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FDA is taking steps to help further accelerate the development of novel drug and biological products for rare diseases. The agency is announcing the opportunity for a limited number of sponsors to participate in a pilot program allowing for more frequent communication with FDA staff to provide a mechanism for addressing clinical development issues.
Selected participants of the Support for Clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program will be able to obtain frequent advice and regular ad-hoc communication with FDA staff to address product-specific development issues, including, but not limited to, clinical study design, choice of control group and fine-tuning the choice of patient population.
The 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) and/or the Center for Drug Evaluation and Research (CDER). Eligibility criteria for the pilot differs between CBER and CDER-regulated products.
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.
FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies. (2023, September 29). Cision PR Newswire.