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Falsification Testing of Instrumental Variables Methods for Comparative Effectiveness Research

Keywords: Comparative effectiveness research;instrumental variables;falsification testing;big data;causal inference

Objectives: To demonstrate how falsification tests can be used to evaluate instrumental variables methods applicable to a wide variety of comparative effectiveness research questions.

Study Design: Brief conceptual review of instrumental variables and falsification testing principles and techniques accompanied by an empirical application. Sample STATA code related to the empirical application is provided in the Appendix.

Empirical Application: Comparative long-term risks of sulfonylureas and thiazolidinediones for management of type 2 diabetes. Outcomes include mortality and hospitalization for an ambulatory care–sensitive condition. Prescribing pattern variations are used as instrumental variables.

Conclusions: Falsification testing is an easily computed and powerful way to evaluate the validity of the key assumption underlying instrumental variables analysis. If falsification tests are used, instrumental variables techniques can help answer a multitude of important clinical questions.

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