FDA – Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)

October 30, 2018 by School of Medicine Webmaster

The FDA Office of Orphan Products Development (OOPD) was created to identify and promote the development of orphan products. Orphan products are drugs, biologics, medical devices, and medical foods that are indicated for a rare disease or condition. The term “rare disease or condition” is defined in 21 U.S.C. 360ee.  As a practical way to implement the statutory definition, for devices and foods as well as for drugs, FDA considers drugs, devices, and medical foods potentially eligible for grants under the Orphan Products Development (OPD) Clinical Trials Grants Program if they are indicated for a disease or condition that has a prevalence, not incidence, of fewer than 200,000 people in the United States. Diagnostics and vaccines are considered potentially eligible for such grants only if the U.S. population to whom they will be administered is fewer than 200,000 people in the United States per year.

There are over 7000 rare diseases that affect ~ 30 million Americans but only a few hundred of these rare diseases currently have approved treatments.  The Orphan Products Grants Program has been supporting clinical trial research since 1983 and has facilitated the marketing approval of more than 60 of those products.  To address the remaining unmet need and the lack of treatments for the majority of rare diseases, FDA is focusing their efforts with this FOA to facilitate and move new therapies along in drug development in safe yet efficient means by encouraging innovative clinical trial methods such as adaptive and seamless trial designs, modeling and simulations, and basket and umbrella trials.  These methods are vital to efficient trials and data evaluation which can expedite drug development.

Research Objectives

This FOA is intended to support clinical studies of products for rare diseases with an unmet medical need. These clinical studies should evaluate safety and/or effectiveness of medical products in a way that will substantially contribute to the body of evidence needed to support marketing approval or provide essential data to facilitate medical product development for rare diseases.

Use of innovative efficient trial designs is encouraged under this FOA.  For example, seamless trial designs, which compress the phases of a trial into one continuous trial, as well as basket, umbrella and platform trials, which allow for testing of multiple drugs and/or multiple diseases using a common infrastructure.  Early stages of product development can also hold significant promise for the advancement of curative treatments for rare diseases. Consideration should also be given to the use of real world data, which has the potential to allow for more efficient design and conduct of clinical trials in the health care setting to answer questions previously though infeasible.  Real world data can also, in certain cases, be analyzed to support medical product development and approval using novel analytical approaches.  In addition, applications that propose adaptive trial designs, simulations, and modeling used toward the study of safety and effectiveness of a product are encouraged. For example, modeling and simulation allow for organization of diverse data sets, optimization of product dosing based on individual physiology and genetics, and can provide a vital tool to help evaluate new treatments in rare diseases where patient populations are inherently difficult to study because of their small size. Many of these approaches are appropriate in the early stages of product development and may hold significant promise for the advancement of therapeutic treatments for rare diseases. Early engagement with FDA review divisions to discuss the use of these innovative tools is recommended prior to submitting a grant application.

Inclusion of patient and caregiver perspectives from the rare disease community is highly encouraged. Patients living with a rare disease or a family member who cares for them, have experiences and knowledge that contribute to important patient preference information such as when assessing benefit/risk.

Deadline:  May 25, 2019 (letters of intent); June 25, 2019 (full proposals)

URL:  https://grants.nih.gov/grants/guide/rfa-files/RFA-FD-20-001.html

Filed Under: Funding Opportunities