The U.S. Food and Drug Administration 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. More information on the program’s eligibility requirements can be found in the Federal Register Notice.
The FDA will be accepting applications to the START program between Jan. 2, 2024, and March 1, 2024. Pilot participants will be selected based on application readiness (e.g., sponsors who demonstrate having the ability to move their development program forward towards a marketing application). The agency will select up to three participants for each center. 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.
Sponsor participation in the pilot will be considered concluded when the development program has reached a significant regulatory milestone, such as initiation of the pivotal clinical study stage or reaching the stage prior to submitting a marketing application (pre-Biologics License Application or pre-New Drug Application meeting stage), as agreed upon with the sponsor.