Written Testimony: Legislative Proposals to Support Patients with Rare Diseases

 Written Testimony Of

Chairman Guthrie, Vice Chair Bucshon, Ranking Member Eshoo, and Members of the Subcommittee, thank you for the opportunity to testify on behalf of the American Society of Gene & Cell Therapy (ASGCT). I am Dr. Terence Flotte, Provost and Dean of the University of Massachusetts Chan Medical School, and Vice President of ASGCT. The Society is a nonprofit professional membership organization comprised of more than 6,200 members. Working in settings such as universities, hospitals, and biotechnology companies, these members are scientists, physicians, patient advocates, and other professionals working together to advance the field of gene and cell therapy.

The mission of ASGCT is to advance knowledge, awareness, and education leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease. ASGCT's strategic vision is to be a catalyst for bringing together diverse stakeholders to reshape the practice of medicine by incorporating the use of these transformative therapies. Many of ASGCT’s members, like myself, have spent their careers in this field performing the underlying research that has led to today’s robust pipeline of cell and gene therapies (CGTs).

America is clearly the world leader in gene therapy, both in terms of pioneering gene therapy science and technology and in the application of gene therapy to human diseases. ASGCT represents many of the scientists, physicians and regulatory professionals from academia, industry and government who have propelled the US into this leadership position. These therapies are transformational and, as such, they have challenged the treatment and payment paradigms of our health care system.¹ My professional experience is rooted in being a physician researcher, and my testimony today will focus on research, product development, regulatory issues, and the patient experience.

¹ The Society supports policies that foster the adoption of, and patient access to, new therapies. However, ASGCT’s support does not imply a position on any individual pricing, coverage, or reimbursement decisions.