Clinical Trial and Natural History Study Grants
Orphan Products Grants
OOPD administers two Orphan Products Grants:
Please view the program sections for more information.
Clinical Trials
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. A major portion of appropriated funds for a given fiscal year provides continued funding of ongoing awards. There are typically 60 to 85 ongoing grant projects every year. OOPD awards approximately five to twelve new grants each year, as resources allow. The rapid increase in the cost of clinical trials in recent years has precluded an increase in the number of new grants. OOPD conducts ongoing grant evaluations to ensure extramural funded studies maintain grant agreement terms and minimize risk to people participating in the clinical research.
FDA is focusing efforts to facilitate and advance new therapies in drug development in safe and efficient means by encouraging innovative clinical trial methods such as adaptive and seamless trial designs, modeling and simulations, and basket and umbrella trials. The use of shared, established infrastructure and resources and collaborative efforts among stakeholders in industry, academia and patient organizations also facilitates efficient product development. These methods are vital to efficient trials and data evaluation which can expedite drug development. Additionally, patients living with a rare disease and their caregivers have experiences and knowledge that contribute to important considerations in product development, such as with trial feasibility.
FY 2023 awards: Clinical trial grants
1. Albert Einstein College of Medicine (Bronx, New York); Aditi Shastri; Phase 1b/2 of danvatirsen monotherapy in combination with venetoclax for the treatment of relapsed/refractory MDS & AML ($2.6 million over four years)
2. Boston Children's Hospital (Boston, Massachusetts); Lynn A Sleeper; Phase 3 study of the Cardiohelp System (an extracorporeal membrane oxygenation circuit) for the treatment of children with severe cardiac failure (approximately $2.4 million over four years)
3. Extend Biosciences, Inc. (Newton, Massachusetts); Laura M Hales; Phase 2 study of EXT608 for the treatment of adults with hypoparathyroidism (approximately $3.6 million over four years)
4. Indiana University-Purdue University at Indianapolis (Indianapolis, Indiana); Sherif S Farag; Phase 1/2 study of sitagliptin, bortezomib and post-transplant cyclophosphamide for the prevention of acute GVHD following allogeneic hematopoietic stem cell transplantation (approximately $2.6 million over four years)
5. Massachusetts General Hospital (Boston, Massachusetts); Stephanie B Seminara; Phase 2 study of Kp-10 for the treatment of patients with idiopathic hypogonadotropic hypogonadism (approximately $2.6 million over four years)
6. Mayo Clinic Rochester (Rochester, Minnesota); Jann N Sarkaria; Phase 1 study of WSDO628 with radiation therapy for the treatment of recurrent high-grade glioma (approximately $2.5 million over four years)
7. New York University School of Medicine (New York, New York); Maria Alejandra Gonzalez-Duarte Briseno; Phase 2 study of dexmedetomidine sublingual film for the ambulatory treatment of hyperadrenergic autonomic crisis in patients with familial dysautonomia (approximately $2.6 million over four years)
8. New York Medical College (Valhalla, New York); Mitchell S Cairo; Phase 2 study of maternal donor derived CMV cytotoxic T lymphocytes and valganciclovir vs valganciclovir in neonates with moderate/severe maternal acquired cytomegalovirus infection (approximately $2.6 million over four years)
9. Ophirex, Inc. (Corte Madera, California); Tim Platts-Mills; Phase 2b study of varespladib for the treatment of snakebite envenoming (approximately $2.7 million over three years)
10. Sloan-Kettering Institute Cancer Research (New York, New York); Joseph H Oved; Phase 2 study of bridging pre-transplant inflammatory dampening (immune suppression) for treatment of bone marrow transplant in primary immune regulatory disorders (approximately $2.6 million over four years)
FY 2023 awards: Clinical trial grants
| 1. Albert Einstein College of Medicine (Bronx, New York); Aditi Shastri; Phase 1b/2 of danvatirsen monotherapy in combination with venetoclax for the treatment of relapsed/refractory MDS & AML ($2.6 million over four years) |
| 2. Boston Children's Hospital (Boston, Massachusetts); Lynn A Sleeper; Phase 3 study of the Cardiohelp System (an extracorporeal membrane oxygenation circuit) for the treatment of children with severe cardiac failure (approximately $2.4 million over four years) |
| 3. Extend Biosciences, Inc. (Newton, Massachusetts); Laura M Hales; Phase 2 study of EXT608 for the treatment of adults with hypoparathyroidism (approximately $3.6 million over four years) |
