Spotlight Speaker: Clinical Trial Endpoints for Therapeutics
Spotlight Speaker: Clinical Trial Endpoints for Therapeutics
Join Dr. Nily Osman, a board-certified neurologist bridging clinical medicine and CNS drug development, as she reveals why clinical trial endpoint selection can make or break therapeutic success—especially in a field where approval rates hover at just 4–7%.
In this essential session, you’ll discover the six hallmarks of a good endpoint—reliability, sensitivity, reproducibility, low variability, biological relevance, and operational feasibility—and why “clinically meaningful” must capture the patient’s voice, not just the clinician’s perspective. Dr. Osman unpacks the endpoint hierarchy from primary to surrogate endpoints, explains why the FDA accepts clinical scales like ADAS-Cog and CDR-SB but rejects plasma biomarkers as primary endpoints, and examines the Aducanumab controversy that triggered FDA advisory committee resignations.
This matters because CNS trials face uniquely high placebo responses lasting 6–12 months in mild cognitive impairment, subtle biological signals, and complex operational demands. After watching, you’ll be able to design study duration to outlast placebo effects, evaluate QALY and payer-economics thresholds of $100k–$150k per QALY, compare fast randomized placebo-controlled versus adaptive trial designs, and assess the operational feasibility—site expertise, rater quality, cost—that determines trial success and market access.
Learning Objective
Learning Goals
▪ Define the characteristics of a good endpoint — reliable, sensitive, reproducible, low-variability,
biologically relevant, and operationally feasible — and explain why “clinically meaningful” must
reflect the patient’s voice, not only the clinician’s.
▪ Distinguish the endpoint hierarchy — primary, secondary, exploratory, biomarker, and surrogate
endpoints — and recognize how the primary endpoint drives trial success, labeling, and investor
due diligence.
▪ Anticipate the placebo-response challenge in CNS trials (which can run 6–12 months in mild
cognitive impairment) and design study duration to “run out” the placebo signal.
▪ Evaluate regulatory considerations — why the FDA accepts clinical scales (ADAS-Cog, CDR-SB)
but not plasma biomarkers as primary endpoints, and how the surrogate-endpoint debate triggered
the Aducanumab advisory-committee resignations.
▪ Weigh the QALY / payer-economics lens (US thresholds of ~$100k–$150k per QALY) and
understand how endpoint choice translates into reimbursement and market access.
▪ Compare trial-design strategies — fast randomized placebo-controlled studies versus integrative
adaptive designs — and assess the operational feasibility (cost, expertise, sites, raters) that often
decides whether a study succeeds.
Resources