1/29/2024 0 Comments Rare disease day piccollage![]() In the Center for Drug Evaluation and Research (CDER), 31 of the 53 novel drug approvals, or 58%, were orphan designated products. Specifically, in 2020, the agency approved 32 novel drugs and biologics with orphan drug designation. In 2020, we continued to see significant progress in the development of treatments for rare diseases, also known as orphan products. Patients with rare diseases often have few or no treatment options. Efforts include collaborative solutions to address current challenges and regulatory innovations to streamline the development process.Īpprovals in 2020 of Novel Drugs and Biologics with Orphan Drug Designation, Treatments for Rare DiseasesĪs the FDA focuses on the COVID-19 pandemic, the agency also remains dedicated to its crucial role in development of treatments for rare diseases. CBER is collaborating with the Foundation for the National Institutes of Health (FNIH) and the National Center for Advancing Translational Sciences (NCATS) on development of a ‘Bespoke Gene Therapy Consortium’ (BGTC) to provide a standardized and efficient approach for development and delivery of bespoke AAV-based gene therapies. Stakeholders recognize the need to have an end-to-end approach for development of individualized (bespoke) gene therapies and sustainable access for patient populations that can benefit from these therapies. The Center for Biologics Evaluation and Research (CBER) is helping to advance the development of individualized therapies for rare diseases that affect one or a few individuals. Bespoke Gene Therapy Consortium (BGTC).In addition, the project includes FDA staff as they share examples of rare disease work across the FDA. To build on our many programs and initiatives aimed at promoting inclusion of the patient voice, we captured brief stories from the rare disease community. It is imperative for the voices of patients to be integrated into all phases of medical product development. People are at the heart of the FDA’s work. FDA Rare Disease Photo and Video Project. ![]() This new RFA includes increased emphasis on natural history studies with high quality and interpretable data elements that can facilitate rare disease product development. The program continues to evolve to address the many unmet needs in rare diseases. Both natural history studies and clinical trials for rare diseases are supported through the Orphan Products Grants Program with a goal to increase the number of treatments for rare diseases with an unmet medical need and exert a broad and positive impact on rare disease drug development. In 2020, there were more than five product approvals associated with the Orphan Products Grants program, including a treatment for thyroid eye disease and a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). This program has supported rare disease clinical trial research since 1983 and has facilitated over 70 product approvals. The FDA funds critical rare disease research through a variety of programs, such as the Congressionally-mandated Orphan Products Grants Program. New request for applications (RFA) for the Orphan Products Grants Program for Efficient and Innovative Natural History Studies Addressing Unmet Needs in Rare Diseases.Additionally, today we are pleased to announce new actions that extend our efforts in this important space: This meeting will bring together stakeholders to highlight strategies that can promote rare disease product development. To recognize Rare Disease Day and engage with the rare disease community, the FDA is holding a virtual public meeting on March 5, 2021. Food and Drug Administration is committed to supporting development of treatments for rare diseases. The COVID-19 pandemic has added urgency, because people with rare diseases are among the most vulnerable to COVID-19. Rare Disease Day is a time to reflect on both the progress that has been made, and the work that needs to be done, to advance rare disease treatments. By: Janet Woodcock, M.D., Acting Commissioner and Janet Maynard, M.D., M.H.S., Director, Office of Orphan Products Development
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