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Simulation and Matching-Based Approaches for Indirect Comparison of Treatments

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Abstract

Estimates of the relative effects of competing treatments are rarely available from head-to-head trials. These effects must therefore be derived from indirect comparisons of results from different studies. The feasibility of comparisons relies on the network linking treatments through common comparators; the reliability of these may also be impacted when the studies are heterogeneous or when multiple intermediate comparisons are needed to link two specific treatments of interest. Simulated treatment comparison and matching-adjusted indirect comparison have been developed to address these challenges. These focus on comparisons of outcomes for two specific treatments of interest by using patient-level data for one treatment (the index) and published results for the other treatment (the comparator) from compatible studies, taking into account possible confounding due to population differences. This paper provides an overview of how and when these approaches can be used as an alternative or to complement standard MTC approaches.

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Notes

  1. We use the Y(X) notation loosely here to indicate that the response or outcome variable is some function of predictors. We do not intend to imply any specific form or relationship. These are made explicit where needed in the text.

  2. Parameterization used in statistical software can vary. The formula provided here is based on the general form of the Weibull distribution. Analysts should verify the formulation used in the software used to perform the analyses to ensure correct calculation of the HR.

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Acknowledgments

K. Jack Ishak, Irina Proskorovsky, and Agnes Benedict are all employees of Evidera, which provides consulting and other research services to pharmaceutical, device, government, and non-government organizations. In their salaried positions, they work with a variety of companies and organizations and are precluded from receiving payment or honoraria directly from these organizations for services rendered. This manuscript was discussed, written, and edited during their standard work hours and they received their standard salaries from Evidera. Dr. Ishak led the conceptual development, drafting, and review/editing of the paper. Ms. Proskorovsky and Ms. Benedict participated in the conceptual development, drafting, and review/editing of the paper. Dr Ishak will act as the overall guarantor of this work.

The authors wish to thank Connie Chen who provided insights into the applicability of the STC and MAIC methods for indirect comparison in practical situations.

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Correspondence to K. Jack Ishak.

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Ishak, K.J., Proskorovsky, I. & Benedict, A. Simulation and Matching-Based Approaches for Indirect Comparison of Treatments. PharmacoEconomics 33, 537–549 (2015). https://doi.org/10.1007/s40273-015-0271-1

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