Data Sharing to Accelerate Therapeutic Development for Rare Diseases: Day 1 & 2


FDA Convening

Data Sharing to Accelerate Therapeutic Development for Rare Diseases: Day 1 & 2


Progress in rare disease drug development is obstructed by gaps in disease characterization, limited understanding of disease natural history and, the small number of people affected by each individual rare disease. Approximately 7,000 rare diseases have been identified and, cumulatively, rare diseases impact 1 in 10 people across the globe. However, FDA-approved treatments are available for less than 10% of rare diseases, presenting significant unmet medical need. The ability to improve basic understanding of disease progression and variability for each rare disease depends on the use of rare disease patient registries and other shared data resources. Shared data platforms and analytics are also key to supporting translational research, innovative trial design, and the efficient development of targeted therapeutics for rare disease patients.

Accordingly, the Duke-Margolis Center for Health Policy, through a cooperative agreement with the U.S. Food & Drug Administration, is convening a public meeting to discuss the importance of data sharing for accelerated therapeutic development in rare disease as well as considerations for sustaining and leveraging a robust data sharing infrastructure to support innovative rare disease trial design. The meeting will explore several topics including:

  • The importance of shared data to support innovation and quality in the drug development pipeline for rare diseases
  • Approaches and challenges to ensuring data quality, standardization, and interoperability in shared data resources
  • The role of regulatory agencies in strengthening rare disease data collection and shared data resources 

Please register above for listen-only access to the 2-day event. Registrants are encouraged to submit questions during the meeting to, and meeting moderators will work them into open discussion. Please feel free to tweet about meeting content and progress by tagging @DukeMargolis on Twitter and using the hashtag #RareDataShare.

Funding for this workshop was made possible in part by a cooperative agreement from the U.S. Food and Drug Administration Center for Drug Evaluation and Research. The views expressed in written workshop materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services nor does mention of trade names, commercial practices, or organizations imply endorsements by the U.S. Government.