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CENTER FOR DRUG EVALUATION AND RESEARCH
Accelerating Rare disease Cures (ARC) Program Update
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CDER’s ARC LEADER 3D Program Releases New Video on Dose-Finding Considerations in Rare Disease Drug Development
The U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) released a video, which was developed as part of the Accelerating Rare disease Cures (ARC) Program’s Learning and Education to ADvance and Empower Rare Disease Drug Developers (LEADER 3D) initiative. The video highlights approaches and considerations sponsors should make when conducting dose-finding studies for rare disease drug development programs.
FDA recognizes that aspects of drug development that are feasible for common diseases may not be feasible for rare diseases and that development challenges are often greater with the increasing rarity of the disease. The small population affected by a rare disease presents additional considerations and calls for appropriate flexibility.
The ARC Program launched LEADER 3D to better understand and address the unique challenges in bringing rare disease therapies to market. As part of the initiative, CDER’s Rare Diseases Team facilitated interviews with the rare disease drug development community and reviewed public docket comments to identify educational opportunities across interest areas in rare disease drug development.
This video and all materials (case studies and other videos) on the LEADER 3D webpage reflect the needs and priorities heard from our valuable partners in the rare disease drug development community. The kind and quantity of data in each rare disease application will be different based on the unique considerations of each development program and must be assessed on a case-by-case basis.
Additional Information
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