Recent Collaborative Government Affairs Actions

ASGCT Opposes Repeal of the Orphan Drug Tax Credit

Updated: December 5, 2017

The Orphan Drug Tax Credit (ODTC) currently allows drug manufacturers to claim a tax credit of 50 percent of the qualified costs of clinical research and drug testing of orphan drugs. The House version of the Tax Cuts and Jobs Bill would completely repeal the tax credit, and the Senate version would reduce the ODTC rate to 27.5 percent (instead of current law’s 50 percent rate). A NORD/BIO economic analysis of the ODTC found that without the ODTC, approximately 33 percent fewer orphan therapies would have been developed over the previous 32 years, and 33 percent fewer orphan therapies will be developed going forward if the tax credit is repealed, which would be a critical blow to individuals with rare diseases. ASGCT signed onto a letter sent to House leaders signed by over 200 rare disease patient organizations opposing repeal of the ODTC.

Orphan Product Extensions Now – Accelerating Cures and Treatment

Updated: October 16, 2017

The OPEN Act (S. 1509) establishes an exclusivity extension, which would provide an additional six months of market exclusivity for a drug or biological product approved by the FDA when the product is additionally approved to treat a new indication that is an orphan disease.

Per the EveryLife Foundation for Rare Disease, scientific literature shows that a single targeted drug is likely to have multiple therapeutic uses and that biopharmaceutical companies can repurpose drugs for the treatment of different diseases. Doing so is faster, cheaper, and presents fewer risks than traditional drug development.

The legislation has bipartisan support, sponsored by Sens. Orrin Hatch (R-UT) and Robert Menendez (D-NJ) and Representatives Bilirakis (R-FL) and Butterfield (D-NC), with 26 total sponsors in the House (9 Democrats, 17 Republicans). It is also supported by 268 patient organizations, including Genetic Alliance, Global Genes, National MPS Society, the National Organization for Rare Disorders, and the Pediatric Cancer Foundation. ASGCT has joined the EveryLife Foundation’s list of supporting organizations.

Sign-On Letter Opposing Restrictions on Fetal Tissue Research

Updated: August 28, 2017

On August 28, 2017, ASGCT joined 62 other organizations in signing on to a letter opposing restrictions on the use of federal funding for fetal tissue or embryonic stem cell research. HR 3358—the House Labor, Health and Human Services (HHS), and Education Appropriations Act, 2018—currently contains a provision that would prohibit federal funds from being used to conduct or support research using human fetal tissue if such tissue is obtained pursuant to an induced abortion. The letter discourages the Senate from adopting restrictions in appropriations, such as those found in HR 3358.

Sickle Cell Disease (SCD) Coalition

Updated: June 7, 2017

ASGCT representatives and other SCD Coalition members attended a briefing on Capitol Hill on June 7, 2017, in partnership with the House Research & Development Caucus, the Congressional Caucus to Cure Blood Cancers and Other Blood Disorders, and the Congressional Sickle Cell Disease Caucus. Arranged by the American Society for Hematology, the SCD Coalition hosted the briefing to highlight advancements in SCD research and treatment options, including gene editing, a promising potentially curative treatment for SCD under current research.

ASGCT is always open to exploring additional collaborative opportunities. Please contact Betsy Foss-Campbell, Strategic Alliance Manager, at for more information.

Government Relations Publications

Read recent ASGCT publications including the white paper on gene editing and our response to the National Center for Advancing Translational Sciences request for information.


ASGCT 24th Annual Meeting

May 11-14, 2021