Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01 Clinical Trials Required)

About this grant

The purpose of this opportunity is to fund clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions. Additionally, through the funding of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of approved treatments for rare diseases and exert a broad and positive impact on rare disease drug development. Grant Number: FOR-FD-25-020.

What the funding covers

• See official listing for award details

How to apply

1. 1Check eligibility: Confirm your organization type (e.g., nonprofit, state government), project alignment, and deadlines from the opportunity synopsis.
2. 2Register: Create a Grants.gov account via Login.gov, complete SAM.gov and UEI registration, and authorize organization representatives (takes 2-4 weeks).
3. 3Prepare application: Select "Apply" to open Workspace, download forms (e.g., SF-424), complete offline/online with validation checks, and upload supporting docs like budgets and narratives.
4. 4Submit and track: Check package for errors, sign/submit via Authorized Organization Representative (AOR), and monitor status with your tracking number
5. 5Applications must be submitted through grants.gov
6. 6Grant Number: FOR-FD-25-020
7. 7Posted: 2025-07-11

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