| 4. Indiana University-Purdue University at Indianapolis (Indianapolis, Indiana); Sherif S Farag; Phase 1/2 study of sitagliptin, bortezomib and post-transplant cyclophosphamide for the prevention of acute GVHD following allogeneic hematopoietic stem cell transplantation (approximately $2.6 million over four years) |
| 5. Massachusetts General Hospital (Boston, Massachusetts); Stephanie B Seminara; Phase 2 study of Kp-10 for the treatment of patients with idiopathic hypogonadotropic hypogonadism (approximately $2.6 million over four years) |
| 6. Mayo Clinic Rochester (Rochester, Minnesota); Jann N Sarkaria; Phase 1 study of WSDO628 with radiation therapy for the treatment of recurrent high-grade glioma (approximately $2.5 million over four years) |
| 7. New York University School of Medicine (New York, New York); Maria Alejandra Gonzalez-Duarte Briseno; Phase 2 study of dexmedetomidine sublingual film for the ambulatory treatment of hyperadrenergic autonomic crisis in patients with familial dysautonomia (approximately $2.6 million over four years) |
| 8. New York Medical College (Valhalla, New York); Mitchell S Cairo; Phase 2 study of maternal donor derived CMV cytotoxic T lymphocytes and valganciclovir vs valganciclovir in neonates with moderate/severe maternal acquired cytomegalovirus infection (approximately $2.6 million over four years) |
| 9. Ophirex, Inc. (Corte Madera, California); Tim Platts-Mills; Phase 2b study of varespladib for the treatment of snakebite envenoming (approximately $2.7 million over three years) |
| 10. Sloan-Kettering Institute Cancer Research (New York, New York); Joseph H Oved; Phase 2 study of bridging pre-transplant inflammatory dampening (immune suppression) for treatment of bone marrow transplant in primary immune regulatory disorders (approximately $2.6 million over four years) |
Searchable Database of Funded Grants
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.
Output Format
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.
Natural History Studies
FDA has funded natural history studies since 2016 to help address significant unmet medical needs for patients with rare diseases. Unlike common diseases, there is little existing knowledge on the presentation, major limitations on day-to-day function, core unmet needs and course of most rare diseases which makes drug development challenging. To address this, it is critical to study the natural history of rare diseases.
A natural history study is a preplanned, observational study intended to track the course of the disease. Its purpose is to identify demographic, genetic, environmental and other variables (e.g., treatment modalities, concomitant medications) that correlate with the disease’s development and outcomes. Information obtained from a natural history study plays an essential role at every stage of product development, such as identifying the patient population, identifying or developing clinical outcome assessments and biomarkers and, when appropriate, serving as external controls. Natural history studies are observational and non-interventional in nature and may be retrospective or prospective.
This program is intended to fund well-designed, protocol-driven natural history studies that address knowledge gaps, support clinical trials and advance rare disease medical products. OOPD has supported more than 15 natural history studies and currently awards new grants every two years. OOPD is focusing efforts to support efficient and innovative natural history studies that advance medical product development in rare diseases and conditions with unmet needs.
FY 2022 awards: Natural history study grants
1. Cincinnati Children’s Hospital Medical Center (Cincinnati, Ohio); Bruce Trapnell; Retrospective study in autoimmune pulmonary alveolar proteinosis to define disease progression and develop and test novel patient-reported outcome measures; $300,000 over two years
2. Columbia University Health Sciences (New York, New York); Mishaela Rubin; Prospective study in advancing product development for hypoparathyroidism; $1.6 million over four years
3. Jaeb Center for Health Research, Inc. (Tampa, Florida); Allison Ayala; Prospective study in gyrate atrophy to characterize the variability of ornithine aminotransferase (OAT) gene related ocular and systemic disease progression; $1.6 million over four years
4. Johns Hopkins University (Baltimore, Maryland); Howard Lederman; Prospective study in ataxia-telangiectasia; $1.6 million over four years
5. University of Minnesota (Minneapolis, Minnesota); David Walk; Retrospective and prospective study in amyotrophic lateral sclerosis of clinic-based multicenter data collection; $1.6 million over four years
6. University of Pennsylvania (Philadelphia, Pennsylvania); David Fajgenbaum; Prospective study in Castleman disease to add data to the ACCELERATE Registry; $1.6 over four years
