[ALS Association](https://www.als.org)

September 30, 2023 | December 31, 2025

No direct funding; program provides FDA collaboration and regulatory guidance for selected proposals.

Supports the development of novel efficacy endpoints for rare disease drug development through FDA collaboration and expert guidance.

--- ## Description The Rare Disease Endpoint Advancement (RDEA) Pilot Program is a collaborative initiative led by the FDA's Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER), in partnership with the ALS Association. The program is designed to advance the development of novel efficacy endpoints for drugs treating rare diseases by facilitating close collaboration between sponsors and the FDA throughout the endpoint development process. The RDEA program aims to foster innovation, share learnings on novel endpoint development, and build regulatory capacity to accelerate the availability of effective therapies for rare diseases. --- ## Due Dates - September 30, 2023: End of initial submission window (Q4 FY2023) - December 31, 2025: Next quarterly submission deadline (end of Q1 FY2026) Proposals are accepted quarterly, with the next deadline on December 31, 2025. --- ## Funding Amount - The RDEA Pilot Program does **not** provide direct research funding or grants. - Selected proposals receive regulatory guidance, structured collaboration with FDA experts, and up to four meetings (one initial and up to three follow-ups) to advance novel endpoint development. --- ## Eligibility - Sponsors must have an active pre-IND or IND for a rare disease. - Exception: Sponsors with or initiating a natural history study where the proposed endpoint will be studied are also eligible. - The proposed endpoint must be a **novel efficacy endpoint** intended to establish substantial evidence of effectiveness for a rare disease treatment. - "Novel" means never used to support drug approval or substantially modified from prior use. - In select cases, proposals for common diseases may be considered if the novel endpoint or methodology could be applicable to rare diseases, with sufficient justification. - Preference is given to proposals with broader impact potential, innovative approaches, and those relevant to multiple diseases or types of endpoints. --- ## Application Process - Submit an RDEA program proposal request by the end of the fiscal quarter (next deadline: December 31, 2025). - Only one RDEA proposal will be accepted per quarter, with a maximum of three per year. - Proposals must follow FDA-specified content and formatting requirements. - See [RDEA Proposal Content and Format](https://www.fda.gov/drugs/development-resources/content-and-format-rdea-proposal-rare-disease-endpoint-advancement-pilot-program) - FDA will notify applicants of selection decisions within 60 days after the quarter's end. - Upon selection, an initial meeting is scheduled within 45 days of receiving a complete meeting package; up to three follow-up meetings may be requested. --- ## Additional Information - Participation in the RDEA program does **not** constitute a special protocol assessment or guarantee regulatory approval. - Proposals involving surrogate endpoints are encouraged to have previously participated in a Type C Surrogate Endpoint meeting. - Public disclosure agreements are required prior to formal meetings. --- ## External Links - [RDEA Pilot Program Overview (FDA)](https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program) - [Submission Deadlines and Process](https://www.fda.gov/drugs/development-resources/submission-deadlines-and-process-rare-disease-endpoint-advancement-pilot-program) - [Content and Format of the RDEA Proposal](https://www.fda.gov/drugs/development-resources/content-and-format-rdea-proposal-rare-disease-endpoint-advancement-pilot-program) - [ALS Association Research Funding Opportunities](https://www.als.org/research/researchers/research-funding-opportunities) --- ## Contact Information | Contact | Email Address | Phone | More Info | |------------------------|-------------------------------------|------------------|------------------------------------------------| | RDEA Program (FDA) | [RDEA.Meetings@fda.hhs.gov](mailto:RDEA.Meetings@fda.hhs.gov) | | [FDA RDEA Program Page](https://www.fda.gov/drugs/development-resources/rare-disease-endpoint-advancement-pilot-program) | | ALS Association | [alsinfo@als.org](mailto:alsinfo@als.org) | +1 800-782-4747 | [ALS Association Contact](https://www.als.org/contact-us) |

Rare Disease Endpoint Advancement Pilot Program - ALS Association

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