The Advocate

Volume 1, Issue 2

April 2021

In This Issue:

ASGCT Supports Flexible Approach to Regulation for Neurodegenerative Diseases
ASGCT Voices Concern Over MACPAC Proposal to Congress
PDUFA VII Direction Appears Promising
Policy News


ASGCT Advocacy

ASGCT Supports Flexible Approach to Regulation for Neurodegenerative Diseases

On April 6, ASGCT submitted comments to the FDA Center for Biologics Evaluation and Research on its draft guidance document, Human Gene Therapy for Neurodegenerative Diseases. Regarding communication with sponsors, the Society requests identification of the criteria the Agency uses to prioritize the granting of INTERACT meetings, given its resource constraints. In addition, ASGCT suggests ongoing dialogue during development regarding ideal endpoints.

To facilitate expedient development of safe and effective therapies, ASGCT also requests flexibility in testing requirements in areas such as:

  • CMC requirements for programs with expedited designations
  • The point in development to perform a potency assay (before BLA filing vs. before Phase 3 studies)
  • The point in development to submit CQA data

ASGCT indicated appreciation of the increased flexibility noted in the FDA recommendation that the residual host cell-DNA levels be set to less than 10 ng/dose, if possible, though the Society continues to caution against listing this suggested limit, as this level may be well below a safe dose of host-cell DNA.

ASGCT Voices Concern Over MACPAC Proposal to Congress

ASGCT shared concerns with MACPAC (the Medicaid and CHIP Payment and Access Commission) that its recommendation to Congress to increase the minimum rebate to states for drugs that receive accelerated approval could negatively affect patient access. MACPAC’s proposal could disincentivize the use of the FDA’s Accelerated Approval Program, increasing the time to market for therapies. ASGCT met with staff at MACPAC in early March to share these concerns, to highlight the unique value of gene therapies, and to express confidence in FDA oversight of product safety and efficacy. MACPAC provides policy and data analysis and advises Congress, the U.S. Department of Health and Human Services, and the states on issues affecting Medicaid and CHIP.

PDUFA VII Direction Appears Promising

The Prescription Drug User Fee negotiations between FDA and the drug industry for the seventh iteration of the program (PDUDA VII) are in the final stages, according to public meeting minutes. While the final commitment letter will likely not be released for several more months, ASGCT is pleased with the direction the new agreement seems to be taking. For instance, industry and FDA have discussed including a Split Real-Time Application Review (STAR) for certain products, which would provide more interaction and assistance to developers. The Society is also encouraged with the focus on properly resourcing the Center for Biologics Evaluation and Research to ensure greater allocation of fee dollars to support the burgeoning pipeline of gene and cell therapies.

Policy News

  • ASGCT released its first quarterly report last week on the landscape of gene, cell, and RNA therapy development from the preclinical to preregistration stages. The data within the publication reflects the continued rapid pace of development in the field, which drives many of the Society’s policy priorities.

  • Members of ASGCT’s Regulatory Affairs Committee and Society staff published a review article in Molecular Therapy: Methods & Clinical Development titled Global Regulatory Progress in Delivering on the Promise of Gene Therapies for Unmet Medical Needs. The article addresses potential solutions for the regulatory challenges of developing gene therapies multi-nationally.

  • Biden continues to build his healthcare team with the confirmation of Xavier Becerra as Secretary of Health and Human Services (HHS) on March 18. Chiquita Brooks-LaSure, nominated as Administrator of the Center for Medicare and Medicaid Services (CMS), had her Senate Finance Committee confirmation hearing on April 15. Eric Lander’s confirmation hearing to become Director of the Office of Science and Technology Policy will take place April 29.

  • The HHS Office of the Inspector General (OIG) issued an advisory opinion last month that allows Novartis to receive an exemption from federal anti-kickback statute (AKS) restrictions, to make Kymriah available at no charge for uninsured or low-income patients who have been denied coverage. While an advisory opinion is limited to the requesting company, this instance of eliminating the AKS barrier to patient access has the potential to pave the way for future requests.

  • Earlier this year ASGCT supported a bill in Georgia to expedite the addition of RUSP conditions to the state’s newborn screening program. On April 9 the Georgia Legislature sent HB 567 to the Governor for his signature.

  • Spotlight on the science that underpins ASGCT advocacy: Dosing began in a clinical trial for a new therapy—CAR-macrophages (CAR M) therapy, which applies CAR engineering to macrophages rather than T-cells in an attempt to target solid cancers. The technique was originally developed by ASGCT member Dr. Saar Gill and then graduate student, Michael Klichinsky, at the University of Pennsylvania.


  • As part of our patient advocacy efforts, we are co-hosting a CME program with the National Organization for Rare Disorders on April 22, to increase knowledge about gene therapy among clinical providers. Register here for The Time Is Now: Understanding Gene Therapy, Applications, and Implications.

  • Remember to register for the ASGCT Annual Meeting May 11-14, which includes these among other policy-related sessions:

    • Regulatory Lessons Learned from COVID-19: Anomaly to Precedent

    • Newborn Screening: Innovative Policies and Technologies to Eliminate the Diagnostic Odyssey

    • Viral Vector Safety: A Renewed Focus on Vector Safety and Innate Immune Responses to Leading Viral Vectors

    • Racial Justice in the Gene Therapy Field

  • ASGCT is hosting a virtual Forum on Gene Therapy for Underserved Populations: Drug Development for Ultrarare Diseases and Lower-Income Countries to address the challenges and solutions to developing sustainable approaches to development. Registration is now open for this June 22 event.

If you'd like to receive The Advocate via email every month, please contact Advocacy Program Specialist Caitlin McCombs.


ASGCT 24th Annual Meeting

May 11-14, 2021

This site uses cookies to offer you a better user experience and to analyze site traffic. By continuing to use this website, you accept our use of cookies.