The Advocate

September 2025: Volume 5, Issue 9

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In This Issue:

The Policy Summit Is Next Week!
Learn About NIH Federal Funding Developments
Read Our Payment Policy Comments
Apply for the ASGCT-AAAS Congressional Policy Fellowship
Congressional Leaders Engage with Federal Health Agencies
Newborn Screening Update
FDA Proposes New Framework: Rare Disease Evidence Principles (RDEP)
Community Quick Take: What to Expect in a Gene Therapy Clinical Trial
In Brief: Policy Updates

ASGCT Advocacy

The Policy Summit Is Next Week!

The countdown is on - the ASGCT Policy Summit kicks off next week in Washington, D.C.! On September 25–26, leaders from across the cell and gene therapy (CGT) ecosystem will gather for high-impact discussions on regulatory policy, payment systems, and access challenges shaping the future of CGTs. This year’s in-person format means more opportunities for real collaboration, strategic networking, and actionable insights you won’t find anywhere else. Don’t miss your chance to get ahead of market and policy dynamics that could define your CGT development strategy - review the agenda and register today.

Learn About NIH Federal Funding Developments

The House Labor-HHS Appropriations Subcommittee met after Labor Day and advanced its Fiscal Year (FY) 2026 funding bill, providing $48 billion for the National Institutes of Health (NIH). That proposal keeps NIH funding essentially flat compared to FY25, rejecting the 40% cut proposed in the President’s Budget Request this spring. The House appropriated $555 million to the Advanced Research Projects Agency for Health, a 37% decrease. By comparison, in July the Senate Appropriations Committee recommended $48.7 billion for NIH on a bipartisan basis, $700 million above the House’s proposal and $400 million above NIH’s FY25 budget. The Senate bill strongly supports CGT research and manufacturing and would also preserve the current facilities and administrative cost reimbursement structure.

The House Appropriations bill includes a new provision to cap facilities & administration (F&A or indirect) costs at 30% for a subset of private institutions with large endowments. Additionally, language from FY25 that protected the broader F&A system from administrative changes was not pulled through for FY26. In the FY26 report that accompanies the budget bill, House appropriators  noted the role of indirect costs in advancing biomedical research but called for changes to the system. ASGCT has advocated against cuts to indirects, leading a sign-on letter with other stakeholders to lay out how that funding is critical to CGT research and development.

Federal funding bills commonly highlight specific programs that appropriators want to prioritize. If signed into law, the House bill would provide $8.2 million for a sickle cell disease (SCD) treatment demonstration program that helps patients access quality, coordinated, comprehensive care through coordinated care teams. The bill encourages NIH to advance clinical research on promising approaches to eradicate SCD, including developing screening technologies and innovative treatments both domestically and globally. ASGCT recently hosted a congressional briefing on SCD that outlined the many access challenges patients face.

On August 21, the Supreme Court ruled in National Institutes of Health v. American Public Health Association to allow the Trump Administration to move forward with cancelling  $783 million in NIH grants tied to DEI, gender identity, and COVID-19 research - which had been challenged in the district courts. The ruling stated that grant termination challenges should instead be filed in the Court of Federal Claims rather than federal district courts.  In the mixed ruling, the high court left  in place the lower court rulings against NIH’s related policy guidance. The decision does not resolve whether the cancellations were legal but means funding can be withheld for now.

Meanwhile, in a separate court ruling, a federal judge ruled that the administration’s freezing of $2 billion in research funding for Harvard was illegal. The administration is expected to appeal. 

Read Our Payment Policy Comments

CMS released the FY26 Inpatient Prospective Payment System (IPPS) final rule. The rule maintains current policies for MS-DRG 018, continuing to exclude clinical trial cases from relative weight calculations, and retains the New Technology Add-On Payment (NTAP) framework. Both developments are in line with ASGCT's comments on the draft IPPS rule in May.

ASGCT also signed onto a stakeholder letter on the FY26 Medicare Physician Fee Schedule (PFS) Proposed Rule, which addresses autologous cell collection and preparation costs for CAR Ts and other gene therapies. Under the FY26 PFS proposal, CMS would continue to bundle cell collection and preparation into the overall CAR T product payment, rather than reimbursing them separately. However, manufacturer payments for these steps would be treated as discounts rather than bona fide service fees, potentially lowering reimbursement for providers. The letter requested CMS provide clarity and avoid policies that risk undervaluing physician work, reducing patient access, and limiting the number of sites able to perform these procedures.

Apply for the ASGCT-AAAS Congressional Policy Fellowship

Dr. Adriana Bankston, ASGCT’s first Congressional Policy Fellow, recently finished her time on Capitol Hill. Over the past year, Dr. Bankston represented the Society through the AAAS Science and Technology Policy Fellowships (STPF) Program. We are grateful for her leadership and contributions, and we look forward to seeing the lasting impact of her work. We are equally excited to welcome Dr. Rami Major as our next ASGCT Congressional Policy Fellow. Dr. Major, whose fellowship term started this month, will carry forward the Society’s commitment to bringing science-based insights to federal policymakers. We look forward to following her experience and the meaningful ways she will help shape policy in support of scientific innovation.

