
The U.S. FDA has launched the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program to speed up the development of therapies for rare diseases.
The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products and Center for Drug Evaluation and Research (CDER) Office of New Drugs will invite selected organizations with rare disease therapy development programs to take part in the program.
The idea of the pilot is to add to and improve current communications between therapeutic product developers and the FDA to improve and speed up the approval process. Currently, meetings between product developers and the FDA during drug development are infrequent and formal, which can lead to development delays if important questions are not answered soon enough.
“We hope the insight gained from this pilot will provide information on how best to facilitate more efficient development of potentially life-saving therapies with rare disease indications and help sponsors generate high-quality, compelling data to support a future marketing application,” said Peter Marks, director of the FDA’s CBER, in a press statement.
“These are complex products, and we recognize the importance of sponsor communication with the FDA to facilitate development of products for patients with unmet medical needs.”
Applications to take part in the pilot will be open during January and February 2024 and will be open to developers of products in clinical trials that have an active investigational new drug (IND) application with either the CBER or CDER and have the ability to move their product forward towards market approval. The two FDA centers will each accept up to three developers and their products to take part in the pilot program.
As well as an active IND, CBER-regulated products need to be “a gene or cellular therapy intended to address an unmet medical need as a treatment for a rare disease or serious condition, which is likely to lead to significant disability or death within the first decade of life.” If regulated by the CDER, the product should be intended “to treat rare neurodegenerative conditions, including those of rare genetic metabolic type.”
Those accepted into the program will receive frequent advice and communication from the FDA to help iron out any issues such as those involving clinical study design, choice of control group and choice of patient population, as well as many others.
Conclusion of the program for each developer will coincide with milestones such as initiating a late-stage trial, or getting to the stage of submitting an application for final market approval.
“We look forward to increased engagement with sponsors developing these important products for the rare disease community,” said Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research.
“We share the goal of delivering potentially life-saving products to patients, and are committed to helping sponsors achieve regulatory milestones, while ensuring the safety, effectiveness and quality of these products.”
More information on the program and its eligibility requirements for potential applicants is published in the Federal Register.