpones discussions on taxes and the debt ceiling, preferring a two-bill strategy.​ 

Potential cuts to Medicaid have been a topic of significant discussion throughout the reconciliation process. The House budget resolution directs the Energy and Commerce Committee to identify $880 billion in savings over the next decade. Given that 93% of the committee's non-Medicare mandatory spending is attributed to Medicaid, achieving these savings without impacting Medicaid would be challenging. The nonpartisan Congressional Budget Office (CBO) released a report indicating that House Republicans cannot achieve their federal spending cut goals without making significant reductions to Medicaid. This has raised concerns among healthcare advocates about potential reductions in coverage and services for millions of Americans. ​ 

ASGCT will continue to monitor how these and other Administration priorities affect the CGT field and our members, and update as FY26 funding moves through Congress.

Senate Acts on Nominees to Lead Health Agencies 

ASGCT has been closely monitoring the nomination process of Agency leaders relevant to CGTs:  

• Robert F. Kennedy Jr. was confirmed as Secretary of Health and Human Services (HHS) in February.   

• Michael Kratsios, nominated to lead the White House Office of Science and Technology Policy (OSTP), had a hearing before the Senate Health, Education, Labor, and Pensions (HELP) Committee on February 25. In his hearing Kratsios defended HHS layoffs, expressed support for emerging technologies, and noted the importance of federal funding for basic research.  

• Dr. Jay Bhattacharya, a health economist at Stanford nominated as Director of NIH, had a hearing in the Senate HELP Committee on March 5. The Senate hearing for Dr. Bhattacharya covered his views on vaccine science, NIH funding, and research priorities, with Bhattacharya expressing support for measles vaccination and promising to carefully review personnel and funding decisions. Dr. Bhattacharya is awaiting full Senate confirmation after the HELP Committee advanced his nomination on March 13.  

• Dr. Marty Makary, a pancreatic surgeon at Johns Hopkins nominated as FDA Commissioner, had a hearing in the Senate HELP Committee on March 6. The nomination hearing focused on vaccines, staffing changes, and the role of regulatory bodies in ensuring safety. The Senate HELP Committee advanced Dr. Makary’s nomination  on March 13. 

• Dr. Mehmet Oz was nominated as Administrator of the Centers for Medicare & Medicaid Services (CMS); the Senate Finance Committee held a hearing on March 14 but has not yet voted to advance his nomination to the full Senate. When asked about potential changes to Medicaid during the reconciliation process, Dr. Oz did not commit to preventing cuts.  

Executive Orders and Administrative Actions Impact CGT

The Trump Administration has issued a series of executive orders (EOs) and other agency policies with potential relevance for CGTs. The following is a summary of key developments in the past month:  

• NIH Grant Reviews: NIH stopped reviewing new grant applications for a period of time due to Administration restrictions notices in the Federal Register, which are required before study section meetings can be held. As of March 19, some study sections have resumed meeting, but it is unclear if other delays are ongoing.   

• Reduction in Force (RIF): The Office of Management and Budget (OMB) directed federal agencies to submit plans by March 13 for significant workforce reductions. While those plans have not been public as of March 19, STAT reported that “[NIH] is expected to cut between 3,400 and 5,000 positions from its workforce of 20,000.” 

• Make America Healthy Again (MAHA) Commission: Immediately following the Senate confirmation of HHS Secretary Robert F. Kennedy Jr., the Make America Healthy Again Commission was announced. The Commission focuses on exploring the reasons behind Americans' health issues, ensuring access to healthy and affordable food, and expanding treatment options and health coverage for lifestyle changes and disease prevention. A fact sheet on the Commission referenced recent advances in gene therapy for Sickle Cell Disease as an example of progress.  

• Public Comments: HHS published a policy statement that the department can forego public comment for “matters relating to agency management or personnel or to public property, loans, grants, benefits or contracts.” The policy statement also points to the “good cause” exception in the Administrative Procedure Act as a pathway to limit public comment opportunities.  

• Drug and Healthcare Pricing: On Feb. 25, President Trump signed an executive order directing the Departments of the Treasury, Labor, and HHS to create a framework within 90 days to enforce existing healthcare price transparency regulations established during his first term. This order mandates that hospitals and insurers disclose actual prices, not estimates, and calls for standardized, comparable pricing information across providers, including prescription drug prices. 

