The Food and Drug Administration (FDA) issued a request for information (RFI) on rare disease clinical trials (press release). The FDA notes that of the 7,000 known rare diseases, less than 10 percent have an FDA-approved treatment available, and rare disease drug development continues to be challenged by the small number of patients and limited understanding of each disease.
The FDA details that it has proposed the establishment of a “Rare Disease Cures Accelerator” to support innovation and quality in the drug development pipeline for rare diseases. The Rare Disease Cures Accelerator would provide a more centralized infrastructure to support: 1) rare disease characterization, 2) development of standard core sets of clinical outcome assessments and endpoints relevant to rare conditions; and 3) support conduct of clinical trials in rare disease populations. The FDA has begun building capabilities for the first two components using fiscal year (FY) 2019 appropriations.
The FDA is interested in understanding what work is currently being done to address the third component of the Rare Diseases Cures Accelerator – improving the design, conduct, and completion of rare disease clinical trials. The FDA notes it is interested in learning practical steps and successful approaches related to startup, implementation, and sustainment of clinical trials networks for rare diseases. Additionally, the agency is specifically interested in responses to the following questions:
- What should be the immediate and long-term objectives of a global clinical trials network;
- How could a global clinical trials network for rare diseases be organizationally structured;
- What kind of investigator experience is needed to start up, expand, and implement a global clinical trials network;
- What are successful models of governance for global clinical trials networks;
- What are potential opportunities to leverage or complement other existing networks;
- What infrastructure is required to startup, implement, and sustain a global clinical trials network;
- What level of funding would be needed to establish a network, potentially expand a network, and sustain a network over the long term;
- What are the key milestones and associated timelines for starting up and expanding to implement a global clinical trials network; and
- What are potential challenges or barriers to starting up, implementing, and sustaining a global rare disease clinical trials network?
Submissions are due July 31.