Advancing CGTs in 2024: ASGCT’s Policy and Advocacy Highlights

As 2024 draws to a close, the Advocacy team at ASGCT reflects on a year filled with impactful policy initiatives and significant milestones for the cell and gene therapy (CGT) field. 

ASGCT’s 2024 Advocacy Highlights 

• Member Expertise Highlights Congressional Testimony and Briefings  

  • For Rare Disease Week in February, ASGCT’s Vice President, Dr. Terry Flotte (right), was invited to testify in front of the House of Representatives Committee on Energy & Commerce Subcommittee on Health. Dr. Flotte highlighted the critical role of CGTs in addressing unmet need for rare diseases. 

  • The Society organized educational virtual briefings for staff in the House and Senate on critical CGT topics including gene editing, HIV, and hemophilia.  

• Federal Agencies Incorporate Society Feedback into Regulations and Strategy 

  • ASGCT successfully influenced the FDA’s finalization of key guidances, including those on genome editing and CAR T-cell products, ensuring a balance between regulatory rigor and innovation-friendly policies.  

  • The Society's feedback guided the National Center for Advancing Translational Sciences (NCATS) Strategic Plan to prioritize rare diseases, decentralized trials, and health equity in the final version. 

  • The Society requested to exclude CGTs from bundled payments in the 2025 Physician Fee Schedule proposed rule was also accepted into the final rule – a step towards adequate reimbursement for CGTs. 

• Society Establishes Inaugural Congressional Policy Fellow 

  • ASGCT’s first Congressional Policy Fellow, Adriana Bankston, PhD, started her work on Capitol Hill to integrate scientific expertise into legislative processes, focusing on policies to advance biomedical innovation. (Applications for next year’s fellowship are due February 24, 2025). 

• Molecular Therapy (MT) Family of Journals Publishes Policy Focused Articles 

  • New this month, Molecular Therapy Methods & Clinical Development published a piece on optimizing regulatory frameworks for gene therapies in rare diseases, authored by members of ASGCT’s Government Relations Committee. A companion piece on payment policy challenges for rare disease drug development is expected to be released early in the new year.  

  • The MT family of journals is actively soliciting additional articles on legislative and regulatory topics as patient access and commercialization issues have an increasing impact on the field. 

2024 Successes 

ASGCT works year-round to advance our goals of improving patient access, increasing research funding opportunities, streamlining FDA regulation, and more. The Society advanced these priorities through key efforts: 

Educational Events 

• The annual Policy Summit highlighted key issues in the field including regulatory harmonization, Medicaid payment reform, and the investment landscape for CGTs​.  

  • Notable speakers this year included Dr. Nicole Verdun, Director of the Office of Therapeutic Products; Dr. Daria Fedyukina, Program Manager with the Advanced Research Projects Agency for Health; Clare Pierce-Wroble, who at the time was with the Domestic Policy Council; and Dr. Joni Rutter, Director of the National Center for Advancing Translational Science. 

  • The event also brought together a host of global regulators, including Dr. Yoshiaki Maruyama from Japan’s Pharmaceutical and Medical Devices Agency (PMDA); Dr. Sol Ruiz representing the Spanish Medicines Agency (AEMPS); and Dr. Sarah Wassmer from Health Canada.  

• The Society engages with key regulators through our annual FDA Liaison Meeting. In 2024, we discussed: 

  • Class Considerations for Determining Safety and Efficacy Outcomes for AAV Gene Therapies    

  • Comprehensive On- and Off-target Analysis for Genome Editing 

• ASGCT hosted the CGT Science Series with the Biotechnology Innovation Organization (BIO) and FDA, aimed at educating FDA reviewers on new scientific developments in the field.   

• On the regulatory side, ASGCT launched registration for the upcoming Class Considerations on Immunogenicity for AAV Gene Therapy Products: Assessing Current Practice and New Data workshop. This event, hosted in partnership with FDA, will be held January 22-23, 2025.

1:1 Policy Engagement 

• ASGCT members and policy staff held constructive conversations with legislative offices about the pipeline for CGTs and anticipated approvals, regulatory considerations, and patient access challenges.   

  • We had in-depth discussions with policy staff of several key jurisdictional committees, including Senate Finance, Energy and Commerce, Appropriations,  House Budget Committee, and House Education & Workforce. 

  • ASGCT also met congressional staff  over the  course of the year, offering an opportunity to educate decision-makers and dive into our legislative priorities.  

• The Society shared our top policy priorities and discussed solutions in meetings with the Domestic Policy Council, Congressional Budget Office (CBO), the House Budget Committee Healthcare Task Force, CMS, and the Centers for Medicare and Medicaid Innovation (CMMI). 

Society Comments + Letters   

• The Society shared feedback with FDA on seven draft guidance documents affecting the field.  

  • FDA’s Draft Strategy Document on Innovative Manufacturing Technologies 

  • Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies  

  • Platform Technology Designation Program for Drug Development 

  • Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products  

  • Considerations for the Use of Human-and Animal-Derived Materials in the Manufacture of Cell and Gene Therapy and Tissue-Engineered Medical Products 

  • Potency Assurance for Cellular and Gene Therapy Products  

  • Advanced Manufacturing Technologies Designation Program 

• We responded to four requests for information (RFIs) from Congress and the National Institutes of Health on:   

  • Improving Americans’ Access to Gene Therapies  

  • The Employee Retirement Income Security Act  (ERISA)   

  • Enacted Cures 2.0 Act 

  • Securing and Enhancing Biomedical Supply Chains  

• We provided responses to several payment policy draft rule proposals: 

  • Hospital Inpatient Prospective Payment System (IPPS)  

  • Hospital Outpatient Prospective Payment System (OPPS) 

  • Additional stakeholder sign-on letters for IPPS and OPPS   

  • Physician Fee Schedule (PFS) 

• The Society joined a partnered sign-on letter supporting the Sickle Cell Disease Comprehensive Care Act. 

If you have questions about any of ASGCT’s policy goals or if you would like to find out about ways to be involved, contact ASGCT’s Chief Advocacy Officer, Margarita Valdez Martínez. 

Caitlin is ASGCT's Manager of Public Policy.

Banner photo by Getty Images Pro, licensed under Canva Pro.

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