Publication
Published on: March 12, 2026
Published in: Hastings Bioethics Forum
Description: In “Genetic Therapies for Rare Diseases: Developing Ethical Regulatory Policies,” a commentary published in the Hastings Bioethics Forum, Carolyn Riley Chapman, former lead of the MRCT Center’s Cell and Gene Therapies project, and co-author Nirvan Bhatia argue that the U.S. should adopt a differentiated approval system for gene-based therapies for rare diseases, conferring different tiers of regulatory approval based on different levels of evidence. The authors contend that this approach would improve patient access to innovative therapies while increasing transparency about evidentiary standards and providing stronger incentives for timely post-marketing studies. The MRCT Center’s Toolkit for Supporting the Design and Conduct of Long-Term Follow-Up Studies for Gene Therapies, released in January, offers practical guidance on related challenges in gene therapy research.
