Session 2 - RISE Together: Data Sharing Across the Rare Disease Ecosystem

Session 2: Ethical Considerations for Sharing Data Objective: This presentation will address the ethical considerations of data sharing for rare disease product development. The presenter will share insights on who should control the use of the data, informed consent, and privacy. Moderator: Gerrit Hamre, Duke-Margolis Institute for Health Policy Presentation: Kayte Spector-Bagdady, University of Michigan Medical School This public workshop, co-convened by the Duke-Margolis Institute for Health Policy and the U.S. Food and Drug Administration (FDA) Rare Disease Innovation Hub, is designed for all stakeholders in the rare disease community to explore data sharing as it pertains to informing development and regulatory review for rare disease therapies. The small patient populations and often heterogenous nature of rare diseases results in a paucity of data, further intensified when multiple sponsors are working to develop medical products for the same disease state. As a result, opportunities for rigorous, high quality data collection would have a significant impact in this space. There may be opportunities for the rare disease community to support and encourage broader access through the sharing of existing data to inform certain areas of rare disease medical product development including disease progression modeling, endpoint selection, inclusion/exclusion criteria, and safety. Normalizing data sharing could help to inform clinical trial protocols, safety monitoring, and risk-benefit assessments throughout the development and post-marketing processes. The workshop focuses on clarifying possible avenues for data sharing and the types of data that can be shared (e.g., safety information, real-world evidence, and deidentified patient data). The workshop also discusses: the promotion of data sharing practices and structures for facilitating data sharing among rare disease medical product development stakeholders; examples of the impact of data sharing in regulatory submissions; a priori considerations for the collection and sharing of high quality data; some of the logistical and legal challenges encountered in data sharing; and whether there are ways that FDA might support data sharing, within the bounds of its authority. This project is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award U19FD006602 totaling $5,192,495 with 100 percent funded by FDA/HHS. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government.