FDA Establishes Pilot Program to Accelerate Rare Disease Therapy Development

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The program will allow sponsors of certain CBER and/or CDER-regulated products more frequent communication with FDA staff.

On Sept 29, 2023, FDA announced a pilot program, Support for clinical Trials Advancing Rare disease Therapeutics (START), intended to support development of novel drug and biological products for rare diseases. The agency is establishing a pilot program for a limited number of sponsors, intended to allow for more regular communication with FDA on addressing clinical development issues. Selected participants will have access to FDA advice and pertinent communication for product-specific development issues such as choice of control group, clinical study design, and fine-tuning the choice of patient population.

To be eligible for the program, participants must be sponsors of products in clinical trials under an active Investigational New Drug application (IND) and regulated by the Center for Biologics Evaluation and Research (CBER) and/or the Center for Drug Evaluation and Research (CDER). The requirements for eligibility for the program differ between CBER and CDER-regulated products. CBER-regulated products must be a gene or cellular therapy being developed as treatment for a currently untreatable rare or serious condition that can lead to significant or death within the first 10 years of life. CDER requires that products be intended to treat rare neurodegenerative conditions, such as those of rare genetic metabolic type.

Applications for the START program will be open between Jan. 2, 2024, and March 1, 2023. Sponsors who show the ability to push their development program forward towards a marketing application will be more likely to be selected. FDA will choose up to three participants for each center. After an appraisal of this pilot program and feedback from participating sponsors, FDA may announce a second iteration, which will be announced at a later date. Participation will finish when the development program has reached a notable regulatory landmark, such as reaching the stage before submitting a marketing application or launch of the pivotal clinical study stage.


The START program is not the only FDA resource available to sponsors of therapies for rare diseases. FDA recently published a Request for Information, intended for stakeholders to give feedback on challenges and opportunities relating to cellular and gene therapies intended to treat rare diseases. It also published a docket for stakeholder feedback under the CEDER Accelerating Rare disease program. This docket will allow FDA to find knowledge gaps in rare disease drug development, and develop public resources on regulatory issues surrounding clinical trial design.

Source: FDA