The U.S. Food and Drug Administration seeks to support innovation and quality in the drug development pipeline for rare diseases via a “Rare Disease Cures Accelerator.” Through this effort, the FDA is seeking to facilitate a cooperative research approach and common standardized platforms to better characterize diseases, incorporate the patient’s perspective in clinical outcome measurements, and build clinical trial readiness in the pre-competitive space. Since 2019, the FDA launched a series of initiatives to commence building capabilities in the areas of 1) a data analytics platform, and 2) a core set of patient-focused measures. Now the FDA requests information on practical steps and successful approaches to startup, implement, and sustain global clinical trial networks, including specific considerations for establishing such networks for a range of rare diseases. See the Federal Register for additional details on the requested information, including questions to the agency.
Why is the FDA doing this?
Well, although progress has been made over the past decade in regards to the planning and conducting of rare disease drug development, there is a long way to go. For example, with 7,000 known rare diseases less than 10% of them have an FDA-approved treatment available. This area of drug development continues to represent challenges, including small numbers of patients and limited understanding of the variability and progression of associated diseases.
The Rare Disease Cures Accelerator
The FDA proposes a Rare Disease Cures Accelerator facilitating a cooperative approach and common standardized platforms to improve characterization of rare diseases while incorporating the patient’s perspective in clinical outcome assessment measures while building clinical trial readiness earlier in the process.
Call to Action: Visit the FDA to review and submit written or electronic comments and information on the notice by July 31, 2020. See the link.