On July 28, 2025, Knowledge Ecology International (KEI) submitted a Freedom of Information Act request for records concerning the Food and Drug Administration (FDA) Orphan Products Grant Program. The program (administered by the FDA’s Office of Orphan Products Development (OOPD)) provides grants for clinical trials and has funded over 700 grants related to orphan clinical trials.
While a list of all the grants made through the program is available on an FDA database, the only information provided to the public is: Title, PI, Institution, Location, Start Date, and End Date.
KEI has sought to match the data from the grant data provided by the FDA with information available in the NIH RePORTER and the NIH ClinicalTrials.Gov databases. However, the search process is difficult, and we are unable confirm whether the datasets align. Some of the issues we have encountered (and noted in our FOIA request) are:
- “The projects with similar or identical titles often appear in different years, with different associated institutions, and locations, making it unclear whether they refer to the same award.
- Sub-projects frequently lack descriptive information (often the abstract text), leaving the grant title as the only indicator. Even then, titles differ across the FDA, NIH RePORTER, and CinicalTrials.gov databases.
- Principal Investigators (PIs) listed in the FDA data sometimes do not appear for a single project in NIH RePORTER, or the search returns unrelated projects.
- Even when the related record can be located, most entries do not include key information, such as the NCT number or a link to ClinicalTrials.gov. Moreover, when NCT information is listed, sometimes it is linked to the incorrect and unrelated clinical study. For example, a project on pancreatic cancer included, in the clinical studies section, a study on hormones in premenopausal women.”
As treatments for rare diseases currently make up roughly half of all new drugs approved by the FDA and have significant impacts on U.S. spending on medicines, it is important to have accurate information on the amount of federal subsidy of the trials, in order to evaluate the efficacy and efficiency of incentives and subsidies for the development of treatments for orphan diseases and the reasonableness of prices for treatments.
KEI’s FOIA request seeks further metadata on the 700+ grants made through the FDA Orphan Products Grant Program, including: NIH Project Number (as used in the NIH RePORTER), NCT identifier of the trial, the enrollment and the grant amount.