Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01) Clinical Trials Required

The U.S. Food and Drug Administration (FDA), through the Office of Orphan Products Development (OOPD), is offering funding under the R01 Research Project Grant to support clinical trials of orphan products addressing unmet needs in rare diseases. This program specifically aims to foster clinical trials that evaluate the efficacy and/or safety of medical products with the goal of obtaining new indications or label changes. The clinical trials funded under this initiative should work towards increasing the number of FDA-approved treatments for rare diseases. In addition, the FDA encourages collaborative and innovative approaches in trial design to streamline and expedite the drug development process. The funding is structured to support one year of initial work with options for three additional years of funding, provided the research meets certain performance and regulatory milestones. The budget allows for up to $650,000 per year in total costs, with a possibility of an additional $250,000 per year for innovative clinical trial designs or methods. Examples of qualifying innovative designs include adaptive, seamless, basket, and platform trials, which can speed up development by compressing phases of trials or testing multiple treatments or diseases simultaneously. The scope of the grant includes clinical trials in all phases of product development (phase 1, 2, or 3). Trials should involve well-justified controls, such as placebos or historical controls. Patient engagement in the trial design and protocol is highly encouraged to ensure feasibility and relevance. Moreover, the use of shared infrastructure, collaboration between academia, industry, and patient organizations, and innovative data sharing platforms are strongly recommended. Eligibility for this funding opportunity is broad, including higher education institutions, nonprofits, small businesses, for-profit organizations, state and local governments, and foreign entities. Applicants should ensure they meet registration requirements such as SAM, eRA Commons, and Grants.gov before submitting an application. Applications must be submitted electronically through Grants.gov, NIH ASSIST, or an institutional system-to-system solution. A letter of intent is optional but recommended to help FDA estimate the review workload. The due dates for submission are October 24, 2023, and October 22, 2024, for new applications. Resubmission applications have separate deadlines of June 4, 2024, and June 3, 2025. Applications will undergo a program responsiveness review and a scientific merit review by an FDA Objective Review Committee. The key review criteria include the strength of the rationale, the quality of the study design, the inclusion of patient input, the experience of the investigator team, and the potential of the study to advance the field and support new product labeling. Applicants must ensure their protocols comply with applicable FDA regulations, including IND/IDE requirements for drugs, biologics, and devices, and that ethical approvals are obtained in advance. Post-submission materials will only be accepted under limited circumstances, such as final IRB approval or confirmation of product supply. The award duration can extend up to four years, with annual continuation dependent on performance, compliance with regulatory requirements, and availability of funds. Financial and performance reporting are required annually, along with regular monitoring by FDA staff to ensure compliance and progress towards study goals.