On May 31, 2024, the FDA announced initiation of the Support for Clinical Trials Advancing Rare Disease Therapeutics ("START") pilot program. The START program, led by the Center for Biologics Evaluation and Research ("CBER") and Center for Drug Evaluation and Research ("CDER"), was designed to "help further accelerate the development of novel drug and biological products for treatment of rare diseases." Selected participants of the program will have the opportunity to obtain increased communication with FDA officials and receive advice for addressing their program-specific needs, including clinical study design, choice of control group, fine-tuning the choice of patient population, leveraging nonclinical information, and product characterization.
The eligibility criteria for participation in the START program is set forth below, and was included in a notice published in the Federal Register on October 2, 2023:
- Joint CBER and CDER Eligibility Criteria:
- "For both CBER and CDER, the program is open to sponsors of products currently in clinical trials under an active investigational new drug application (IND) and IND has been submitted in or converted to Electronic Common Technical Document (eCTD) format, unless the IND is of a type granted a waiver from eCTD format."
- For both CBER and CDER, the program is open to sponsors who have demonstrated substantial effort to ensure that Chemistry, Manufacturing, and Controls (CMC) development aligns with clinical development."
- CBER-Specific Eligibility Criteria: "For CBER, eligible products must be a gene or cellular therapy intended to address an unmet medical need as a treatment for a serious rare disease or condition, which is likely to lead to significant disability or death within the first decade of life."
- CDER-Specific Eligibility Criteria: "For CDER, products must be intended to treat rare neurodegenerative conditions, including those of rare genetic metabolic etiology."
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