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Expectations and clinical meaningfulness of randomized controlled trials
Ronald C Petersen1, Paul S Aisen2, J Scott Andrews3
1Department of Neurology, Mayo Clinic, Rochester, Minnesota, USA.
Alzheimer'S & Dementia : the Journal of the Alzheimer'S Association
|February 7, 2023
Summary
Interpreting Alzheimer's disease (AD) clinical trial results requires careful consideration of meaningful change. Experts convened to discuss how to best assess therapeutic benefits and slow disease progression, potentially modifying outcome expectations.
Area of Science:
- Neurology
- Clinical Trials
- Gerontology
Background:
- Alzheimer's disease (AD) pathology begins years before symptoms manifest, offering multiple intervention windows.
- Assessing therapeutic impact in AD clinical trials necessitates defining clinically meaningful change.
- Early intervention and consistent assessment are crucial for evaluating treatment efficacy.
Purpose of the Study:
- To discuss key considerations for interpreting data from cognitive and functional measures in AD clinical trials.
- To define what constitutes a clinically meaningful benefit or slowing of Alzheimer's disease progression.
- To re-evaluate expectations for therapeutic intervention outcomes in AD.
Main Methods:
- Convened a work group of experts.
- Reviewed existing discussions on clinically meaningful change in AD therapeutics.
- Focused on interpreting data from cognitive and functional measures.
Main Results:
- Considerable discussion exists regarding the definition of clinically meaningful change in AD trials.
- Early intervention strategies and 18-month trial assessments are critical.
- Expectations for therapeutic outcomes in AD may require modification.
Conclusions:
- Interpreting AD clinical trial data requires a nuanced understanding of meaningful change.
- Expert consensus is needed to standardize the assessment of therapeutic benefits.
- Modified expectations may be necessary for evaluating future AD interventions.
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