ASGCT Gears Up for 2021 Policy Progress

2020 Highlights

One role of the Society is to advocate for policies that are significant to its members in advancing the field of gene and cell therapy. In 2020, ASGCT furthered that goal through a number of key efforts, including:

• The third annual Policy Summit, which addressed responsible use of technology, newborn screening, patient access to new therapies, and regulatory policy
• The third annual ASGCT/FDA gene therapy liaison meeting to discuss key regulatory considerations in the field
• The first-ever collaborative event with the Brazilian Association of Cellular and Gene Therapy (ABTCel-Gen) to foster development of gene and cell therapies
• Stakeholder education and engagement via submission or sign on to 20 letters, as well as the development of policy recommendations on funding, reimbursement, and access issues including:
  • Robust NIH and FDA funding levels
  • Proposed regulatory rules impacting gene and cell therapy development
  • Payment policy improvements related to Medicare reimbursement and payment models
  • Newborn screening of conditions treatable by gene therapy

2020 Policy Successes

• Inclusion of some of ASGCT’s recommendations in six final FDA guidance documents on gene therapy
• Increases of $1 billion in NIH funding and of $42 million in FDA funding for Fiscal Year 2021
• Inclusion of ASGCT-recommended language in a bill to reauthorize the CW Bill Young Cell Transplantation Program that would codify attention to research funding for regenerative medicine into the Public Health Service Act (PHSA) and the explicit inclusion of gene therapies and genetically modified cells in the definition of regenerative medicine
• The use of a different methodology by the Centers for Medicare and Medicaid Services (CMS) to create a new Medicare Severity-Diagnosis Related Group (MS-DRG) for CAR T-cell therapy that more adequately reimburses providers (exclusion of clinical trial cases in the calculation)
• Finalization of a CMS rule that provides 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)
• Withdrawal of the Oklahoma Health Care Authority’s application for the Healthy Adult Opportunity (HAO) program, which could have restricted access to gene and cell therapies
• Re-establishment, after a lapse in its functioning, of the Advisory Committee on Heritable Disorders in Newborns and Children, a discretionary advisory committee that considers additions of conditions to the Recommended Uniform Screening Panel (RUSP)
• Invitation to join the Newborn Screening (NBS) Modernization Advisory Board, contributing to a multi-stakeholder effort to assess and advocate for improvements in the NBS system to incorporate new therapies

2021 Policy Priorities

This new year brings a new administration and new policy goals for ASGCT, including:

• Increased NIH funding for gene and cell therapy research
• Maximized Medicare reimbursement mechanisms for new gene and cell therapies
• Increased access to gene therapies for individuals covered by Medicaid
• Decreased barriers to new payment models for gene and cell therapies
• Improved processes for adopting new conditions onto the RUSP for newborn screening
• Improved FDA CBER resourcing and early communications with CBER
• Established regulatory requirements that promote efficient development of gene therapies
• Contribution to improved patient access to gene therapy in low- to middle-income countries

Input Needed! Continuing our policy successes and advancing our goal of unmitigated access to gene and cell therapies requires involvement from our members. Your participation is valuable! Here is how you can help: 

• Provide input on your challenges in obtaining NIH research funding (academic members)
• Look for your state’s Medicaid coverage policies for approved gene therapies and let us know if you are able to locate them
• Nominate yourself to serve on a policy-related committee by January 31
• Learn more by registering for the Annual Meeting and the policy-related Pre-Meeting Workshops preceding the meeting

Please contact ASGCT Policy and Advocacy Manager Christina Mayer regarding the above or with policy-related comments or suggestions.

Francesca Cook, MPH, is chair of the ASGCT Government Relations Committee.

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