HQ Team
October 4, 2023: The FDA is planning a pilot program to accelerate the development of drugs and biological products for rare diseases by helping companies gather relevant data and conduct trials.
The agency announced the opportunity for a limited number of sponsors to participate in the program to allow more frequent communication with FDA staff.
This would provide a mechanism for addressing clinical development issues, according to an FDA statement.
The FDA will start accepting applications to the program between Jan. 2, 2024, and March 1, 2024. Pilot participants will be selected based on application readiness. The agency will select up to three participants for each centre.
Second iteration
Following an evaluation of this pilot and feedback from selected sponsors, the agency may consider a second iteration, which would be announced in the Federal Register at a later date, according to the statement.
Selected participants of the Support for Clinical Trials Advancing Rare Disease Therapeutics Pilot Program will be able to obtain frequent advice and regular ad-hoc communication with FDA staff.
They would be in a position to address product-specific development issues, including clinical study design, choice of a control group and fine-tuning the choice of patient population.
“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, M.D., PhD, director of the FDA’s Center for Biologics Evaluation and Research.
‘Unmet medical needs’
“These are complex products and we recognise the importance of sponsor communication with the FDA to facilitate the development of products for patients with unmet medical needs.”
The START program will be open to sponsors of products currently in clinical trials under an active Investigational New Drug application (IND), regulated by the Center for Biologics Evaluation and Research (CBER) or the Center for Drug Evaluation and Research (CDER).
In addition to having an active IND, eligible CBER-regulated products must 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.
Under CDER’s eligibility criteria, the product must be intended to treat rare neurodegenerative conditions, including those of rare genetic metabolic type.
‘Life-saving potential’
“We look forward to increased engagement with sponsors developing these important products for the rare disease community,” said Patrizia Cavazzoni, MD, 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.”
