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LEADER 3D: Announcing New Rare Disease Drug Development Educational Materials
Today, the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (CDER) published two case studies and a case study user guide, 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.
These case studies highlight approaches successfully used by sponsors when designing and conducting rare disease drug development programs. The cases include:
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Fosdenopterin (Nulibry), which demonstrates the use of a single adequate and well-controlled investigation and confirmatory evidence to support substantial evidence of effectiveness, and
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Olipudase alfa-rpcp (Xenpozyme), which illustrates a clinical dose escalation strategy, informed by multiple data sources, for a rare disease drug program.
- To accompany the case studies, a user guide was also published to assist with navigating the case studies, provide key concepts, and additional resources on key regulatory topics.
Note: Case studies are not intended or designed to provide specific strategies for obtaining product approval. Rare disease drug development is not one-size-fits-all. The type and quantity of data in each rare disease application will be different based on the unique considerations of each development program and must therefore be assessed on a case-by-case basis.
[READ THE CASE STUDIES]
The ARC Program launched LEADER 3D to better understand and address the unique challenges in bringing rare disease products to market. As part of the initiative, CDER’s Rare Diseases Team worked with an independent contractor to conduct interviews with the rare disease drug development community and performed a review of public docket comments to identify educational opportunities across topics of interest in rare disease drug development. These case studies and the other materials on the LEADER 3D website are reflective of the needs and priorities heard from our valuable partners in the rare disease drug development community.
Additional Information
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