The Advocate

Volume 1, Issue 7

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September 2021

In This Issue:

In-Person Policy Summit Registration Closes Sept. 16
ASGCT Events Address AAV Vector Integration
PDUFA Commitment Letter Includes ASGCT-Recommended Provisions
Policy News



In-Person Policy Summit Registration Closes Sept. 16

In-person registration for the 2021 Policy Summit ends tomorrow, September 16, and on-site attendance is limited, so register now to secure your spot! This hybrid event will be held September 22-24 virtually and in person at the JW Marriott Washington, DC. The Policy Summit will feature sessions on the following topics and more:

  • A fireside chat with Demetrios Kouzoukas, former director of the Center for Medicare and principal deputy administrator at CMS

  • Medicaid payment policy for gene and cell therapies

  • AAV vector integration

  • Policies related to accelerated approval

  • Other regulatory issues, including a keynote address by Andi Lipstein Fristedt, deputy commissioner for policy, legislation, and international affairs at FDA

See the full agenda on our website. ASGCT has instated a number of safety policies, including physical distancing and a universal indoor mask requirement for in-person attendees; read about all our safety plans here.

ASGCT Events Address AAV Vector Integration

Last month ASGST held a roundtable on AAV vector integration, which addressed preclinical research on the integration profile of AAV vectors, considerations for clinical risk assessment, and areas in need of further research. Watch the entire event on demand. To hear a summary of insights from the roundtable you can register to attend the hybrid Policy Summit, which features a session dedicated to the topic. In-person registration is limited and closes September 16, so register soon! At the Policy Summit you will also learn how the FDA's thinking on AAV integration is evolving and about risk/benefit considerations from the perspective of hepatologist Graham Foster, PhD.

ASGCT Advocacy

PDUFA Commitment Letter Includes ASGCT-Recommended Provisions

In August, the US Food and Drug Administration (FDA) released the commitment letter outlining proposed performance goals for the upcoming reauthorization of the Prescription Drug User Fee Act for FYs 2023-2027 (PDUFA VII). The PDUFA commitment letter includes the following proposals that ASGCT had requested in its comments last year on PDUFA VII:

  • Substantial strengthening of the Center for Biologics Research and Evaluation (CBER) staff capacity and capability to meet the increasing demands of the rapid expansion of submissions for cell and gene therapy products. Additional resources will include support of:

  • Increased CBER staff, including review staff

  • CBER staff recruitment and training

  • Facilitation of communication between CBER and sponsors through streamlining and harmonizing of cell and gene therapy product procedures, processes, and interactions, which will enhance regulatory consistency.

  • Development of a draft guidance on post-approval study requirements by the end of FY 2025, related to ASGCT’s request for attention to this area.

  • Updating of the Guidance for Industry: Expedited Programs for Regenerative Medicine Therapies for Serious Conditions by the end of FY 2025, that includes additional thinking on approaches and processes relating to CMC, including CMC readiness to utilize expedited programs.

The commitment letter included additional provisions that could be beneficial for the field, such as pilot programs to advance rare disease endpoints and to use split real-time application review.

Policy News


ASGCT Policy Summit

September 22-24, 2021

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