The application window for the 2026-2027 ASGCT-AAAS Congressional Policy Fellowship is now open. This unique opportunity is designed for scientists interested in engaging directly with the policymaking process, learning how legislation is developed, and applying their expertise to inform decisions that impact the future of research and patient care. For ASGCT members eager to explore the intersection of science and policy, this fellowship offers invaluable career development and the chance to make a tangible difference in advancing the CGT field.

Congressional Leaders Engage with Federal Health Agencies

The last month included several opportunities for Congress to engage directly with health agencies relevant to CGTs. On September 5 the NCATS Alliance, of which ASGCT is a member, organized the first congressional staff tour of NCATS laboratories, where NCATS Director Dr. Joni Rutter and her team highlighted key programs including the Clinical and Translational Science Awards (CTSA), National Clinical Cohort Collaborative (N3C), and rare disease research initiatives. From disease modeling to drug development platforms, translational science infrastructure is essential for advancing CGT treatments from bench to bedside. Congressional guests included Dana Richter (L-HHS Chair Capito), Kathleen Bochow (Sen. Boozman), Brittany Brignac (Sen. Rounds), and David Steury (Rep. DeGette).

On September 3, the House Energy & Commerce Subcommittee on Health held a hearing titled "Examining Opportunities to Advance American Health Care through the Use of Artificial Intelligence Technologies," exploring AI's expanding role in healthcare delivery and drug development. The discussion is particularly relevant to the CGT sector, where AI and machine learning are increasingly being applied to accelerate research and development, including predicting tumor epitopes for CAR T-cell therapies and facilitating the identification of appropriate antigens and binding sites for improved therapeutic design. ASGCT’s Policy Summit will host a session on Artificial Intelligence for Genetic Medicine Development, for any members looking for more information on how to apply these new tools to your bench and regulatory work. 

Finally, on September 4 the Senate Finance Committee held a hearing with HHS Secretary Robert F. Kennedy, Jr. Lawmakers sought information about recent vaccine policy decisions from the Food and Drug Administration (FDA) and Centers for Disease Control & Prevention (CDC), the $50 billion Rural Health Transformation Program, and various chronic disease initiatives through the Make America Healthy Again (MAHA) Commission. The administration expressed support for advanced therapies in its original MAHA Commission fact sheet, highlighting gene therapies for SCD. As a follow-up to the Senate Finance hearing, Dr. Susan Monarez, former CDC Director, was invited to testify in the Senate HELP Committee today.

Newborn Screening Update

ASGCT’s Policy Summit this month will feature a session on newborn screening (NBS), including a discussion on the current state of the system and ongoing efforts for reform. That session is especially timely, as Congress has been actively engaged with NBS in recent weeks. The Energy & Commerce Subcommittee on Health advanced the Newborn Screening Saves Lives Reauthorization Act on September 10. Dr. Kim Schrier (WA-08), a pediatrician and member of the committee, pointed to gene therapy clinical trials for cystic fibrosis as a success stemming from a strong NBS system. Meanwhile, both the House and Senate Appropriations Committee bill reports address funding for newborn screening. The House text acknowledged the approval of a gene therapy for metachromatic leukodystrophy (MLD) and requested an update on nominations to the Recommended Uniform Screening Panel (RUSP) for both MLD and Duchenne muscular dystrophy (DMD). The Senate text calls for a briefing on the activities of the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC), which was disbanded by the Department of Health and Human Services (HHS) in April.

FDA Proposes New Framework: Rare Disease Evidence Principles (RDEP)

FDA has proposed a new framework, the Rare Disease Evidence Principles (RDEP), to accelerate development of therapies for very  small, rare disease patient populations with serious unmet needs. RDEP would allow approval based on one adequate and well-controlled trial plus strong confirmatory evidence, such as natural history studies, external controls, case reports, or pharmacodynamic data. To be eligible, therapies must target a known genetic defect that drives the disease, involve patient populations generally under 1,000 in the US, and address conditions that lead to rapid disability or death with no existing therapies. Sponsors must apply to FDA before pivotal trials, and requests will be reviewed in consultation with the Rare Disease Policy and Portfolio Council. Drugs approved under RDEP may also carry post-marketing requirements to further confirm safety and effectiveness.

Community Quick Take: What to Expect in a Gene Therapy Clinical Trial

In a new 20-minute episode, Dr. Amy Waldman explores what participation in an investigational gene therapy trial means and how to best prepare for the unknowns. She answers common questions about trial design, compassionate use, realistic expectations for participants, potential risks, and why long-term follow-up is so critical.