ASGCT's Annual Advocacy Plan

Over the past year, ASGCT’s public policy activities supported the interests of its members in the gene and cell therapy fields. The Society advocated for legislative policies, educated governmental entities about gene and cell therapy, and kept the research community informed about relevant developments.

Highlights include:

• Participating as a member of the Sickle Cell Disease (SCD) Coalition in a briefing on Capitol Hill to highlight advances in SCD research and treatment options, including bone marrow transplantation and gene editing. Additionally, the Society supported the Sickle Cell Trait Resolution calling for sickle cell trait research, surveillance, and public education.
• Providing information to the Senate Committee on Health, Education, Labor, and Pensions through the testimony of two members—Board member Matt Porteus, MD, PhD and Katrine Bosley—at a congressional hearing, Gene Editing Technology: Innovation and Impact.
• Opposing a tax on graduate student tuition waivers. The proposed tax was not included in the final tax bill.
• Opposing repeal of the Orphan Drug Tax Credit, which allows drug manufacturers to claim a tax credit for a portion of the qualified costs of clinical research and drug testing of orphan drugs. The tax credit was retained in the final tax bill, although at the reduced rate of 25 percent, from 50 percent.
• Joining the EveryLife Foundation for Rare Diseases’ list of supporters of the OPEN ACT (Orphan Product Extensions Now, Accelerating Cures and Treatment), which would establish an exclusivity extension of an additional six months of market exclusivity for a drug or biological product being repurposed for a rare disease treatment.
• Supporting timely assignment of interim codes for CAR T-cell therapies through a letter to the Centers for Medicare and Medicaid Services (CMS).

Looking into 2018, ASGCT will continue to represent its members in similar and new ways. As ASGCT monitored the proposed funding of the NIH in 2017, it will continue to do so in 2018 to support maximal funding, especially for basic, translational, and clinical research related to gene and cell therapy.

The Society continues to support sufficient funding of the FDA through its new membership in the Alliance for a Stronger FDA.

ASGCT is also participating in Rare Disease Week and will have representation at a number of events throughout the week, including Lobby Day on Feb. 27 (hosted by the EveryLife Foundation for Rare Diseases) and Rare Disease Day at the NIH on Mar. 1 (hosted by NCATS and the NIH Clinical Center).

A new area of emphasis for 2018 will be addressing the issues surrounding the value of gene therapy. As gene therapies have now come to market, ASGCT will promote discussion among stakeholders of how to maximize patient access to these life-changing and potentially lifesaving treatments.

The Society will provide comments to relevant FDA draft guidance documents this year. Offering expert input into the regulatory process can guide the efficient, safe development of new gene and cell therapies for the treatment of a number of diseases with significant unmet needs.

Finally, ASGCT is collaborating on a legislative fly-in hosted by the Alliance of Regenerative Medicine on May 23 to offer members of the regenerative medicine community the opportunity to speak with their members of Congress on issues of importance to the field.

All these efforts will serve to educate policymakers and other governmental bodies to guide informed decision making on these crucial topics, and to promote legislative and regulatory support of the field in this exciting phase of its development.

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