CDER ARC’s LEADER 3D Announces New Rare Disease Drug Development Educational Materials
The U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) published two new case studies 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 sponsors successfully used when designing and conducting rare disease drug development programs. The case studies are:
(1) Mavorixafor (Xolremdi), which demonstrates the use of the win ratio method in a rare disease program to prioritize the components of a composite clinical endpoint based on their relative clinical importance.
(2) Lumasiran (Oxlumo), which illustrates the use of natural history and registry data to inform biomarker-based endpoint selection for a rare disease drug program.
FDA recognizes 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. These case studies are not intended or designed to provide strategies for obtaining product approval. 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.
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. These case studies and other materials on the LEADER 3D webpage reflect the needs and priorities of our partners in rare disease drug development.
In Case You Missed It: RDEA Pilot Program Workshop Materials Available
Recording and slides are now available from a workshop convened by FDA and Triangle CERSI on May 18, 2026, that focused on the development of surrogate endpoints for drugs and biological products intended to treat rare diseases. At this Rare Disease Endpoint Advancement (RDEA) Pilot Program workshop, Advancing Novel Surrogate Endpoints for Rare Disease Drug Development, participants discussed mechanistic and translational science evidence needed to support use of novel surrogate endpoints in rare disease marketing applications. Discussion also included new approach methodologies, digital health technology, and harnessing varied sources of patient-level real-world data.
More rare disease drug development resources are available on CDER’s Accelerating Rare disease Cures Program webpage.
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