Legislative Updates

ASGCT Partners with ARM for Legislative Fly-In

Updated: April 10, 2018

ASGCT members are invited to register to attend a Legislative Fly-In presented by the Alliance for Regenerative Medicine (ARM) in collaboration with the American Society of Gene & Cell Therapy in Washington D.C. on May 23.

Each participant will be assigned to a group based on their home state and districts. Attendees will meet with key members of Congress and federal officials to describe the immense near-term potential of advanced therapies and the need for legislation that supports the development and access to transformative gene and cell therapies.

Sickle Cell Disease Legislation Passes House, Introduced in Senate

Updated: March 6, 2018

The House of Representatives last week passed HR 2410, the Sickle Cell Disease Research, Surveillance, Prevention, and Treatment Act. Among other provisions, this legislation would enhance research and surveillance efforts, which would improve the current understanding of this life-long disease and potentially identify more curative options. Treatments for this disease are limited and often focus on relieving symptoms, reducing pain, and avoiding crises and complications. At this time, hematopoietic stem cell transplantation is the only cure. Several gene therapy trials are now underway for sickle cell disease (SCD), with promising early results. No explicit funding for SCD has been provided since 2009.

Also last week, Senator Cory Booker (D-NJ) and Senator Tim Scott (R-SC) introduced the companion bill yesterday (S 2465), which was referred to the Senate Health, Education, Labor and Pensions Committee. ASGCT signed on to a letter of support, along with 66 other organizations, to Senators Scott and Booker. In a round table discussion yesterday, Senator Booker provided more information about the bill and spoke with patients, a parent, and physicians about its significance.

ASGCT Supports PACT Act

Updated: February 22, 2018

ASGCT sent a letter of support to Rep. Erik Paulsen for the Protecting Access to Cellular Therapies (PACT) Act, which he sponsored. This legislation would ensure that hospitals providing hematopoietic cell transplantation (HCT) to Medicare beneficiaries receive adequate payments. Typical cell acquisition costs for HCTs currently consume all or most of the Medicare reimbursement rate, which is insufficient to also cover hospital costs for the lengthy inpatient stay required for these transplants. The PACT Act would require Medicare to cover acquisition costs separately from the Medical Severity Diagnosis Related Group, as it does for solid organ transplants. Doing so would protect access for Medicare beneficiaries who are in need of a life-saving bone marrow or cord blood transplant for serious blood cancers and blood diseases, including leukemia, multiple myeloma, and sickle cell disease. Find more information at Be the Match.

ASGCT Supports Biomedical Research Act

Updated: February 22, 2018

ASGCT has joined over two dozen other organizations in signing on to support the National Biomedical Research Act (S. 2212). This bill would provide a new funding stream of $5 billion annually for ten years to a Biomedical Research Fund to be distributed to the NIH and FDA, supplementing existing appropriations. The funding would be targeted to select research categories, including “disruptive innovation”—breakthrough research on diseases with unmet medical needs or for which current treatments are inadequate. Other focus areas relevant to ASGCT’s priorities include basic research, research by early career scientists, regulatory science, and the development of new medical products. Sen. Elizabeth Warren introduced the legislation, along with 15 original co-sponsors, as a way to restore some of the NIH funding cuts that occurred for over a decade. The bill would bring the NIH budget back to approximately 2006 levels, adjusted for inflation.

Impact of the Tax Plan on Gene and Cell Therapy

Updated: December 22, 2017

Both chambers of the United States Congress ushered through a bill to drastically transform American tax policy. That reconciled bill was signed by the president with a couple of added provisions that both maintain conditions for graduate researchers and maintains half of the Orphan Drug Tax Credit, a research incentive used to develop groundbreaking treatments for rare diseases. Earlier in the reconciliation process, 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 ODTC as part of his long history as an advocate for rare disease research. When the House of Representative first aimed to eliminate the tax credit entirely, ASGCT signed onto a letter sent to House leaders signed by over 200 rare disease patient organizations opposing repeal of the ODTC. Additionally, the Trainee committee issued a position statement about how the tax bill would hurt graduate students who receive tuition waivers from their universities: Why the House Tax Plan on Tuition Waivers is Bad for Science and for America.

Read ASGCT's Response to the Tax Law

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.

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 and Robert Menendez and Reps. Gus Bilirakis and G.K. Butterfield, 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.

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. 

Regulatory Updates

Read about regulatory changes that effect the field of gene and cell therapy. 

22nd Annual Meeting
April 29 – May 2 | Washington D.C.