Expert perspectives on the design and communication of endpoints in Alzheimer's disease clinical trials

Abstract

Disease‐targeted therapies (DTTs) for Alzheimer's disease (AD) represent a major advance, yet challenges remain on how best to interpret and communicate their benefits. As clinical development increasingly targets the earliest stages of AD (before symptoms emerge and early symptomatic AD), there is a need for novel, complementary endpoints and improved communication of treatment benefits. The Global CEO Initiative on Alzheimer's Disease convened a multi‐stakeholder working group to develop recommendations for improving the design and communication of endpoints in AD clinical trials. In the near term, communication can be improved by contextualizing treatment effects at the individual level, including framing benefits in terms of responder analyses and “time saved” over defined timeframes. In parallel, continued development of complementary endpoints is needed to enable more sensitive, scalable, and patient‐centered measurements aligned with early intervention. These recommendations aim to support stakeholders in evaluating and communicating the meaningful benefits of current and emerging therapies.