FDA Seeks Advice on Building Rare Disease Trial Network
Sponsors, investigators and patient advocates have a chance to weigh in on the FDA’s development of a network to support the conduct of clinical trials in rare disease populations.
The agency is gathering information and advice on how to start, implement and sustain a global rare disease network, asking the public a series of questions, including:
- What should be the immediate and long-term objectives?
- What kind of investigator experience is needed to implement the network?
- What are successful models of governance?
- What level of funding is needed?
- What are key milestones and timelines?
Commenters do not need to address all of the questions, according to the FDA notice issued today. Comments are due by July 30.
The effort is part of the FDA’s Rare Disease Cures Accelerator program, which seeks to “facilitate 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.” The agency already has begun work on two other phases of the program: development of a data analytics platform, and the creation of standardized sets of patient-focused clinical outcome assessments and endpoints for specific disease indications.
To read the notice, click here: https://bit.ly/2TSHmgh.