September 8, 2025

9:30 am ET - 1:00 pm ET

Location:

Ethics of Rare Disease Research 

Topic: Ethics of Rare Disease Research

Abstract

Research in rare diseases presents several ethical challenges which have to date been under-explored. Decisions about whether to initiate rare disease research in the first place can be complicated by economic realities, given that the eventual market for rare disease therapies may be quite limited. How should companies and research funders generally balance economic and feasibility considerations with the needs of rare patient groups? Other challenges characterize the conduct of rare disease research. Some of these involve issues related to privacy and confidentiality. The smaller the patient population, the easier it may be to identify participants and individuals within clinical trial datasets, even when efforts are made to protect privacy. Others involve how to be fair toward rare disease patients eager to participate in research, which include determining where to run trials and provide fair access. Finally, determining the scope of post-trial obligations toward rare disease populations, and what is owed to rare disease patients who appear to benefit from therapies that lack efficacy in wider populations, can be complicated. How should sponsors and other research stakeholders think about these issues?      

This meeting is open to sponsors of the Bioethics Collaborative. For more information about the Bioethics Collaborative and how to become a sponsor, click here.