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  1. Developing Products for Rare Diseases & Conditions

About Orphan Products 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 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.

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.