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.

Highlights

• Global CEO Initiative on Alzheimer's Disease (CEOi) convened a multi-stakeholder working group to develop recommendations for Alzheimer's disease (AD) endpoint design and communication

• Earlier intervention and disease-targeting therapies are reshaping AD outcome measurement needs

• The working group identified two priorities: improving communication of existing measures and advancing novel endpoints

• Patient-centered approaches, including responder analyses and time-saved estimates, may improve interpretation of treatment benefits

• Novel endpoints should be sensitive to early disease changes, scalable, accessible, and aligned with patient and care partner priorities

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