CMMI Terminates Demonstration Models; CGT Access Model Will Proceed  

The Centers for Medicare & Medicaid Services Innovation Center (CMMI) has announced plans to  end six demonstration models launched under prior administrations. Among the canceled projects are two drug-focused payment models: the Accelerating Clinical Evidence model and the Medicare $2 Drug List. Important for ASGCT members: The Cell and Gene Therapy Access Model was not included in the list of models being terminated. This model, which enables CMS to negotiate outcomes-based agreements with sickle cell disease gene therapy manufacturers to remove burden on states, remains intact.  

Community Quick Take Tackles Immune Reponses to AAV 

ASGCT recently released a Community Quick Takes module focusing on the immune system's role in adeno-associated virus (AAV) gene therapy, as a follow up to the virtual workshop Class Considerations on Immunogenicity for AAV Gene Therapy Products: Assessing Current Practice and New Data. 

Pre-existing antibodies against specific AAV vectors are a crucial factor in determining patient eligibility for gene therapy, as different products have varying antibody thresholds. Managing immune responses is therefore essential, especially for higher-dose therapies targeting organs like the brain, where rapid antibody formation can occur. Ongoing research aims to develop strategies for immune modulation and redosing to enhance treatment efficacy and patient access. Be sure to check out our full Patient Education materials for more details AAV therapeutics. 

Read Our Regulatory Guidance Comments to FDA

ASGCT recently submitted comments to FDA on the draft guidance Expedited Programs for Serious Conditions—Accelerated Approval of Drugs and Biologics, emphasizing the importance of maintaining a predictable and flexible Accelerated Approval (AA) pathway for CGTs. Recommendations focused on ensuring CGTs remain eligible for AA, providing clearer criteria for early-stage sponsor engagement, refining FDA’s approach to surrogate endpoints, and recognizing alternative confirmatory trial designs, including real-world evidence and platform-based regulatory efficiencies. Gene therapies often provide rapid clinical benefits that may not align with traditional clinical trial endpoints; ASGCT urged FDA to maintain flexibility in evidentiary standards to avoid unnecessary delays in patient access. We thank FDA for its continued commitment to accelerating cures and expanding safe and effective treatment options for patients with serious medical conditions. 

ASGCT also appreciated the opportunity to comment on FDA’s draft guidance Frequently Asked Questions – Developing Potential Cellular and Gene Therapy Products. In our letter ASGCT expressed support for FDA’s efforts to provide regulatory clarity for new CGT developers; we also shared, however, that the document would be more useful if expanded to address complex challenges faced by experienced sponsors. ASGCT’s key recommendations focused on expanding the scope of the FAQ guidance, clarifying requirements for characterization testing and nonclinical study expectations, refining FDA’s approach to surrogate and intermediate clinical endpoints, providing additional details on tumorigenicity study expectations, and ensuring regulatory language accurately reflects current scientific and clinical realities. 

Share Your Insights on Gene Therapy Regulation  

Gene therapies hold immense promise for revolutionizing healthcare by addressing genetic disorders. However, the field’s complexity and evolving regulatory landscape pose significant challenges, particularly when navigating diverse global requirements. To tackle these hurdles and shape harmonized regulatory strategies, your input is crucial.  

The Hertfordshire University Pharmacy School is conducting a research survey to explore global cell and gene therapy regulatory concerns. ASGCT members are invited to share their valuable insights. The survey is anonymous and takes no more than 5 minutes to complete. Please complete the survey by March 31.

Empowering Patients Summit Available On Demand 

ASGCT hosted the Empowering Patients Summit earlier this month, bringing together stakeholders to address cell and gene therapy issues directly affecting patients and caregivers. The presentations delivered exceptional, accessible content that translated complex information into lay-friendly terms to help patients along the diagnostic and treatment odyssey, with speakers representing diverse perspectives from academia, industry, patient advocacy groups, and regulatory agencies. AgencyIQ noted this event was one of the few recent public speaking engagements for CBER Director Peter Marks. To view Dr. Marks' presentation and all the other valuable content from our presenters, watch the content on demand. 

Events

Save the Date for the 2025 Policy Summit

The Policy Summit will return September 25-26, 2025, at the Washington Marriott Georgetown in Washington, D.C.! Save the date now and watch this newsletter in the coming months to learn the latest on speakers, topics, and registration.