Rare Disease Innovation, Science, and Exploration (RISE) Workshop Series

Overview

With many rare diseases lacking approved therapies these patient populations face unique challenges to medical product development, including small patient populations, heterogeneity in disease presentation and progression, and limited natural history. To address this issue, the Duke-Margolis Institute for Health Policy, under a cooperative agreement with the U.S. FDA, is co-convening the Rare Disease Innovation, Science, and Exploration (RISE) Workshop series with the Rare Disease Innovation Hub. The RISE public workshops bring together medical product developers, patients, academic researchers, regulators, clinicians, and patient organizations to discuss critical challenges and opportunities in advancing the ecosystem for medical products to treat rare diseases. Workshop topics focus on cross-cutting issues and are informed by submissions to the public docket.

Workshops

On the RISE: Controls in Rare Disease Clinical Trials for Small and Diminishing Populations

Individualized Therapies on the RISE

RISE Together: Data Sharing Across the Rare Disease Ecosystem

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.