The Advocate

Volume 2, Issue 4

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April 2022

In This Issue:

Save the Date for the 2022 Policy Summit!
Comments Coming on FDA's CAR T Cell, Genome Editing Guidance Docs
Increased Funds for NIH, FDA Included in Biden's FY22-23 Budget Request
Bills Seek to Reconcile Accelerated Approval Pathway
New Value-based Purchasing Bipartisan Bill Released
Senate Committee Advances PREVENT Pandemics Act
Policy News



ASGCT’s fourth annual Policy Summit will take place September 26-27 at the JW Marriott in a hybrid format – attendees will be able to join either in Washington, D.C. or online. New this year, the Policy Summit will be a 1.5-day program with a dialed-in focus on the key issues in regulatory and government affairs policy for gene and cell therapies. Information on the agenda and registration is coming soon! In the meantime, catch up with recaps of previous Policy Summits.

ASGCT Adocacy

Comments Coming on FDA's CAR T Cell, Genome Editing Guidance Docs

FDA released two new draft guidance documents last month: Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products and Human Gene Therapy Products Incorporating Human Genome Editing. The Society plans to submit comments to share our members’ on-the-ground, expert recommendations in these two critical areas of CGT development. Comments are due to FDA on June 16.

Increased Funds for NIH, FDA Included in Biden's FY22-23 Budget Request

Late last month, President Biden submitted his annual budget request to Congress. This request outlines the administration’s health policy and objectives, which include several gene therapy-specific items. ASGCT is tracking multiple areas of the President’s budget request, most notably $49 billion requested for the NIH (a 0.6% increase over FY22 when excluding funds intended for ARPA-H) and $3.7 billion to FDA (a 10% increase over FY22). ASGCT will continue to support robust appropriations for NIH.

The President’s request also designates $5 billion of NIH’s proposed budget for the creation of an Advanced Research Projects Agency for Health (ARPA-H). While initially established under the umbrella of Health and Human Services (HHS),Secretary Becerra announced the administration’s intention to house ARPA-H under the National Institutes of Health (NIH), with shared administrative functions but reporting directly to the Office of the Secretary. ARPA-H will serve to fund traditionally "high-risk" biomedical research to keep pace with this period of innovation. Of these opportunities for funding, ASGCT supports the growth of platform approaches to development of gene therapies, specifically improvements in manufacturing capabilities.

Bills Seek to Reconcile Accelerated Approval Pathway

In legislative hearings last month, the House Energy and Commerce Committee reviewed two proposed bills seeking to reform FDA’s Accelerated Approval Program. The program is intended to address unmet medical need by using surrogate endpoints to demonstrate efficacy and get breakthrough treatments to patients more quickly.

ASGCT believes robust post-market surveillance is critical to ensure approved products remain safe and efficacious; as more gene and cell therapies are approved by FDA that require further post-market assessment, it is critical that those assessments are designed to answer the scientific questions at hand, be practical to effectuate in the market, and not impede patient access. The Society will continue to engage with and educate key organizations and individuals on the unique nature of gene and cell therapies, and how the benefits and challenges of the accelerated approval pathway might be addressed to best benefit patients who rely on innovation for the timely development of new treatments.

New Value-based Purchasing Bipartisan Bill Released

Last week, legislators introduced the Medicaid Value-based Purchasing (VBP) for Patients Act. The bill aims to remove barriers for entering into VBP arrangements under Medicaid. These types of agreements help mitigate the costs of some of these transformative therapies by tying the cost of the product to the therapeutic benefit or outcome. ASGCT supports Medicaid payment policies that foster the adoption of, and patient access to, new therapies while avoiding undue financial strain to entities within the healthcare system. VBP arrangements in particular spread risks between payers and manufacturers and distribute costs more equitably based on individual patient outcomes, which has the potential to improve access to these lifesaving treatments. The Society is encouraged to see bipartisan interest on this issue and will monitor the bill as it moves through the House.

Senate Committee Advances PREVENT Pandemics Act

The Senate Health, Education, Labor and Pensions (HELP) Committee voted (20-2) to send an amended version of the PREVENT Pandemics discussion draft to the Senate for their consideration. ASGCT commented on the discussion draft back in February and is encouraged to see provisions retained that would facilitate the development of gene and cell therapies across more disease areas by harnessing the power of platforms and promote greater patient access to approved therapies.

Policy News

  • Last month the European Medicine Agency recommended to approve a new gene therapy, Janssen’s Carvykti, for the treatment of multiple myeloma. The drug was approved with conditional marketing authorization, similar to fast-track designations in the U.S.

  • The FDA approved Yescarta to move to an earlier line of therapy, allowing its use in large B-cell lymphoma after one initial treatment has failed. Yescarta’s clinical trial results show the CAR T-cell therapy outperformed chemotherapy and stem cell transplants and extended event-free survival time.

  • Mississippi became the first state this year to enact what’s known as Recommended Uniform Screening Panel (RUSP) alignment legislation, an approach that aims to implement newborn screening for new conditions within three years of being added to the RUSP.

  • Peter Marks, director of CBER, published an op ed advocating for reform and innovation in some aspects of the gene therapy development paradigm. Marks highlighted the potential for new approaches to the use of novel endpoints and clinical trial designs, manufacturing process standardization, enhanced communication with regulatory authorities, and global regulatory convergence to improve patient access.

  • The International Council for Harmonization released a new draft guidance earlier this month on extrapolating data in drug development. Specifically, the guidance aims to establish guidelines around applying data from adult trials to pediatric populations.

  • Rep. Fred Upton, co-sponsor with Rep. Dianna DeGette of Cures 2.0, announced earlier this month that he will not seek re-election and will retire after 36 years in the House of Representatives.

  • Despite the acceleration in the gene therapy pipeline, clinical holds and unfavorable market conditions have forced multiple companies to restructure, cutting jobs, withdrawing from global markets, and prioritizing certain indications.

  • CMS announced that Medicare coverage of Alzheimer’s gene therapy treatment Aduhelm, which received accelerated approval status earlier this year, will be limited to those in clinical trials. This decision came after months of controversy surrounding the drug’s efficacy in post-market studies. 


ASGCT 25th Annual Meeting

May 16-19, 2022

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