Vertex Pharmaceuticals scored a minor coup this week when the Food and Drug Administration signed off on a wider use of its banner drug for cystic fibrosis. But the decision wasn’t based on a large, rigorous clinical trial — or any study involving humans at all. Instead, the FDA took the word of a test tube.
The agency looked at lab-test results and determined that Vertex’s drug, Kalydeco, would benefit an additional number of cystic fibrosis patients, pointing out that the drug had already demonstrated its safety in earlier human trials.
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