Legislative and Regulatory Updates


CMS Issues Interim Q Code for Tisagenlecleucel

Updated: November 8, 2017

The Centers for Medicare and Medicaid Services (CMS) has issued an interim Q code for the reporting of the use of tisagenlecleucel (Kymriah), effective as of January 1, 2018. The code is Q2040, for tisagenlecleucel, up to 250 million CAR-positive viable t cells, including leukapheresis and dose preparation procedures, per infusion. The timing of the recent FDA approval of this CAR T-cell therapy for certain instances of pediatric acute lymphoblastic leukemia prevents CMS distribution of a permanent J code until at least 2019. ASGCT supported this and similar future requests for timely assignment of interim codes for CAR T-cell therapies through a letter to CMS. Use of specific codes could facilitate expedited, more accurate reimbursement decisions, to allow patient access to these potentially lifesaving therapies.

Orphan Drug Tax Credit

Updated: November 8, 2017

The Orphan Drug Tax Credit (ODTC) allows drug manufacturers to claim a tax credit of 50 percent of the qualified costs of clinical research and drug testing of orphan drugs. 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over the previous 32 years, and 33 percent fewer orphan therapies will be developed going forward if the tax credit is repealed, which would be a critical blow to individuals with rare diseases.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 Congressional leaders signed by over 200 rare disease patient organizations in support 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.

FDA Orphan Products Grants Program Funding of Rare Disease Natural History Studies

Updated: October 10, 2017

For the first time, on October 7 the FDA awarded six new research grants for natural history studies in rare diseases to conduct rare disease natural history studies, through its Orphan Products Grants Program. The FDA is providing a total of $6.3 million over the next five years to fund four natural history studies and, through a partnership with NCATS Therapeutics for Rare and Neglected Diseases program, the FDA received $3.5 million to be combined with FDA funding to fund an additional two studies.

Such research can inform clinical trial development, and may lead to the use of natural history models to augment or replace placebo arms in studies of therapies for very rare diseases, for which trial recruitment can be difficult and for which withholding treatment may pose ethical concerns.

The grants support research on Friedreich’s ataxia; pregnancy and lactation-associated osteoporosis; sarcoidosis; sickle cell anemia to determine biomarkers of endothelial function changes in chronic kidney disease; Angelman syndrome; and myotonic dystrophy type 1 to determine biomarkers and clinical endpoints.  

ASGCT Supports Sickle Cell Disease Legislation

Updated: Septmeber 26, 2017

On Septmber 26, 2017 Congresswoman Barbara Lee (CA) distributed a dear colleagues letter to the House of Representatives requesting cosponsors for the Sickle Cell Trait Resolution. ASGCT was among the supporters listed in the letter. The resolution calls on the Department of Health and Human Services to develop a surveillance and public awareness campaign regarding the importance of knowing one’s sickle cell trait status.

Updated: October 19, 2017

On October 19, HR 2410: Sickle Cell Disease Research, Surveillance, Prevention, and Treatment Act was reported from the House Committee on Energy and Commerce, with an additional sponsor, Rep. G. K. Butterfield (NC). This bill would amend the Public Health Service Act to reauthorize a sickle cell prevention and treatment demonstration program and to provide for sickle cell disease research, surveillance, prevention, and treatment. The bill was committed to the Committee of the Whole House and placed on the Union Calendar. 

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.

Recent Collaborative Government Affairs Actions

Learn about ASGCT's most recent legislative actions and join our collaborative efforts. 

2018
21st Annual Meeting
May 16 - 19 | Chicago
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