Resources for Applicants and Grantees
FDA Office of Orphan Products Development (OOPD) rare disease research grants
The FDA’s rare disease grants programs offer funding opportunities for rare disease research. Applicants may find the resources below useful when preparing to apply for an orphan product grant and grantees will find information below on what to expect and what is required after an award is made.
On this page:
- Key Information
- Educational Webinars and Videos
- Other Useful Information
- Historical Requests for Applications and Funding Announcements
Key Information
- Current Funding Opportunities
- Orphan Products Grants Program
- Rare Neurodegenerative Disease Grants Program
- Frequently Asked Questions for Orphan Products Grant Applicants
- Scientific Publications
- Contact Information
Educational Webinars and Videos
- Orphan Products Grants Overview
- Video Discussion on Natural History Studies
- Natural History Studies and Registries in the Development of Rare Disease Treatments Hybrid Public Workshop (May 2024, recording)
- How Rare Disease Patients Can Move Scientific Discovery Forward
- Natural History Video for Grant Applicants
- Patient Affairs Staff: Patients Matter Video Series
- Overview of Sponsor-Investigator Roles and Responsibilities in Clinical Investigations for Drugs and Biologics Orphan Products Grantees
- This presentation is an overview of the requirements and recommendations for Orphan Drug Grantees who conduct clinical investigations with an orphan drug or biologic product. The information is meant to serve as an introduction to the roles and responsibilities of personnel involved in conducting a clinical trial with orphan drugs. This overview is not intended to be all-inclusive or a substitute for formal training in good clinical practice.
- Please use the slide deck to access all the links in the training presentation.
- Test your knowledge by using the self-assessment quiz and answers.
Other Useful Information
- Application Guide: How to Apply
- FDA’s Role: ClinicalTrials.gov Information
- Study Data Standards Resources
IND
- Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings
- IND Application Information and Procedures
- Information for Sponsor-Investigators Submitting Investigational New Drug Applications (INDs)
- Small Business and Industry Assistance: Frequently Asked Questions on the Pre-Investigational New Drug (IND) Meeting
Related Guidances
- E6(R2) Good Clinical Practice: Integrated Addendum to ICH E6(R1)
- Good Clinical Practice
- Rare Diseases: Common Issues in Drug Development
- Rare Diseases: Early Drug Development and the Role of Pre-IND Meetings
- Rare Diseases: Natural History Studies for Drug Development
- Interacting with the FDA on Complex Innovative Trial Designs for Drugs and Biological Products
- Adaptive Design Clinical Trials for Drugs and Biologics Guidance for Industry
General FDA Center Information
- CDER: About the Center for Drug Evaluation and Research
- CBER: About the Center for Biologics Evaluation and Research
- CDRH: About the Center for Devices and Radiological Health
- CFSAN: About the Center for Food Safety and Applied Nutrition
FDA Center Guidances for Industry
- CBER: Guidance, Compliance & Regulatory Information (Biologics)
- CDER: Guidances (Drugs)
- CDRH: Guidance Documents (Medical Devices and Radiation-Emitting Products
- RFA-FD-24-024: Natural History, Clinical Outcome Assessment, and Biomarker Studies of Rare Neurodegenerative Diseases (U01) Clinical Trials Optional
- RFA-FD-23-030: Systematic Review of Clinical Outcome Assessments (COAs) for Communication Brain-Computer Interface Devices (cBCIs) in Amyotrophic Lateral Sclerosis (ALS) (UH2/UH3) Clinical Trials Not Allowed (nih.gov)
- RFA-FD-23-028: Natural History and Biomarker Studies of Rare Neurodegenerative Diseases (U01) Clinical Trials Optional (nih.gov)
- RFA-FD-23-001: Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)
- RFA-FD-22-001: Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases (R01) Clinical Trials Not Required
- RFA-FD-21-001: Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01) Clinical Trials Required (nih.gov)
- RFA-FD-20-001: Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01) (nih.gov)
- RFA-FD-19-001: Natural History Studies Addressing Unmet Needs of Rare Diseases: Orphan Products Research Project Grant (R01) (nih.gov)
- NOT-FD-18-006: Notice of Cancellation of the October 2018 Receipt Date for RFA-FD-16-043 (nih.gov)
- RFA-FD-16-043: Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01) (nih.gov)
- RFA-FD-15-001: Clinical Studies of Safety and Effectiveness of Orphan Products Research Project Grant (R01) (nih.gov)
- RFA-FD-11-001: Clinical Studies of Safety and Effectiveness of Orphan Products Research Project Grant (R01) (nih.gov)
- RFA-FD-09-001: Clinical Studies of Safety and Effectiveness of Orphan Products Research Project Grant (R01) (nih.gov)
October 2024
September 2023
- FDA awards 10 new clinical trial studies [ARCHIVED], funded by Congress through the Orphan Products Grants Program, to support the development of medical products for rare diseases
October 2022
October 2021
- FDA Awards 11 Grants to Clinical Trials to Develop New Medical Products for Rare Disease Treatments [ARCHIVED]
October 2020
October 2019
- FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases - October 8, 2019 [ARCHIVED]
- FDA awards 2 grants for natural history studies in rare diseases - October 8, 2019 [ARCHIVED]
October 2017
- FDA awards 6 grants for natural history studies in rare diseases - October 6, 2017 [ARCHIVED]
- FDA awards 15 grants for clinical trials to stimulate product development for rare diseases - October 6, 2017 [ARCHIVED]
October 2016
September 2015
- FDA Awards 18 grants in fiscal year 2015 to Stimulate Drug and Device Development for Rare Diseases [ARCHIVED]
October 2013
September 2013
2012
- Fiscal Year 2012 OPD New Awards (PDF, 21 KB) [ARCHIVED]