Workshop B: Decoding Clinical Endpoints in ALS: Revolutionizing Drug Development with Emerging Biomarkers, ALS-FRS-R & Digital Endpoints
Time: 9:00 am
day: Pre Conference Day Workshop B
Details:
This workshop explores the ALS Functional Rating Scale-Revised (ALSFRS-R) as a cornerstone clinical endpoint, addressing its limitations and strategies to enhance its impact. We’ll investigate the role of digital biomarkers, such as wearables and smartphone-based assessments, alongside other primary and secondary endpoints like respiratory function, survival, and quality of life. Attendees will gain actionable insights into building a robust, multidimensional framework for trial design, aligned with regulatory requirements, to accelerate ALS drug development and approvals.
- Critically examine the ALSFRS-R to analyze the strengths, limitations, standardization concerns and key considerations when using ALSFRS-R as a clinical endpoint, including its sensitivity to disease heterogeneity
- Evaluate the role of wearables, smartphone-based assessments, and AI-driven analysis as digital endpoints in capturing nuanced progression signals for ALS
- Integrate diverse endpoints to discuss the interplay of primary, secondary, and exploratory endpoints—such as respiratory function, survival rates, and quality-of-life measures—in building a comprehensive efficacy profile
- Streamline approvals to align endpoint selection with regulatory expectations, fostering more efficient pathways for drug approval
