FDA Seeks Comments on Rare Disease Clinical Trials Network

Rare Daily Staff

The U.S. Food and Drug Administration is seeking comments about how it should best establish and sustain global clinical trial networks for a range of rare diseases.

The call follows efforts by the agency to establish a Rare Disease Cures Accelerator to support innovation and quality in the drug development pipeline for rare diseases.

Rare disease therapies for the first time in 2018 represented the majority of new molecular entities approved by the agency, but less than 10 percent of the estimated 7,000 known rare diseases having an approved therapy available. The FDA is hoping to address challenges of rare disease drug development, such as the small patient populations and heterogeneity of rare diseases, through a cooperative approach and common standardized platforms to better characterize rare diseases, incorporate the patient’s perspective in clinical outcome assessment measures, and build clinical trial readiness in the pre-competitive space.

In September 2019, it began building capabilities including a data analytics platform to promote the secure sharing of existing patient-level data and encourage the standardization of new data collection. It also began a pilot grant program to develop standard core sets of clinical outcome assessments and endpoints for specific disease indications as part of the agency’s patient-focused drug development efforts.

The agency is seeking comments from patients, patient advocates, researchers, health professions, industry, and others about the long-term objectives of a global clinical trials network, how it should be structured and governed, opportunities to leverage, and infrastructure and funding requirements. It would also like to hear thoughts on milestones and timelines, as well as potential challenges and barriers to consider.

Comments are due by July 31. Additional information including questions that could be addressed can be found online at the Federal Register notice.