The Advocate

June 2025: Volume 5, Issue 6

Want to stay up to date on news and events relevant to patients, caregivers, and others affected by rare diseases?

In This Issue:

Register for the 2025 Policy Summit
ASGCT Leadership Joins CBER Roundtable on CGTs
Watch the January AAV Immunogenicity Workshop
Provide Feedback on Proposed New F&A Model
Read Our IPPS Comments for FY26
Learn More About the FY26 Federal Budget Details
Senate Continues to Debate FY25 Reconciliation
In Brief: Policy Updates
Around the Field

ASGCT Advocacy

Register for the 2025 Policy Summit

The Policy Summit will be held Sept. 25-26 at the Washington Marriott Georgetown in Washington, D.C.! The 2025 Policy Summit will dive into regulatory policy developments; clinical access bottlenecks; European payment systems for CGTs; and CMC harmonization efforts. Keep an eye on this newsletter to get updates on session topics, confirmed speakers, and networking opportunities. We’ll see you there!

ASGCT Leadership Joins CBER Roundtable on CGTs

ASGCT President Terry Flotte, MD, speaks during a CBER roundtable on CGTs.

FDA gathered academic leaders and patient advocates for a Cell & Gene Therapy Roundtable on June 5. FDA Commissioner Dr. Martin Makary and CBER Director Dr. Vinayak Prasad hosted a wide-ranging discussion on accelerating safe, affordable access to CGTs.

ASGCT President Dr. Terence Flotte spotlighted the de-prioritization of commercially pre-viable therapies and urged FDA to expand Platform Designations, embrace Advanced Manufacturing Technologies, and preserve the Rare Pediatric Disease Priority Review Voucher (PRV). ASGCT’s Immediate Past-President Dr. Paula Cannon traced the field’s evolution from bone-marrow transplantation to bespoke CRISPR therapies like baby KJ's, arguing that accumulated experience now allows responsible regulatory acceleration. Other panelists called for continuation of FDA pilot programs, adoption of flexible trial designs, strong post‐approval surveillance, and expanded use of the Accelerated Approval pathway. Participants also discussed that current payment and reimbursement models are misaligned with the realities of one-time treatments.

At the close of the roundtable, HHS Secretary Robert F. Kennedy Jr., NIH Director Dr. Jay Bhattacharya, and CMS Administrator Dr. Mehmet Oz pledged coordinated federal action. They pointed to sickle cell disease as a test case for implementing value-based payment models that could help expand access to CGTs with high upfront costs. They also underscored the importance of flexible evidence standards and innovative financing mechanisms to ensure these treatments reach more patients. Check out ASGCT’s blog for a full event summary.

Watch the January AAV Immunogenicity Workshop

ASGCT’s Class Considerations on Immunogenicity for AAV Gene Therapies virtual workshop is available to watch on the ASGCT website. This free public event brought together leading regulatory, academic, and industry experts to explore shared immunology-related challenges across AAV gene therapies - including immune responses, antibody testing, redosing strategies, and the role of preclinical models. The workshop offered actionable insights for developers and researchers, addressing both scientific and regulatory considerations.

Provide Feedback on Proposed New F&A Model

The Joint Associations Group (JAG) on Indirect Costs released their draft proposal for alternatives to the current Facilities & Administration (F&A) reimbursement model for federal research grants. JAG is seeking feedback on the model by June 22. You can find the full list of JAG resources here, including the proposal, feedback link, and previous webinar recordings.

Read Our IPPS Comments for FY26

ASGCT submitted a comment letter in response to the FY26 Inpatient Prospective Payment System (IPPS) proposed rule. ASGCT voiced support for the Center for Medicare and Medicaid Services’ (CMS) continued refinement of the Medicare Severity Diagnosis-Related Group 18 (MS-DRG 18) - related to CAR T-cell therapies - to more accurately reflect the resource needs of CAR Ts. However, the Society emphasized that broadening this DRG to include non-genetically modified immunotherapies risks distorting reimbursement rates. In addition, ASGCT reiterated the critical importance of the New Technology Add-On Payment (NTAP) program in enabling access to transformative cell and gene therapies. The Society urged CMS to adopt several improvements to NTAP, including establishing multiple review periods per year, increasing the cap to 100% of product costs, and allowing more flexibility in DRG creation for rare disease products. In addition to sending our own comments, ASGCT added our voice to a group letter with scientific societies and patient advocate groups stressing similar points.

