Co-Principal Investigators: Karen J. Maschke, The Hastings Center; Michael K. Gusmano, Lehigh University; Jennifer B. McCormick, Penn State

Funder: National Human Genome Research Institute of the National Institutes of Health

August 2024

Several gene therapies have been approved over the last few years and hundreds more are being tested, raising hopes for patients with rare disorders, various cancers, and complex neurological diseases. But there are ethical and policy challenges on the path from research to the clinic, including: What is sufficient evidence of safety and effectiveness to justify approval of a gene therapy? How can safety and effectiveness of approved therapies be monitored in the clinic? How can these therapies be made accessible and affordable to patients?

For this three-year project, beginning in 2024, the principal investigators, working with an advisory committee, will explore how gene therapy’s ethical and policy challenges are being identified and addressed. Information will be gathered through analysis of federal and state policy documents, a survey of physicians from four specialties (hematology, infectious disease, neurology, and oncology), and in-depth interviews with experts from six domains (medicine, translational gene therapy research and development, the Food and Drug Administration processes for drug approval and post-marketing review, patient advocacy, and ethical issues in genetics and genomics).

Outcomes of the project will include recommendations on addressing gene therapy challenges.