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Patient groups helped with draft guidance on ALS drug development and revision of Alzheimer's draft guidance; agency also issues draft guidance on pediatric seizure, final guidances on DMD and migraine.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Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.
Beyond submitting proposed guidances to FDA, stakeholders may develop patient registries, conduct natural history studies, and establish partnerships to help advance patient-focused drug development, agency suggests.