Clinical Trials Grants Program (Rare Diseases)

Clinical Trials Grants Program (Rare Diseases) is sponsored by U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD). This program funds clinical trials evaluating efficacy and/or safety in support of a new indication or change in labeling to address unmet needs in rare diseases or conditions. Research on treatments for beta-ketothiolase deficiency would be eligible.

Orphan Products Grants Awarded | FDA Skip to in this section menu Medical Products for Rare Diseases and Conditions Grant Programs to Support Development of Medical Products for Rare Diseases Orphan Products Grants Program Orphan Products Grants Awarded Orphan Products Grants Program Clinical Trials Grants Program Awards Clinical trial grants for orphan products are a proven method of successfully fostering and encouraging the development of new safe and effective medical products for rare diseases and conditions.

The FDA’s Office of Orphan Products Development (OOPD) awards new clinical trial grants annually. For more information, see Clinical Trials Grants Program . FDA has awarded hundreds of grants under this program since 1983.

The program has funded clinical trials that have facilitated the approval of more than 85 products including drugs, biologics, and devices for rare diseases. The most recent awards are listed below. For a full list of grants awarded since the program's inception, please visit our searchable list of funded grants .

Detailed instructions for searching the list Instructions below refer to the Orphan Product Grants Program database . During searches, if more than one field is populated, results of the search will display as if the word “and” was put between the search criteria. For example, searching 'interferon' as a product and 'Smith' as an investigator will return only matches where both criteria are met.

If you want all records that include either one or both criteria (i.e., an 'or' search), the searches must be run separately. A search with no criteria will return all grants, current and/or previous, depending on the button selected.

Disease Indication, Product Name, or Grant Title Entering a search term in the Disease Indication field or Grant Title field will find any occurrence of the term in either of these fields, i.e., both fields are searched automatically. The database does not use a standard terminology, and searching both fields will increase the possibility of finding the appropriate records.

For example, some records may have a disease name in the grant title that is listed differently in the indication. Searches on product name search only the Product Name field, therefore, if the search does not return expected records, the search should be repeated with the product name searched under the Grant Title field.

Principal Investigator, Institution, or Location Grants can be searched by the name of the Principal Investigator, Institution, or Location. For Location, enter a city, state, or country. States should be entered as two letter abbreviations, e.g., 'NJ'.

Entering only a location (e.g., 'NJ') will return all grants for investigators in New Jersey. For 'country', only countries outside the United States need to be entered. Results can be displayed as a list or downloaded as an Excel file.

If displayed as a list, the number of records specifies how many records are returned on each page displayed. The default is 100 records per page. Output can also be sorted by several options, e.g., investigator or date.

Note: If you need help with accessing information in different file formats, see Instructions for Downloading Viewers and Players . FY 2025 Clinical Trials Program Grants Awarded In FY 2025, FDA received 58 grant applications and awarded 3 new clinical trials that will help to accelerate the development of safe and effective treatments to address the significant and often unmet needs of those affected by rare diseases.

The new awards will provide more than $6 million to clinical researchers over the next four years to advance the development of medical products for rare diseases. This includes additional funding to support innovative and efficient trial designs that can be used to advance treatments through product development and as models for future drug development in rare diseases.

Reference: Request for Applications (RFA FD-23-001) Awardee Principal Investigator Description of Project Amount (approximate) Duration Icahn School of Medicine at Mount Sinai (New York, NY) Desnick, Robert Phase 1/2 Study of Oral Ciclopirox for the Treatment of Congenital Erythropoietic Porphyria $2.

6 million 4 years Research Institute at Nationwide Children's Hospital (Columbus, OH) Fouladi, Maryam Phase 2 Molecularly-Guided Umbrella Study of Multiple Products Including Ribociclib and Everolimus for the Treatment of newly-diagnosed High Grade Gliomas $3.

2 million 4 years University of Pennsylvania (Philadelphia, PA) Barta, Stefan Phase 1 Study of CD5-deleted Chimeric Antigen Receptor Cells (Senza5 CART5) for the Treatment of T Cell Non-Hodgkin Lymphoma (NHL) $3. 4 million 4 years Natural History Studies Grants Program Awards FDA has funded natural history studies since 2016 to help address significant unmet medical needs for patients with rare diseases.

For more information, see: Natural History Studies Grants Program . FY 2024 Natural History Studies Grants Awarded In FY 2024, FDA received 53 natural history grant applications and funded three new grants totaling more than $4. 7 million spread over four years to continue to advance the understanding of rare diseases.

This includes one award made with additional support from the FDA Center for Drug Evaluation and Research (CDER) Accelerating Rare disease Cures (ARC) program. These studies will address knowledge gaps in support of future clinical trials.

