Journal article
Understanding Treatment Effect Estimates When Treatment Effects Are Heterogeneous for More Than One Outcome
Applied health economics and health policy, Vol.16(3), pp.381-393
06/2018
DOI: 10.1007/s40258-018-0380-z
PMCID: PMC6437676
PMID: 29589296
Abstract
Patient-centred care requires evidence of treatment effects across many outcomes. Outcomes can be beneficial (e.g. increased survival or cure rates) or detrimental (e.g. adverse events, pain associated with treatment, treatment costs, time required for treatment). Treatment effects may also be heterogeneous across outcomes and across patients. Randomized controlled trials are usually insufficient to supply evidence across outcomes. Observational data analysis is an alternative, with the caveat that the treatments observed are choices. Real-world treatment choice often involves complex assessment of expected effects across the array of outcomes. Failure to account for this complexity when interpreting treatment effect estimates could lead to clinical and policy mistakes.
Our objective was to assess the properties of treatment effect estimates based on choice when treatments have heterogeneous effects on both beneficial and detrimental outcomes across patients.
Simulation methods were used to highlight the sensitivity of treatment effect estimates to the distributions of treatment effects across patients across outcomes. Scenarios with alternative correlations between benefit and detriment treatment effects across patients were used. Regression and instrumental variable estimators were applied to the simulated data for both outcomes.
True treatment effect parameters are sensitive to the relationships of treatment effectiveness across outcomes in each study population. In each simulation scenario, treatment effect estimate interpretations for each outcome are aligned with results shown previously in single outcome models, but these estimates vary across simulated populations with the correlations of treatment effects across patients across outcomes.
If estimator assumptions are valid, estimates across outcomes can be used to assess the optimality of treatment rates in a study population. However, because true treatment effect parameters are sensitive to correlations of treatment effects across outcomes, decision makers should be cautious about generalizing estimates to other populations.
Details
- Title: Subtitle
- Understanding Treatment Effect Estimates When Treatment Effects Are Heterogeneous for More Than One Outcome
- Creators
- John M Brooks - University of South CarolinaCole G Chapman - University of South CarolinaMary C Schroeder - University of Iowa
- Resource Type
- Journal article
- Publication Details
- Applied health economics and health policy, Vol.16(3), pp.381-393
- DOI
- 10.1007/s40258-018-0380-z
- PMID
- 29589296
- PMCID
- PMC6437676
- NLM abbreviation
- Appl Health Econ Health Policy
- ISSN
- 1175-5652
- eISSN
- 1179-1896
- Grant note
- RC4 AG038635 / NIA NIH HHS ME-1303-6011 / Patient-Centered Outcomes Research Institute
- Language
- English
- Date published
- 06/2018
- Academic Unit
- Pharmacy Practice and Science
- Record Identifier
- 9984366025302771
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