- Orphan Products Grants
OOPD administers two Orphan Products Grants:
- Clinical Trials Grants Program
- Natural History Studies Grants Program
Please view the program tabs for more information.
- Clinical Trials
Orphan products clinical trials grants are a proven method of successfully fostering and encouraging the development of new safe and effective medical products for rare diseases/conditions. OOPD funded grants ensure that product development occurs in a timely manner with a very modest investment. At any one time, there are typically 60 to 85 ongoing grant-funded projects. A major portion of the appropriated funds (typically approximately $15.5 million) for a given fiscal year go towards continued funding of prior approved grants. The rapid increase in the cost of clinical trials in recent years has precluded an increase in the number of new OOPD grants. OOPD conducts ongoing grant evaluations to ensure extramural funded studies, which involve human subjects, maintain grant agreement terms and minimize FDA’s exposure to risk of violations in human subjects’ protection requirements.
To address the remaining unmet need and the lack of treatments for the majority of rare diseases, FDA is focusing their efforts to facilitate and move new therapies along in drug development in safe yet efficient means by encouraging innovative clinical trial designs (e.g., adaptive, basket, umbrella trials), innovative methods (e.g., data modeling and simulations), the use of existing infrastructure, collaborative efforts between stakeholders, and early and ongoing patient engagement. All of these are vital to achieving efficient trials and data evaluation to expedite rare disease drug development.
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.
- Natural History Studies
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.
Definition of Natural History Studies: The natural history of a disease is traditionally defined as the natural course of a disease from the time immediately prior to its inception, progressing through its pre-symptomatic phase and different clinical stages to the point where the disease has ended without external intervention. A natural history study describes the course of a disease over time, identifying demographic, genetic, environmental, and other variables that correlate with its development and outcomes. These studies are likely to include patients receiving the current standard of care (e.g., supportive care, unapproved treatment options), which may alter some otherwise natural manifestations or course of the disease. Natural history studies can be prospective or retrospective.
History of OOPD Funding Natural History Studies: OOPD launched the Natural History grants in 2016. 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 product development. OOPD has issued two RFAs and committed a total of $13.9 million since the inception of this program in 2016, of which $3.5 million was provided through a partnership with the National Institutes of Health’s (NIH) National Center for Advancing Translational Sciences (NCATS). We are hopeful that these important studies will provide key information about how these rare diseases progress over time and can ultimately assist making the development process more efficient.
Past and Current OOPD Supported Natural History Grants
Prospective Study in Angelman Syndrome
Tan, Wen Hann
Children's Hospital Corp
Prospective Study in Friedreich Ataxia
Children's Hospital of Philadelphia
Prospective Study in Pregnancy & Lactation Associated with Osteoporosis
Columbia University Medical Center
New York, NY
Retrospective Study in Sarcoidosis
University of Iowa
Iowa City, IA
Prospective Study in Sickle Cell Anemia: Determine Biomarkers of Endothelial Function Changes in Chronic Kidney Disease
University of Tennessee Health Science Center
Prospective Study in Myotonic Dystrophy Type 1 to Determine Biomarkers in Clinical Endpoints
Virginia Commonwealth University
Prospective Study in Medullary Thyroid Carcinoma
University of Texas MD Anderson Cancer Center
Prospective Study in Cardiac Disease in Duchenne Muscular Dystrophy (DMD)
Vanderbilt University Medical Center