Duchenne Muscular Dystrophy: FDA Supports Broader Outcome Measures, Biomarkers
This article was originally published in The Pink Sheet Daily
Embracing patient community recommendations, FDA draft guidance says historically controlled trials may support efficacy in some cases.
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US FDA proposed increasing staffing and resources for both programs during the next user fee program cycle, but industry representatives did not believe it necessary.
Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.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.