Duchenne Muscular Dystrophy: FDA Supports Broader Outcome Measures, Biomarkers
This article was originally published in The Pink Sheet Daily
Executive Summary
Embracing patient community recommendations, FDA draft guidance says historically controlled trials may support efficacy in some cases.
You may also be interested in...
Patient-Focused Drug Development, Complex Trial Design May Not Get More Funding In PDUFA VII
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.
Rare Diseases: US FDA Gives More Guidance On Premarket Safety Evaluation, Historical Controls
Agency specifies several approaches to augment safety assessment in a new draft guidance, which also encourages participation by patients and their advocates in the drug development process.
Patient Experience Data: US FDA Guidance Offers Other Ways To Share
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.