Policy Goals for 2022: Increasing Gene Therapy Access, Funding

The Society works to advance the field by advocating for policies in the areas of FDA regulation, patient access, research funding, and genetic screening and testing. Read on to learn how we plan to do this for the rest of this year, as well as our accomplishments of 2021.

2022 Goals 

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

• FDA/Regulatory Policy: Improve CBER resourcing, early communications (INTERACT meetings), CMC review, and platform regulation 

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

• Access and Reimbursement of Gene Therapies: Increase provider reimbursement for new gene and cell therapies, correct disparate reimbursement based on site of service, and improve access to products (agnostic to payor) 

• 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  

2021 Highlights

In 2021, ASGCT furthered its goals through a number of key efforts, inlcuding:

• The first-ever hybrid Policy Summit, which addressed Medicaid coverage considerations, accelerated approval considerations, patient views on therapy opportunities and challenges, and many other hot topics

• The fourth annual ASGCT/FDA gene therapy liaison meeting to discuss key regulatory considerations in the field

• A Roundtable on AAV Integration discussing the knowns and unknowns of AAV risk, translatability of animal models, and regulatory implications

• Membership in the Bespoke Gene Therapy Consortium to help accelerate the delivery of gene therapies for rare disease patients

• Society feedback to FDA on four new draft guidance documents that impact the field

• Three global events with partner societies in Brazil, South Africa, and India

• Stakeholder education through 18 Society-led letters or partner sign-ons addressing key topics such as:

  • User fee reauthorization and support for CBER

  • The critical role and unique challenges of Chemistry, Manufacturing, and Controls (CMC) in gene and cell therapy development

  • Effectuation of innovative payment models for gene and cell therapies including value-based purchasing arrangements under Medicaid

  • Appropriate Medicare reimbursement levels to providers to facilitate patient access to cutting-edge new products

  • Support for translational research and cross-sector partnerships

  • Robust NIH and FDA funding levels

  • Newborn screening of conditions treatable by gene therapy

2021 Advocacy Successes

• The Centers for Medicare & Medicaid Services (CMS) issued a final rule that in 2022 will provide states, private payers, and manufacturers with more flexibility to enter into value-based purchasing (VBP) arrangements for innovative therapies by allowing reporting of multiple Medicaid best prices. ASGCT has advocated since 2018 for the removal of barriers to VBP models for gene therapies.  

• As a result of advocacy from ASGCT and many other organizations that helped highlight the value of robust federal funding, the final 2021 omnibus budget bill, signed into law December 27, 2021, included FY 2021 increases of $1 billion for NIH and $42 million for FDA. 

• ASGCT-recommended language was included in a bill to reauthorize the CW Bill Young Cell Transplantation Program, which would codify attention to research funding for regenerative medicine into the Public Health Service Act (PHSA) and would explicitly include gene therapies and genetically-modified cells in the definition of regenerative medicine. 

• ASGCT provided valuable input on the strategic plans for the National Eye Institute (NEI) and the National Institute of Neurological Disorders and Stroke (NINDS).  

• After advocacy from a coalition of organizations including ASGCT and led by the EveryLife Foundation, new laws were passed in Georgia and Ohio to better ensure their state newborn screening (NBS) programs stay up to date with new additions to the federal Recommended Uniform Screening Panel (RUSP). 

• Outcomes from the FY22 Inpatient Prospective Payment System (IPPS) final rule were primarily positive based on ASGCT recommendations. In its comments to the proposed rule, the Society advocated for further consideration of the appropriateness and patient access implications before grouping these two types of therapies together on a long-term basis. 

• The PDUFA VII commitment letter includes proposals that ASGCT had requested in its comments, including additional CBER staffing and training; improved formal and informal communications between FDA and sponsors; and additional guidance on post-approval study requirements and CMC readiness. 

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

Ms. Valdez Martínez is ASGCT's Director of Policy and Advocacy.