The Advocate

Volume 1, Issue 1

March 2021

In This Issue:

ASGCT Stresses the Value of CAR T-Cell Therapy for Multiple Myeloma
Society Commends FDA for Draft Guidance on Individualized ASOs
Policy News
Register for Pre-Meeting Workshops on Policy and Clinical Development

ASGCT Advocacy

ASGCT Stresses the Value of CAR T-Cell Therapy for Multiple Myeloma

Last week, ASGCT signed on to a response letter to ICER’s draft evidence report on treatments for heavily pre-treated relapsed and refractory multiple myeloma, including two CAR T-cell therapies (ide-cel and cilta-cel). Along with other members of a CAR T-cell therapy working group, ASGCT expressed concern that the clinical and financial data utilized in the report are premature for the evaluation of these two CAR T-cell therapies because the clinical benefits to patients are still evolving. In the letter, the groups recommend ICER further consider factors such as the value of a one-time therapy and the significant increase in overall survival of patients receiving CAR T-cell therapy compared to standard-of-care treatments. Upon approval of these valuable therapies for new indications, ASGCT will continue to advocate for full coverage and improved reimbursement of providers for CAR T-cell therapies to facilitate patient access.

Society Commends FDA for Draft Guidance on Individualized ASOs

Because ASGCT is supportive of the development of molecular therapies for very rare diseases to address unmet needs, the Society last week submitted comments to FDA CDER on a draft guidance document on IND submissions for individualized antisense oligonucleotides. ASGCT focused on commending FDA for its provision of procedural guidance for these smaller trial populations for sponsor-investigators who are less likely to have extensive experience with regulatory processes. The Society also recommended including the following:

A reference to guidance on regulatory considerations and data and quality recommendations.
Provision of suggestions about the situations in which the administrative and procedural aspects outlined in the guidance document would not be applicable to developing other individualized therapeutic modalities, to potentially broaden the scope of guidance.

Policy News

Share ASGCT’s new CAR T-cell therapy resources that introduce patients and their families to payment policy challenges to access these therapies. This information is timely with FDA approval of a fourth CAR T-cell therapy over the last month (Breyanzi) and accelerated approval for a new indication for Yescarta.
Elizabeth Fowler will lead the CMS Center for Medicare and Medicaid Innovation (CMMI), a position that does not require Senate approval. The Biden Administration has nominated other key health positions, including:
Xavier Becerra as Secretary of HHS, who received an evenly-divided committee vote on March 3 after his Senate Finance Committee hearing and is awaiting a vote in the full Senate.
Chiquita Brooks-LaSure as Administrator of the Center for Medicare and Medicaid Services (CMS).
Eric Lander to lead the Office of Science and Technology Policy (OSTP).
As part of ASGCT’s global access efforts, the Society is contributing to a pre-meeting course and a plenary session at the Brazilian Association of Cell and Gene Therapy (ABTCel-Gen) Annual Meeting, to be held virtually on April 28 – May 1.
A recent GAO report raised concerns about a large backlog of FDA inspections. While much of the current backlog is related to the COVID-19 pandemic, FDA CBER has faced ongoing challenges in addressing the rapidly increasing demands of gene and cell therapy development. ASGCT consistently advocates for FDA to receive the resources it needs for staffing levels to keep pace with the needs of the field.

Events

Register for Pre-Meeting Workshops on Policy and Clinical Development

ASGCT has a full slate of in-depth pre-meeting workshops on May 10 focused on policy, clinical development, and career development

10 a.m.–2 p.m. (ET)

Patient Advocates’ Role in Advancing Gene Therapy
Recent Developments in Global Regulation of Gene Therapies
Transitioning from Academics to Industry

3–7 p.m. (ET)

Early-Stage Development
Issues in Market Access
Standards in Gene Therapy and Genome Editing

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