7. Vanderbilt University Medical Center (Nashville, Tennessee); Evan Brittain; Prospective study in pulmonary arterial hypertension and genetically susceptible individuals; $1.6 million over four years
8. Virginia Commonwealth University (Richmond, Virginia); Nicholas Johnson; Prospective study in myotonic dystrophy type-1 to establish biomarkers and clinical endpoints; $1.6 million over four years
FY 2022 awards: Natural history study grants
| 1. Cincinnati Children’s Hospital Medical Center (Cincinnati, Ohio); Bruce Trapnell; Retrospective study in autoimmune pulmonary alveolar proteinosis to define disease progression and develop and test novel patient-reported outcome measures; $300,000 over two years |
| 2. Columbia University Health Sciences (New York, New York); Mishaela Rubin; Prospective study in advancing product development for hypoparathyroidism; $1.6 million over four years |
| 3. Jaeb Center for Health Research, Inc. (Tampa, Florida); Allison Ayala; Prospective study in gyrate atrophy to characterize the variability of ornithine aminotransferase (OAT) gene related ocular and systemic disease progression; $1.6 million over four years |
| 4. Johns Hopkins University (Baltimore, Maryland); Howard Lederman; Prospective study in ataxia-telangiectasia; $1.6 million over four years |
| 5. University of Minnesota (Minneapolis, Minnesota); David Walk; Retrospective and prospective study in amyotrophic lateral sclerosis of clinic-based multicenter data collection; $1.6 million over four years |
| 6. University of Pennsylvania (Philadelphia, Pennsylvania); David Fajgenbaum; Prospective study in Castleman disease to add data to the ACCELERATE Registry; $1.6 over four years |
| 7. Vanderbilt University Medical Center (Nashville, Tennessee); Evan Brittain; Prospective study in pulmonary arterial hypertension and genetically susceptible individuals; $1.6 million over four years |
| 8. Virginia Commonwealth University (Richmond, Virginia); Nicholas Johnson; Prospective study in myotonic dystrophy type-1 to establish biomarkers and clinical endpoints; $1.6 million over four years |
Other Past and Ongoing OOPD Supported Natural History Grants
Title
PI
Institution
Location
Start Date
End Date
1
Prospective Study in Angelman Syndrome
Tan, Wen Hann
Children's Hospital Corp
Boston, MA
09-15-2017
08-31-2022
2
Prospective Study in Friedreich Ataxia
Lynch, David
Children's Hospital of Philadelphia
Philadelphia, PA
09-01-2017
08-31-2022
3
Prospective Study in Pregnancy & Lactation Associated with Osteoporosis
Cohen, Adi
Columbia University Medical Center
New York, NY
09-01-2017
08-31-2022
4
Retrospective Study in Sarcoidosis
Gerke, Alicia
University of Iowa
Iowa City, IA
09-01-2017
08-31-2020
5
Prospective Study in Sickle Cell Anemia: Determine Biomarkers of Endothelial Function Changes in Chronic Kidney Disease
Ataga, Kenneth
University of Tennessee Health Science Center
Memphis, TN
09-01-2017
08-31-2022
6
Prospective Study in Myotonic Dystrophy Type 1 to Determine Biomarkers in Clinical Endpoints
Johnson, Nicholas
Virginia Commonwealth University
Richmond, VA
09-01-2017
08-31-2022
7
Prospective Study in Medullary Thyroid Carcinoma
Grubbs, Elizabeth
University of Texas MD Anderson Cancer Center
Houston, TX
09-01-2019
07-31-2023
8
Prospective Study in Cardiac Disease in Duchenne Muscular Dystrophy (DMD)
Soslow, Jonathan
Vanderbilt University Medical Center
Nashville, TN
09-01-2019
08-31-2023
Other Past and Ongoing OOPD Supported Natural History Grants
|
|
Title |
PI |
Institution |
Location |
Start Date |
End Date |
|
1 |
Prospective Study in Angelman Syndrome |
Tan, Wen Hann |
Children's Hospital Corp |
Boston, MA |
09-15-2017 |
08-31-2022 |
|
2 |
Prospective Study in Friedreich Ataxia |
Lynch, David |
Children's Hospital of Philadelphia |
Philadelphia, PA |
09-01-2017 |
08-31-2022 |
|
3 |
Prospective Study in Pregnancy & Lactation Associated with Osteoporosis |
Cohen, Adi |
Columbia University Medical Center |
New York, NY |
09-01-2017 |
08-31-2022 |
|
4 |
Retrospective Study in Sarcoidosis |
Gerke, Alicia |
University of Iowa |
Iowa City, IA |
09-01-2017 |
08-31-2020 |
|
5 |
Prospective Study in Sickle Cell Anemia: Determine Biomarkers of Endothelial Function Changes in Chronic Kidney Disease |
Ataga, Kenneth |
University of Tennessee Health Science Center |
Memphis, TN |
09-01-2017 |
08-31-2022 |
|
6 |
Prospective Study in Myotonic Dystrophy Type 1 to Determine Biomarkers in Clinical Endpoints |
Johnson, Nicholas |
Virginia Commonwealth University |
Richmond, VA |
09-01-2017 |
08-31-2022 |
|
7 |
Prospective Study in Medullary Thyroid Carcinoma |
Grubbs, Elizabeth |
University of Texas MD Anderson Cancer Center |
Houston, TX |
09-01-2019 |
07-31-2023 |
|
8 |
Prospective Study in Cardiac Disease in Duchenne Muscular Dystrophy (DMD) |
Soslow, Jonathan |
Vanderbilt University Medical Center |
Nashville, TN |
09-01-2019 |
08-31-2023 |