Legislative and Regulatory Updates


Impact of the Tax Plan on Gene and Cell Therapy

Updated: December 21, 2017

Both chambers of the United States Congress ushered through a bill to drastically transform American tax policy. That reconciled bill is soon headed to the White House with a couple of added provisions that both maintain conditions for graduate researchers and reinstate a fraction of the Orphan Drug Tax Credit, a research incentive used to develop groundbreaking treatments for rare diseases.

Read ASGCT's Response

ASGCT Opposes Repeal of the Orphan Drug Tax Credit

Updated: December 12, 2017

In anticipation of reconciliation, ASGCT president Dr. Helen Heslop and Government Relations Committee chair Dr. Rachel Salzman sent a letter to the chairman of the Senate Finance Committee and member of the reconciliation committee for the Tax Cuts and Jobs Act. The letter calls upon Sen. Hatch to save the Orphan Drug Tax Credit (ODTC) as part of his long history as an advocate for rare disease research.

"The Society appreciates your long history of encouraging the innovation of treatment of rare diseases through your sponsorship of the Orphan Drug Act and the OPEN ACT and your service as Senate co-chair for the Rare Disease Congressional Caucus, among other efforts," the doctors wrote in their letter. "ASGCT urges you to continue this support by advocating for the preservation of the Orphan Drug Tax Credit in the final version of the Tax Cuts and Jobs Act."

The 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over the previous 32 years, and 33 percent fewer orphan therapies will be developed going forward if the tax credit is repealed, which would be a critical blow to individuals with rare diseases.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.

Trainee Committee Opposes House Tax Bill Provision

Updated: December 1, 2017

The Trainee committee wrote a news article on how the The Tax Cuts and Jobs Act (HR 1) will hurt graduate students who receive tuition waivers from their universities. Currently, graduate students are only taxed on the stipends they receive from their academic institutions. However, the House aims to change that by counting tuition waivers as income. This change will harm the field of gene and cell therapy. The Trainee Committee explains Why the House Tax Plan on Tuition Waivers is Bad for Science and for America.

ASGCT Responds to CMMI Plan to Test New Pricing and Payment Models

Updated: November 16, 2017

ASGCT submitted a comment on November 16, 2017 to a request for information from the Center for Medicare and Medicaid Innovation (CMMI), which requested input on its plans to test models in eight focus areas, including new pricing and payment model designs for prescription drugs, with the goals of empowering beneficiaries as consumers, providing price transparency, increasing choices and competition to drive quality, reducing costs, and improving outcomes. ASGCT comments included a recommendation that CMMI consider testing existing and new payment methodologies for gene and cell therapies to ensure access to care to these durable and potentially curative treatments. Additionally, ASGCT recommended that if outcomes-based testing models for FDA-approved gene therapies are created, CMMI establishes a process to obtain input from experts in the field to contribute to identifying the criteria that will define successful outcomes, as well as the anticipated time frame for such criteria to be attained.

FDA Issues Comprehensive Regenerative Medicine Policy Framework

Updated: November 16, 2017

The FDA announced a comprehensive regenerative medicine policy framework, outlined in two finalized guidance documents, and two draft guidance documents. The two final guidance documents clarify the FDA’s interpretation of the risk-based criteria for manufacturers to use to determine whether a product is subject to the FDA’s pre-market review. The two draft guidelines provide important information to spur and streamline development of innovative regenerative therapies. The draft guidance documents both have 90-day comment periods.

ASGCT Members Inform the Senate HELP Committee About Gene Editing

Updated: November 14, 2017

At a recent hearing on gene editing technology, the Senate Committee on Health, Education, Labor, and Pensions (HELP) listened to testimony from experts in the field about scientific, regulatory, and ethical aspects of the topic. Two of the three invited witnesses at the hearing are members of ASGCT—Matt Porteus, MD, PhD, a member of the ASGCT Board of Directors, and Katrine Bosley. Speakers informed the committee about gene editing technologies, which have now entered the clinical trials phase of research. All three speakers indicated that current, robust regulatory mechanisms are in place to ensure the safe use of gene editing for its intended medical purposes. Read more about the hearing and download our press release.

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 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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