Pursuing Accelerated Approval in Rare Diseases: Leveraging Novel Biomarkers & Endpoints to Support Regulatory Decision Making
9:00 am - Wednesday 27th January 2027
- Evaluating the use of validated biomarkers and surrogate endpoints as earlymindicators of treatment benefit to generate rapid evidence for regulatory bodies
- Exploring regulatory expectations for novel endpoint qualification in rare muscular disorders
- Learning from successful accelerated approval case studies to identify best practices and increase the likelihood of regulatory success
