The FDA’s Orphan Products Grants Program offers 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.
See our current funding opportunities including instructions and helpful hints for applying for each grant as well as FAQs for applicants.
- Orphan Products Grants Overview
- Video Discussion on Natural History Studies
- Workshop on Natural History Studies in Rare Diseases: Meeting the Needs of Drug Development and Research
- How Rare Disease Patients Can Move Scientific Discovery Forward
- Natural History Video for Grant Applicants
- Patient Affairs Staff: Patients Matter Video Series
- Good Clinical Practice Training
- 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.
- Application Guide: How to Apply
- FDA’s Role: ClinicalTrials.gov Information
- Study Data Standards Resources
- 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
- 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