WASHINGTON — FEBRUARY 23, 2026
The Plausible Mechanism draft guidance creates a novel framework through which cutting-edge treatments tailor-made for patients with ultra-rare diseases can be used as a basis for FDA approval,” said Center for Drug Evaluation and Research Acting Director Tracy Beth Høeg, MD, PhD. “We anticipate our Plausible Mechanism draft guidance will inspire industry to place increased focus on individualized therapies, thereby driving innovation, improving safety, lowering costs and offering more patients with ultra-rare diseases a unique shot at a life-saving treatment.”
Provocative commentary:
After genomic profiling has been obtained for an individual's cancer, doesn't every such cancer become a "rare" disease?