Looking Back at 2023's Policy and Advocacy Highlights

ASGCT works to advance the goals of improving patient access, increasing research funding opportunities, streamlining FDA regulation, and so much more. Read on to learn about what our Policy and Advocacy team is proud of and excited about from the last year

2023 Advocacy Highlights

• Medicaid coverage practices for approved gene and cell therapies: Existing barriers and proposed policy solutions was published on current challenges surrounding patient access to CGTs under the public payor system, and potential federal levers to enforce full coverage to label.  

• Staff and members met in Washington, D.C. for briefings in both the House and Senate on CAR-T applications and access, as well as separate briefings on the future of sickle cell disease and the treatment pipeline. 

• New policy-focused articles were published in Molecular Therapy (MT) – Methods and Clinical Development, including a commentary on comparability and phase-appropriate product development plans from ASGCT’s CMC Committee.   

  • The MT family of journals is looking to include more legislative and regulatory articles as post-approval, access, and commercialization issues become more widely apparent.  

• ASGCT, OTP (formerly OTAT), and other stakeholders engaged in a half-day workshop on immune responses to AAV vectors, requirements for immunogenicity testing, and the utility of companion diagnostics.  

2023 Successes 

In 2023, the Society advanced its priorities through key efforts such as: 

• The fifth annual Policy Summit, which highlighted key issues in the field including coding and reimbursement, patient-centered development, manufacturing/CMC, standards, and more.   

  • Notable speakers included Dr. Nicole Verdun, Director of the Office of Therapeutic Products (OTP) new super office, Dr. Renee Wegrzyn, Director at the Advanced Research Projects Agency for Health (ARPA-H), and Laura McWright, Deputy Director at the Centers for Medicare & Medicaid Services (CMMI). Summit attendees heard remarks from Reps. Brett Guthrie (KY-02) and Scott Peters (CA-50), as well as Rep. Anna Eshoo’s (CA-16) office. 

• Stakeholder advocacy through three Society-led letters and partner sign-ons addressing key topics such as:  

  • The fiscal year 2024 Inpatient Prospective Payment System 

  • The power of associations in supporting their respective fields and the broader economy 

  • The adjustments to the New Technology Add on Payment (NTAP) 

• Society feedback to FDA on three draft guidance documents that impact the field.  

• Responses to FDA and OSTP requests for information on: 

  • Harnessing biotechnology and biomanufacturing policy to support research and development 

  • Collaborative efforts to support safe and effective development of individualized therapies  

• Comments to the public workshop on Advancing the Utilization and Supporting the Implementation of Innovative Manufacturing Approaches. ASGCT’s response highlighted the need for advanced manufacturing technologies (AMTs) in order to streamline the development process for CGTs. 

• A new CGT Science Series with the Biotechnology Innovation Organization aimed at educating FDA reviewers on key scientific areas of interest to ASGCT members.  

• Continued conversations with legislators and their staff about the pipeline for CGTs, anticipated approvals, and patient access challenges.  

  • The Society met with key legislative offices, and staff from the US Senate Finance and HELP Committees.  

  • In the U.S. House of Representatives, ASGCT met with Energy & Commerce Committee staff, and staff on the Ways and Means committee. 

• Educational and introductory meetings with CMMI, ARPA-H, and OSTP to discuss policy solutions. 

• Addressing changes to existing reimbursement policies that may threaten patient access to innovative therapies, or place undue burden on developers and manufacturers. 

•  The Congressional Policy Fellowship which will place an ASGCT member in an office on Capitol Hill for one year in order to provide science-based support for policymakers in the CGT space. 

If you have questions about any of ASGCT’s policy goals or would like to find out about ways to be involved, please contact me.

Margarita Valdez Martínez is ASGCT's director of policy and advocacy.

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