Advocacy Reflects on Accomplishments and Looks Ahead to Future Efforts

2019 Highlights

One job of the Society is to advocate for the work of its members in the field of gene and cell therapy. With the help of other stakeholders in the field, here are some of the ways we did that in 2019. Last year, we:

• Hosted 354 attendees in an expanded, three-day Policy Summit in Washington, D.C. addressing global regulatory, payment, access, and ethical issues
• Held the second annual ASGCT/FDA gene therapy liaison meeting in Washington, D.C. to discuss key regulatory considerations in the field
• Co-hosted three briefings on Capitol Hill on policies relating to gene therapy for sickle cell disease and on Medicaid policies for CAR T-cell therapy
• Submitted 11 letters and established in-person relationships with 93 legislative staff members, giving policy recommendations on:
  • NIH funding levels
  • Common issues in drug development for rare diseases
  • Value assessment of transformative therapies
  • Novel payment models for gene therapies
  • Medicare coverage and reimbursement of CAR T-cell therapy
  • Clinical use of germline gene editing

As a result of those efforts, here are some of the accomplishments we’re proud of from 2019:

• Addition of ASGCT-recommended language to a sickle cell trait resolution that recognizes the importance of the development of, and access to, new treatments for sickle cell disease
• Inclusion of ASGCT-recommended revisions to FDA guidance documents on expedited pathways for regenerative medicine and rare disease drug development
• ASGCT’s invitation to become a voting member of the United States Pharmacopeial (USP) Convention, a standards-setting organization
• Streamlining of regulatory oversight through the elimination of the Recombinant DNA Advisory Committee’s review and reporting requirements for human gene therapy protocols
• Establishment of a Medicare national coverage determination for CAR T-cell therapies
• An increase in the cap on new technology add-on payments, which increased reimbursement levels to providers for CAR T-cell therapies
• Inclusion of the issue of novel payment models in proposed legislation
• Passage of the Pandemic and All-Hazards Preparedness and Advancing Innovation Act, which included a provision to address the potential role of gene editing in national health security
• Increases of $2.6 billion in NIH funding and of $92 million in FDA funding for 2020

2020 Priorities

The Society will continue to focus on additional policy priorities identified in its next three-year strategic plan in 2020. Related to our strategic priority on access, ASGCT policy staff have met with legislators as part of the organization’s support of reauthorization and strengthening the federal newborn screening program for improved diagnosis of genetic disorders that may be treated with approved gene therapies. Additionally, ASGCT supports a new provision in the bill that would make it easier to find out how to nominate a condition for inclusion on the Recommended Uniform Screening Panel (RUSP). Another ASGCT-supported new provision would encourage states to expedite adoption of testing of conditions added to the RUSP.  

ASGCT has also been increasing engagement with other organizations. Last month, the Society submitted comments in response to the National Eye Institute’s request for information on its next strategic plan. We emphasized the need to prioritize gene and cell therapy research generally, as well as in specific areas, such as vector development, large animal models, multigenic indications, and preclinical study of gene editing in ocular contexts. ASGCT also sponsored the Foundation for the NIH’s International Summit in Human Genetics and Genomics last summer. The summit aims to assist researchers from developing countries as they work to expand knowledge and infrastructure related to the research and treatment of genetic diseases. This year, ASGCT will be sponsoring one of the early career scientists to attend the month-long training program at the National Human Genome Research Institute.

To stay up to date on all of our efforts, make sure you read our blog and follow us on Twitter at @ASGCTherapy.

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