Disease-Specific Guidances Reflect US FDA's 'Nimble, Collaborative, Patient-Focused' Approach
Patient groups helped with draft guidance on ALS drug development and revision of Alzheimer's draft guidance; agency also issues draft guidance on pediatric seizure, final guidances on DMD and migraine.
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ALS advocates succeed in getting the FDA to soften its stance on use of randomized, placebo-controlled trials for drug development; final guidance also encourages patient-reported outcomes.
Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.