Harrison JE, Paulsen R, Acaster S, Herring W. Are we patient and care-partner centric enough in early Alzheimer's disease clinical trials? The potential limitations of using legacy clinical outcome assessments (COA) in the new era of Alzheimer's disease drug development. Presented at the ISPOR 2022 Conference; May 17, 2022. Washington, DC.

PURPOSE: To evaluate current clinical outcomes assessments (COA) measures and strategies in mild cognitive impairment (MCI) and early Alzheimer's Disease (AD) and explore whether the use of legacy AD measures in the new era of early AD drug development is limiting our ability to identify patient meaningful benefit.

DESCIPTION: The recent paradigm shift in AD drug development, from later stage symptomatic AD to disease modifying monoclonal antibody (MAB) therapies in MCI and early AD, has received a lot of attention: good and bad. With a lot of focus on biomarkers and price, the patient relevance of these new treatments appears to have been lost, or lost in translation, as legacy definitions of meaningful change and difference have been applied to this new context.

Fit for purpose of COAs in MCI and early AD clinical trials should measure patient relevant concepts, using appropriate item wording and response options, reported by and appropriately informed respondent, to be able to define and appropriately interpret patient benefit.

This workshop will explore whether we can effectively evaluate the patient relevant meaningful benefit of new MAB therapies with the legacy AD COAs, typically being used in clinical trials.

This workshop will include presentations to address:

What do we know about what matters most to MCI and early AD patients and their care-partners?

How well do current COA used in MCI and early AD trials address concepts that matter in MCI and early AD?

How can we currently evaluate patient relevant meaningful benefit in MCI and early AD and what should we consider for the future?

How can we leverage COA trial data to support patient, care-partner and payer relevant long-term treatment benefits.

The content of this workshop will be interest to clinicians, payers, regulators, study sponsors, COA scientists, health economists and patient associations in the AD field.

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