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Economic Considerations in Designs and Modifications of Multiarm, Multistage Adaptive and Adaptive Platform
Mwayi Kachapila1, Samuel Watson2, Thomas Pinkney3
1Health Economics Unit, Institute of Applied Health Research, College of Medical and Dental Sciences, University of Birmingham, Birmingham, England, UK; Global Health and Global Surgery Unit, National Institute for Health and Care Research (NIHR), University of Birmingham, Birmingham, England, UK.
Summary
Embedding health economics into adaptive trial designs is emerging but limited. While cost-effectiveness analyses can inform decisions in multiarm, multistage (MAMS) trials, their use in guiding trial modifications remains restricted.
Area of Science:
- Clinical Trial Design
- Health Economics
- Biostatistics
Background:
- Uncertainty exists regarding the value of integrating economic considerations into adaptive trial designs.
- Multiarm, multistage (MAMS) adaptive and adaptive platform trials offer complex frameworks for evaluating multiple interventions simultaneously.
Purpose of the Study:
- To systematically review analytical methods and factors for incorporating health economic analyses into MAMS adaptive and adaptive platform trials.
- To assess how economic considerations influence the design and modification of these advanced trial structures.
Main Methods:
- Systematic literature search across major databases (EMBASE, MEDLINE, Web of Science, Scopus, ClinicalTrials.gov) up to August 2023.
- Screening of articles by two blinded reviewers.
- Narrative synthesis of methods used in health economic analyses and their impact on trial design.
Main Results:
- Seventeen articles were included: 4 economic evaluations and 13 protocols.
- No trials reported using pre-trial economic evaluations to inform initial design.
- Interim cost and benefit estimations were possible in 14 articles, with 5 interim cost-effectiveness analyses, 3 of which informed decisions on trial arms.
Conclusions:
- Health economics is being incorporated into some MAMS adaptive and adaptive platform trials to guide modifications.
- The application of economic evidence in these trials is limited by design and circumstance, despite its potential value for decision-making.
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