The U.S. Food and Drug Administration (FDA) is bolstering the development of treatments for rare diseases through its Orphan Products Grants Program, awarding $17.2 million to seven new clinical trials in fiscal year 2024. This funding aims to propel promising treatments through clinical development, addressing conditions that affect fewer than 200,000 people in the U.S. The grants, including one Phase 3 trial, signify a crucial step in bridging the gap between early research and successful treatment for rare diseases.
Orphan Products Grants Program
Launched as part of the Orphan Drug Act of 1983, the Orphan Products Grants Program provides financial support for research and development of drugs, biologics, medical devices, and medical foods targeting rare diseases. Administered by the FDA Office of Orphan Products Development (OOPD), the program has facilitated the approval of over 85 products since its inception.
The program encompasses two key funding opportunities: the Clinical Trials Grants Program and the Natural History Studies Grants Program. The Clinical Trials Grants Program focuses on evaluating the safety and efficacy of potential treatments, while the Natural History Studies Grants Program gathers data on the progression of rare diseases over time without treatment. This data is crucial for designing effective clinical trials and defining meaningful endpoints for future drug development.
Clinical Trials Grants: Eligibility and Review
The FDA's Orphan Products Clinical Trials Grants Program is open to various entities, including academic institutions, industry sponsors, non-profit organizations, and public or private entities, both within and outside the U.S. Eligible clinical trials must aim to obtain data on the safety and/or efficacy of a product for diagnosing or treating a rare disease. Both randomized controlled trials and other designs, such as single-arm or open-label studies, may qualify, regardless of whether the study is early-stage (Phase 1) or later-stage (Phase 2 and 3).
Applications undergo a rigorous peer-review process, considering factors such as rationale, study design, inclusion of patient input, investigator qualifications, infrastructure, financial resources, and the potential to advance the field. Consultation with the relevant FDA review division occurs during the review process to determine whether the proposed study will provide acceptable data that could contribute to product approval.
Financial Support and Strategic Alignment
The FDA offers substantial financial support for qualified projects, with Phase 1 clinical trials eligible for up to $250,000 per year and Phase 2 and 3 clinical trials eligible for up to $500,000 per year. Funding typically lasts up to four years, with potential extensions under certain circumstances. Securing a Clinical Trials Grant provides valuable insight, indicating that the FDA review team likely considers the proposed study capable of generating acceptable data for potential product approval.
FY2024 Grant Recipients
For FY2024, the FDA received 51 grant applications and awarded seven new clinical trials, allocating over $17.2 million to advance the development of medical products for rare diseases. The awardees include six early-stage trials (Phase 1, Phase 1/2, and Phase 2) across rare cancers, hematology, endocrinology, and ocular diseases, as well as one Phase 3 trial for microcystic lymphatic malformations, a rare genetic skin disease with no FDA-approved therapies. This Phase 3 study utilizes a single-arm, baseline-controlled design, representing an innovative and efficient approach.
The FDA's Orphan Products Grants Program remains a critical component of efforts to develop treatments for rare diseases, driving innovation and bringing much-needed therapies to patients affected by these conditions.
