Looking Back at a Year of Advocacy Progress

2022 Successes 

The Society works through proactive and reactive advocacy channels to further the policy goals of improving patient access, increasing research funding opportunities, streamlining FDA regulation, modernizing payment policy models, and more. In 2022, ASGCT advanced the priorities of the Society through a number of key efforts such as: 

• The Fourth Annual Policy Summit, which discussed key issues in the field including diversity in CGT, accelerated approval, PDUFA reauthorization, and many other hot topics  

• ASGCT’s fifth FDA Liaison Meeting addressed key regulatory topics for researchers and legislators, with two presentations from the Society and one from FDA on RMAT applications 

• Stakeholder education through 8 Society-led letters and partner sign-ons addressing key topics such as: 

  • Sickle cell disease legislation 

  • FY23 appropriations 

  • Synthetic oligonucleotides 

  • Pandemic preparedness 

  • Potency assays 

  • PDUFA VII 

• Society feedback to FDA on fifth new draft guidance documents that impact the field 

• An expert roundtable with ARM and FDA to address stakeholder questions on potency assay requirements and considerations  

• Commentary titled Planning for progress: A US regulatory approach to advancing the clinical development of gene therapies, which explores how regulators and the new PDUFA commitments can best support CGT development   

• Bipartisan briefing with the House Cancer Caucus on CAR T-cell therapy applications, pipeline, and access considerations 

• New monthly newsletters to Capitol Hill sharing the Society’s key issues and activities including diversity in clinical trials, CAR-T basics, and disease-specific topics such as sickle cell anemia 

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

2022 Advocacy Highlights: 

• As a result of advocacy from ASGCT and many other organizations who helped to highlight the value of robust federal funding, the final 2022 omnibus budget bill, signed into law December 23, 2022, included FY 2023 increases of $2.5 billion for NIH and $226 million for FDA.  

• Inclusion of ASGCT recommended language in FDA’s final guidance on Human Gene Therapy for Neurodegenerative Diseases. This guidance now addresses concerns on manufacturing controls, comparability analysis, potency assay validation, and purity requirements. 

• Final Studying Multiple Version of a CGT Product guidance reflects ASGCT comments, and provides further clarity on the timing of separate versions of IND submissions for the same CGT product.  

• PDUFA reauthorized with ASGCT provisions including reforms to the accelerated approval pathway, efforts to improve clinical trial diversity, provisions for platform technologies, and the creation of an advanced manufacturing program. 

• In partnership with RTI International, ASGCT supported a newborn screening modernization study and provided subject matter experts to support the first of two publications, published earlier this year.  

• A committee-led review was published in Molecular Therapy detailing Medicaid coverage challenges and patient access considerations for cell and gene therapies following a review of three key products.  

2023 Goals 

The Society’s policy goals include, but are not limited to, the following:  

• FDA/Regulatory Policy: Implement CBER changes and OTAT, CMC review, and platform regulation 

• Medicaid Policy: Educate on the need for access to cell and gene therapies for individuals covered by Medicaid, with a focus on the pipeline and the need for proactive solutions now 

• Patient Access to Gene Therapies: Support patient access to approved therapies including full coverage of FDA-labeled indications, appropriate use of utilization management tools, and establishing innovative payment mechanisms 

• Genetic Testing and Screening: Support broader adoption of RUSP conditions in states, accelerate adoption of new conditions onto the RUSP, enable providers to use whole genome sequencing where appropriate to diagnose rare diseases more rapidly 

• Research Funding: Increase funding for gene and cell therapy research through NIH and ARPA-H  

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

Caitlin McCombs is ASGCT's Senior Advocacy Program Specialist.

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