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An intuitive review of methods for observational studies of comparative effectiveness

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Abstract

I use diagrams to illustrate the sources of potential selection bias in observational studies of comparative effectiveness. I adapt these diagrams for three hypothetical scenarios that clarify the strengths and weaknesses of two prominent methods used to account for potential selection bias: propensity scores and instrumental variables. After reviewing the fundamentals of how to apply each method, including new developments that make implementation easier, I refer to some recent studies that illustrate how choice of method can affect estimates. I conclude by emphasizing that many studies with apparently rich sources of data are nevertheless unlikely to produce unbiased estimates and that conceptual modeling can help identify these problems in advance.

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Acknowledgements

This research was supported by Grant Number IAD 06-112 from the Health Services Research and Development Service of the U.S. Department of Veterans Affairs. All opinions expressed in this paper are those of the author and do not necessarily reflect the official position of the U.S. Department of Veterans Affairs or of Boston University. The author wishes to thank Matt Maciejewski, Paul Hebert, Ann Hendricks, Austin Frakt, and an anonymous reviewer for helpful comments.

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Correspondence to Steven D. Pizer.

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Pizer, S.D. An intuitive review of methods for observational studies of comparative effectiveness. Health Serv Outcomes Res Method 9, 54–68 (2009). https://doi.org/10.1007/s10742-009-0045-3

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