Learn More About the FY26 Federal Budget Details

The fiscal year (FY) 2026 federal appropriations process is underway. In recent weeks, additional details have become available related to FDA and NIH budget proposals:

The President’s FY26 budget request calls for a 4% decrease in overall FDA funding. FDA is requesting a 11% cut to congressional appropriations, while simultaneously increasing user fees by 4%. The U.S. House Appropriations Committee subsequently released their draft budget bill, which follows the President’s request. The Committee’s report includes language encouraging FDA to modernize its approach to CGTs by expanding the use of accelerated approvals, incorporating novel measures to assess safety and efficacy, and improving coordination with patients and across FDA centers on acceptable levels of risk.
NIH Director Bhattacharya also published the FY26 budget justification for NIH, providing funding estimates for each institute and rationale for the administration's proposed $18 billion (40%) reduction in NIH funding. The proposed budget includes a significant consolidation of NIH Centers alongside changes to grantmaking policies, including a cap on senior-level salaries and a 15% ceiling on F&A reimbursements for grant recipients.
On June 10 the Senate Appropriations Subcommittee on Labor, HHS, and Education held a hearing with Director Bhattacharya on the proposed FY26 NIH budget; ASGCT submitted a statement for the record highlighting breakthroughs made possible by federally funded research and reiterating the importance of facilities and administration (F&A) reimbursements. In the hearing, U.S. Senate Appropriations Committee Chair Collins emphasized that NIH cuts pose a threat to the scientific workforce, patient access, and US global competitiveness - her remarks can be viewed here. The U.S. House Appropriations Committee is expected to mark up its budget in July.

Senate Continues to Debate FY25 Reconciliation

Late in May, the U.S. House of Representatives passed their version (H.R.1) of the FY25 reconciliation package. On June 16 the US Senate Finance Committee released their version of the bill text, which addresses several key healthcare provisions linked to CGTs.

Changes to current Medicaid policy include the imposition of work requirements and shortening enrollment periods, which could result in 10.9 million people becoming uninsured - a particular concern for the rare and pediatric disease communities. The bill also takes a two-tiered approach to freeze or lower state provider taxes: states that have not expanded Medicaid will have their rates frozen, while expansion states face gradual reductions and caps. States use provider taxes to increase their federal reimbursement level for Medicaid services. Separately, the bill would expand the Inflation Reduction Act’s exemption from Medicare price negotiations to include drugs with multiple orphan indications, not just those with a single rare disease use. Finally, the package prohibits PBMs from using tactics like spread pricing under Medicaid, reflecting long-standing reform efforts.

The bill also includes tax proposals that would impact hospitals, providers, and research institutions. First, a proposed limit on deductions for certain pass-through entities would impact healthcare providers who operate through partnerships or LLCs, effectively raising their taxes. Second, the bill proposes increased taxes on private university endowments, though at a level lower than the House’s version (a maximum 8% tax rate compared to the House’s 21%). Academic institutions have raised concerns about that proposal’s potential negative impacts on research and innovation.

The Senate will spend the coming weeks discussing their version, with additional changes likely; once finalized, it will need to pass with a simple majority vote. At that point it will be sent back to the House for another vote, where the Senate’s changes may create challenges to passage.

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In Brief: Policy Updates

FDA Industry Listening Tour: FDA launched a national listening tour to engage directly with pharmaceutical and biotech industry leaders. Four meetings have been held to date; FDA Commissioner Makary and CBER Director Prasad discussed takeaways from the first roundtable on their FDA Direct podcast.
FDA Leadership Vision: Commissioner Makary and CBER Director Prasad outlined their priorities for a “new FDA,” calling for faster drug approvals and greater use of AI and big data in regulatory science. They emphasized that they envision shifting FDA to be more proactive, transparent, and health-focused.
New Pathways at FDA: FDA Commissioner Makary announced the agency is exploring new pathways and pilot programs to accelerate drug development, with plans to release details in the near future. He advocated for the use of real-world data and synthetic controls beyond traditional randomized controlled trials.
FDA Post-marketing Data: FDA released its FY23 annual report on the performance of drug and biologics firms in meeting post-marketing requirements and commitments. The report details the status of required and agreed-upon post-approval studies, including compliance with reporting timelines and fulfillment of study obligations.
CMMI Strategic Plan: The Centers for Medicare & Medicaid Services (CMS) Innovation Center (CMMI) has released a new 2025 strategic plan. CMMI oversees health care pilots including the CGT Access Model.

Around the Field

Two federal agencies recently issued requests for input on advancing medical research through technology. The National Library of Medicine wants feedback on research gaps in biomedical informatics and data science that could be addressed through its funding programs (due July 14), while the National Cancer Institute is seeking input on AI benchmarks for cancer research and care (due July 29).
FDA has granted a platform technology designation to a gene therapy vector, the first time such a designation has been publicly announced. This designation allows developers to apply existing data from one treatment to others using the same platform, helping accelerate the development and review of future gene therapies.
The National Academies’ Committee on Strategies to Enhance Pediatric Health Research is seeking input from both patients and researchers to inform their work. Share your feedback to help shape future priorities and improve pediatric research infrastructure. Pediatric health researchers can submit their responses by July 1. Patients, families, and caregivers are invited to share their experiences by July 13. 
FDA is seeking public suggestions for topics for its upcoming “Rare Disease Innovation, Science, and Exploration (RISE)” workshop series. These workshops will focus on challenges that affect multiple rare diseases and explore innovative, science-driven solutions. Comments are due by December 31, 2025 and can be submitted here.