Clinical trial endpoints define what success looks like, but patients and caregivers are rarely the ones setting that definition, and the gap between what trials measure and what patients actually value is showing up in post-approval evidence, regulatory submissions, and the design rooms where protocols are written.
The question of which outcomes a clinical trial should measure has grown more complicated as therapies have become more targeted, patient populations more specific, and regulators more attentive to patient-focused drug development. Endpoint selection has historically been driven by expert clinical teams, regulatory precedent, and analytical convention, but each of those inputs has a blind spot. Together they are being challenged.
