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FDA Continues to Make Inroads in Rare Disease Drug Development

Every year, Rare Disease Week seeks to bring together the Rare Disease Community to exchange experiences, insights, and challenges, and discuss future innovations for rare diseases in our country.

At the Center for Drug Evaluation and Research’s (CDER) Accelerating Rare Disease Cures (ARC) Program, we believe events like these are a real opportunity to learn, share and support the people who are impacted by rare diseases.

FDA continues ongoing efforts through the ARC program to accelerate cures and therapies for the millions of people affected by rare diseases in our country. We wanted to share with you some of the work and resources both in partnership with and for the rare disease community.

• The ARC Program’s Year Two Annual Report
  Read about how the program is driving innovation through scientific and regulatory advancement.

• Recent Drug Approvals
  The ARC program adds recent CDER drug approvals on the ARC website and highlights novel approvals for rare diseases, including recently approved chenodiol. We also encourage you to read the recently released New Drug Therapy Approvals 2024 report,  which profiled CDER’s extensive work for rare diseases.

• LEADER 3D
  The Learning and Education to Advance and Empower Rare Disease Drug Developers initiative offers educational and learning opportunities for all those involved in or would like to learn about the design and conduct of rare disease clinical trials. Here you will find new videos and written educational content that helps summarize and inform important information related to rare disease drug development.

• Upcoming and Recent Events
  Learn more about upcoming and recent events with which the ARC program is involved. Conferences, workshops, and meetings focused on rare disease drug development serve as key platforms where the rare disease community unites to exchange experiences, insights, challenges, and discuss future innovations. As an example, we invite you to visit the May 13, 2024, Natural History Studies and Registries in the Development of Rare Disease Treatments | Reagan-Udall Foundation website.

• Guidances
  Review guidance documents that are relevant to rare disease drug development, organized by topic.

Together, through engagement, collaboration and innovation, we can make meaningful strides toward transforming the lives of those affected by rare diseases.

This is an automated message delivery system. Replying to this message will not reach DDI staff. If you have comments or questions, please contact us at: 1-888-INFO- FDA (1-888-463-6332) or 301-796-3400 from 8:00 am - 4:30 pm ET Monday - Friday. You can also email us at: druginfo@fda.hhs.gov.