The Advocate

Volume 2, Issue 9


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September 2022

In This Issue:

Last Chance to Register for 2022 Policy Summit
Society Responds to Synthetic Oligonucleotide Guidance
GCTs Require Special Consideration in Infrastructure Act
States Are Implementing Restrictive Medicaid Coverage for Gene and Cell Therapies
Get to Know Your P&A Team!
Policy News

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Events


Last Chance to Register for 2022 Policy Summit

We are less than one week until the Policy Summit! This hybrid event will take place September 26-27 virtually and in person at the JW Marriot Washington, D.C. In-person space is limited, so make sure to register soon to secure your spot. Both in-person and virtual registrants will have access to on-demand sessions following the event, so don’t worry about missing any content.

This year’s keynote speaker is John Coster, PhD, from the division of pharmacy at the Center for Medicare and Medicaid Services (CMS). Dr. Coster will discuss Medicaid coverage of gene and cell therapies and he'll provide an overview of the Biden Administration's efforts to make gene and cell therapies more accessible to patients. Dr. Coster's lunchtime keynote will cover the following topics and more:

  • A level setting, historical perspective on the authority of CMS and their interactions with state programs

  • The role of CMS in ensuring Medicaid beneficiaries have equitable access to new gene and cell therapies

  • How CMS is implementing changes to the Medicaid best price rule to facilitate new value-based arrangements

  • Further actions CMS might consider in working with states to ensure equitable patient access to cell and gene therapies

Following Dr. Coster’s presentation, both in-person and virtual attendees can participate in an audience Q&A. Join your colleagues in productive conversations about the future of Medicare and Medicaid coverage of these advanced therapies.

ASGCT Advocacy


Society Responds to Synthetic Oligonucleotide Guidance

In addition to responding to draft FDA guidance documents, ASGCT weighed in on the proposed updates to the Screening Framework Guidance for Providers of Synthetic Double-Stranded DNA. Originally created by the Department of Health and Human Services (HHS) in 2010, the existing guidance has served as a series of best practices for users and manufacturers of nucleic acid synthesis technologies. This is of increasing interest as these starting materials advance at a rapid pace and are becoming the backbone of advanced therapies.

A new blog post by ASGCT Treasurer Paloma Giangrande, PhD, details ASGCT’s comments on the proposed screening updates for synthetic oligonucleotides. ASGCT’s comments addressed many of the practical barriers to implementation and touched on several key areas of proposed changes. Comments included concerns about screening length of RNAs for biosecurity, streamlining data sharing when appropriate, and additional preemptive purchasing requirements. ASGCT will continue to engage in this space and address regulatory and legislative barriers to the development of oligonucleotide therapies.

GCTs Require Special Consideration in Infrastructure Act

ASGCT recently submitted comments related to the Infrastructure Investment and Jobs Act (“Infrastructure Act”), which establishes a new requirement for drug manufacturers to provide “refunds” to the government for discarded amounts of a product after administration. The Society encouraged CMS to develop policies that acknowledge the unique nature of gene and cell therapies and provide a flexible and nuanced approach.

The Infrastructure Act requires manufacturers to provide a refund to CMS for “certain discarded amounts from a refundable single-dose container or single-used package drug.” The amount of the refund is the difference between the discarded amount and an applicable percentage, which is at least ten percent, of total charges for the drug in a given quarter This becomes challenging for gene and cell therapies which have specific considerations around manufacturing and administration. Generally, gene and cell therapies are designed to be implemented through a one-time (or few-time) dose. Effective administration of the treatment thus necessitates having a sufficient quantity of the therapy available at the one time of administration. Insufficient or “barely enough” amounts could lead to inferior outcomes and risk of the health and well-being of the patient. In addition, the personalized, low-dose characterization of these therapies requires additional steps in preparation that may contribute to “waste.” The legislation excluded radiopharmaceuticals, certain drugs requiring filtration, and certain new drugs from the requirement. Due to the complex preparation and administration techniques for gene and cell therapies, ASGCT is also requesting an exemption from CMS.

The Society acknowledges the intent of the Infrastructure Act and supports CMS’ efforts to reduce product waste and patient costs. Due to the personalized and limited-dose nature of gene and cell therapies, ASGCT asks that these treatments be excluded from this provision. For more detail and background on ASGCT’s comments to CMS, read our full comments online.

States Are Implementing Restrictive Medicaid Coverage for Gene and Cell Therapies

ASGCT’s Government Relations Committee has undertaken a project to help elucidate the issues surrounding Medicaid coverage of gene and cell therapies, and recently published an editorial to highlight the initial findings. The review compared a selection of state Medicaid and Medicaid managed care organization (MCO) coverage policies for three FDA approved advanced therapies – Luxturna, Kymriah, and Zolgensma. The results of this survey, along with anecdotal evidence from patients and providers, confirm that some states are implementing restrictive Medicaid coverage of cell and gene therapies, often limiting access to populations defined by the eligibility criteria for the clinical trial(s) rather than the FDA-approved labeled indication.

ASGCT is in the process of building out this editorial piece into a longer white paper. The findings from the data will be used to help identify barriers to full coverage and propose federal policy solutions to help the Medicaid system better accommodate the incoming wave of gene and cell therapy approvals. This white paper will be submitted for peer review before being distilled into a one-pager to be used as data-driven support in conversations with legislators and regulators. As the pipeline for cell and gene therapies continues to grow, ASGCT encourages federal and state regulators to take proactive steps to ensure that payment systems are ready and designed for this type of innovation so that patients can readily access these therapies.

Get to Know Your P&A Team!

As the cell and gene therapy field continues to grow, your ASGCT Policy and Advocacy (P&A) team is finding new ways to support research, development, and commercialization of these therapies through public policy channels. The goal of ASGCT’s P&A department is to proactively inform legislators and regulators about gene and cell therapies, and to develop policy relationships that will help  promote broad patient access to approved therapies.

Caitlin McCombs, MBA, works as an Advocacy Program Specialist in the P&A department. In her role, she provides administrative support to the Government Relations, Regulatory Affairs, CMC, and Ethics committees for their various projects. Caitlin has been with ASGCT since March 2021 and has recently focused on congressional outreach, supporting a monthly newsletter to Capitol Hill and various congressional briefings on cell and gene therapy. Caitlin is also managing the drafting process for a forthcoming white paper on Medicaid coverage policies, which will serve as a detailed follow-up to a recent Molecular Therapy editorial on the subject.

Caitlin completed her degree in 2021 and as such serves as the department’s “resident young person,” a prestigious title earned by her unwillingness to send a fax and overzealous use of the exclamation point.

Have a question about ASGCT’s policy priorities or want to get involved? Contact Caitlin at any time at cmccombs@asgct.org, or reach the P&A team at advocacy@asgct.org.

Policy News


2022

ASGCT Policy Summit

Watch On Demand Through Oct. 27

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