Specifically, one study will utilize a collaborative network and novel machine learning techniques to assess patient-reported outcomes and stratify patients with pediatric intestinal failure (PIF) (the inability of the gastrointestinal tract to sustain life without supplemental nutrition or intestinal transplant).

This has the potential to advance clinical trial design as there is currently no consensus on disease stratification or specific outcome measures in PIF.

Another study will use machine learning to create a patient network and identify clinical outcomes and prognostic risk factors in children with autosomal recessive polycystic kidney disease (ARPKD), which will have the potential to advance the clinical study of promising candidate therapies identified in animal models.

Another study will bring together international centers of excellence, patient advocacy groups, and industry partners currently developing interventions for congenital central hypoventilation syndrome (CCHS).

The study aims to define clinical, functional, and quality of life assessments to develop effective treatment strategies in this complex disease that includes a life-threatening inability to regulate breathing necessitating life-long ventilatory support.

Reference: Request for Applications (RFA-FD-22-001) Awardee Principal Investigator Description of Project Amount (approximate) Duration Children's Hospital of Philadelphia (Philadelphia, Pennsylvania) Erum Hartung Retrospective and prospective study in autosomal recessive polycystic kidney disease (ARPKD) to create PKDnet to inform future clinical trial design $1.

6 million 4 years Lurie Children's Hospital of Chicago (Chicago, Illinois) Debra Weese-Mayer Prospective study in congenital central hypoventilation syndrome to establish measures that correlate with disease severity and inform therapeutic development $1.

6 million 4 years University of Pittsburgh At Pittsburgh (Pittsburgh, Pennsylvania) Simon Horslen Prospective study in pediatric intestinal failure to inform natural history and develop outcome measures $1.

5 million 4 years Other Past and Ongoing OOPD-Supported Natural History Grants Awardee Principal Investigator (Last Name, First Name) Description of Project Location (State, City) Start Date (YYYY-MM-DD) End Date (YYYY-MM-DD) Children's Hospital Corp. Tan, Wen Hann Prospective Study in Angelman Syndrome Massachusetts, Boston 2017-09-15 2022-08-31 Children's Hospital of Philadelphia Lynch, David Prospective Study in Friedreich Ataxia Pennsylvania, Philadelphia 2017-09-01 2023-02-28 Cincinnati Children’s Hospital Medical Center Trapnell, Bruce Retrospective Study in Autoimmune Pulmonary Alveolar Proteinosis Ohio, Cincinnati 2022-09-01 2025-08-31 Columbia University Health Sciences Rubin, Mishaela Prospective Study in Hypoparathyroidism New York, New York 2022-09-01 2026-08-31 Columbia University Medical Center Cohen, Adi Prospective Study in Pregnancy & Lactation Associated with Osteoporosis New York, New York 2017-09-01 2023-08-31 Jaeb Center for Health Research, Inc. Ayala, Allison Prospective Study in Gyrate Atrophy Florida, Tampa 2022-09-15 2027-08-31 Johns Hopkins University Lederman, Howard Prospective Study in Ataxia-Telangiectasia Maryland, Baltimore 2022-09-01 2026-08-31 University of Iowa Gerke, Alicia Retrospective Study in Sarcoidosis Iowa, Iowa City 2017-09-01 2020-08-31 University of Minnesota Walk, David Retrospective and Prospective Study in Amyotrophic Lateral Sclerosis Minnesota, Minneapolis 2022-09-20 2026-08-31 University of Pennsylvania Fajgenbaum, David Prospective Study in Castleman disease Pennsylvania, Philadelphia 2022-09-01 2026-08-31 University of Tennessee Health Science Center Ataga, Kenneth Prospective Study in Sickle Cell Anemia Tennessee, Memphis 2017-09-01 2023-08-31 University of Texas MD Anderson Cancer Center Grubbs, Elizabeth Prospective Study in Medullary Thyroid Carcinoma Texas, Houston 2019-09-01 2024-07-31 Vanderbilt University Medical Center Brittain, Evan Prospective Study in Pulmonary Arterial Hypertension Tennessee, Nashville 2022-09-01 2026-08-31 Vanderbilt University Medical Center Soslow, Jonathan Prospective Study in Cardiac Disease in Duchenne Muscular Dystrophy (DMD) Tennessee, Nashville 2019-09-01 2025-02-28 Virginia Commonwealth University Johnson, Nicholas Prospective Study in Myotonic Dystrophy Type 1 to Determine Biomarkers in Clinical Endpoints Virginia, Richmond 2017-09-15 2027-02-28 Historical requests for applications and funding announcements Grants program contact information