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Clinical Trial and Natural History Study Grants

Orphan Products Grants

OOPD administers two Orphan Products Grants:

  1. Clinical Trials Grants Program
  2. Natural History Studies Grants Program

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.

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.



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