Orphan Products Grants Program

Objective: To support the clinical development of drugs, biologics, medical devices or medical foods for use in rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy.


Background: FDA OOPD provides grants for clinical studies on safety and/or effectiveness that will either result in, or substantially contribute to, market approval of these products. 


OOPD receives about 100 applications/year, and funds 10-15 new grants per fiscal year. Since 1983, FDA has provided more than $320 million for more than 530 grants for studies on rare diseases. The annual budget ≈ $14-15 million and greater than 50 FDA approved products were at least partially funded through the OOPD Grants Program.


Examples of products partially funded by OOPD grants approved for marketing:

·         Kalydeco (ivacaftor): Cystic fibrosis

·         Berlin Heart EXCOR Pediatric Ventricular Assist Device: Bridge to cardiac transplantation for         pediatric patients.

·         Xiaflex (collagenase): Dupuytren's disease

·         Folotyn (pralatrexate): Relapsed T-Cell non-Hodgkin’s lymphoma

·         Anascorp (Centruroides (Scorpion) Immune F(ab')2 (Equine) Injection): Envenomation by               poisonous scorpions in the US

·         Valchlor (mechlorethamine): Mycosis fungoides

·         Elaprase (idursulfase): Enzyme replacement therapy for patients with

·         MPS II (Hunter Syndrome).


·         Clinical study of an orphan disease or condition

·         A study must advance info towards a market approval

·         Must have active IND/IDE (not on clinical hold)

·         Compliance with Good Clinical Practices (GCP)

·         Human Subjects Assurance from OHRP (Office of Human Research Protections) “Federal-Wide        Assurance or FWA” (www.hhs.gov/ohrp)

·         IRB approval

·         Evidence that drug product is sufficiently available

Key Dates:

1.      The application due dates are February 1, 2017; and February 7, 2018.

2.      The resubmission due dates are October 16, 2017; and October 15, 2018.

3.      The anticipated start dates are November 2017; and November 2018.

4.      The opening date is December 4, 2014.

5.      The expiration dates are February 8, 2018, and October 16, 2018, (resubmission).

Application Process:

·         Step 1: Obtain a Dun and Bradstreet (DUNS) Number

·         Step 2: Register with System for Award Management (SAM) (formerly Central Contractor               Registration (CCR)) Show citation box

·         Step 3: Obtain Username & Password on Grants.gov

·         Step 4: Authorized Organization Representative (AOR) Authorization

·         Step 5: Track AOR Status

·         Step 6: Register with Electronic Research Administration (eRA) Commons


Application Materials:

Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01; RFA-FD-16-043) and Clinical Studies of Safety and Effectiveness of Orphan Products Research Project Grant (R01; RFA-FD-15-001) available at grants.gov



OOPD Website

Erica K. McNeilly Presentation to the Joint EMA/FDA/MHLW-PMDA Workshop