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?
These questions are the focus of a new project led by Karen J. Maschke, PhD, a senior research scholar at The Hastings Center; Michael K. Gusmano, PhD, a professor of health policy at Lehigh University; and Jennifer B. McCormick, PhD, MPP, an associate professor at Penn State College of Medicine. The $1,484,725 three-year project is supported by the National Human Genome Research Institute of the National Institutes of Health.
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).
“How gene therapy challenges and the associated ethical and policy issues are characterized and addressed have implications for patient safety and health outcomes, clinicians and health care systems, gene therapy developers and research funders, and public trust in and support of the translational gene therapy enterprise,” says Maschke.
Outcomes of the project will include recommendations on addressing gene therapy challenges.
For more information, contact:
Susan Gilbert
Director of Communications
The Hastings Center
gilberts@thehastingscenter.org
845-424-4040 x244