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# Pilot Seeks to Advance Rare Disease Drugs
- URL: https://www.fdaweb.com/pilot-seeks-to-advance-rare-disease-drugs/
- Published: 2023-09-29T12:00:00.000Z
- Updated: 2026-09-14T18:59:15.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5155456

FDA has announced a new pilot program to “further accelerate the development of novel drug and biological products for rare diseases.” A limited number of selected participants will test the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program and its ability to offer frequent advice and regular ad-hoc communication with FDA staff to address product-specific development issues, including clinical study design, choice of 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,” CBER director **Peter Marks** is quoted in a [release](https://lnks.gd/l/eyJhbGciOiJIUzI1NiJ9.eyJidWxsZXRpbl9saW5rX2lkIjoxMDAsInVyaSI6ImJwMjpjbGljayIsInVybCI6Imh0dHBzOi8vd3d3LmZkYS5nb3YvbmV3cy1ldmVudHMvcHJlc3MtYW5ub3VuY2VtZW50cy9mZGEtbGF1bmNoZXMtcGlsb3QtcHJvZ3JhbS1oZWxwLWZ1cnRoZXItYWNjZWxlcmF0ZS1kZXZlbG9wbWVudC1yYXJlLWRpc2Vhc2UtdGhlcmFwaWVzP3V0bV9tZWRpdW09ZW1haWwmdXRtX3NvdXJjZT1nb3ZkZWxpdmVyeSIsImJ1bGxldGluX2lkIjoiMjAyMzA5MjkuODMzNzUxMDEifQ.i23UIK7b4yPG1hJWd-sPabnvT2EiZJkvSYpV0j06kQg/s/1255931999/br/227156495578-l?ref=fdaweb.com) as saying. “These are complex products and we recognize the importance of sponsor communication with the FDA to facilitate development of products for patients with unmet medical needs.”

The program will be open to sponsors who have an active IND involving products currently in clinical trials that are regulated by the CDER/CBER. “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,” the agency says. “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 this [*Federal Register* Notice](https://lnks.gd/l/eyJhbGciOiJIUzI1NiJ9.eyJidWxsZXRpbl9saW5rX2lkIjoxMDIsInVyaSI6ImJwMjpjbGljayIsInVybCI6Imh0dHBzOi8vd3d3LmZlZGVyYWxyZWdpc3Rlci5nb3YvcHVibGljLWluc3BlY3Rpb24vMjAyMy0yMTIzNS9zdXBwb3J0LWZvci1jbGluaWNhbC10cmlhbHMtYWR2YW5jaW5nLXJhcmUtZGlzZWFzZS10aGVyYXBldXRpY3MtcGlsb3QtcHJvZ3JhbT91dG1fbWVkaXVtPWVtYWlsJnV0bV9zb3VyY2U9Z292ZGVsaXZlcnkiLCJidWxsZXRpbl9pZCI6IjIwMjMwOTI5LjgzMzY4NjAxIn0.%5FKKaBzO6hIixhQQFaKEQX3GvSTxdxwURwmXWa%5Fyh624/s/1255931999/br/227108257272-l?ref=fdaweb.com). 

FDA says it will be accepting pilot applications between 1/2/2024 and 3/1/2024, and “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). Up to three participants for each Center will be selected, but after an internal evaluation, the agency may consider a pilot extension to allow more participants.

“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,” FDA says.