This opinion article from The Seattle Times will be a story familiar to many in the rare disease community. Clinical trials showed promise, and patients expected a green light for drug approval. Instead, the U.S. Food and Drug Administration issued a “refusal-to-file” letter and declined to ...examine the trial data. The FDA insists on large trials showing clear evidence of a drug’s clinical effectiveness, meaning that symptoms improve or disappear. But that doesn’t make sense for rare diseases. The agency’s denial was devastating for families, and the FDA needs to reconsider how it evaluates #raredisease drugs.