RISE Workshop 3 - RISE Together: Data Sharing Across the Rare Disease Ecosystem

The Rare Disease Innovation Hub’s third RISE Workshop, entitled RISE Together: Data Sharing Across the Rare Disease Ecosystem, took place on March 30, 2026. The workshop explored data sharing as it pertains to informing development and regulatory review for rare disease therapies. The small patient populations and often heterogeneous nature of rare diseases result 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 focused 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 discussed: 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 potential ways that FDA might support data sharing within the bounds of its authorities.

Meeting materials and a full recording of the workshop are available here.