|
|
CENTER FOR DRUG EVALUATION AND RESEARCH
Accelerating Rare disease Cures (ARC) Program Update
|
|
CDER ARC’s LEADER 3D Announces New Rare Disease Drug Development Educational Materials
Today, the U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) published four additional 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 cases include:
- Belimumab (Benlysta), which demonstrates the use of a Bayesian analysis design to determine the efficacy of a treatment for systemic lupus erythematosus.
- Chenodiol (Ctexli), which describes the use of a biomarker and natural history data to support repurposing chenodiol for the treatment of cerebrotendinous xanthomatosis.
- Inebilizumab-cdon (Uplizna), which demonstrates the use of a novel organ-tissue specific flare definition to inform a primary endpoint for the treatment of immunoglobulin G4-related disease (IgG4-RD).
- Omaveloxolone (Skyclarys), which demonstrates the use of a natural history study as confirmatory evidence to support substantial evidence of effectiveness for the treatment of Friedreich’s ataxia.
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. 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 the rare disease drug development community.
Additional Information
|
|
|
|
