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How to Select the Right Cost-Effectiveness Model?

A Systematic Review and Stepwise Approach for Selecting a Transferable Health Economic Evaluation Model for Rheumatoid Arthritis

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Abstract

Objective

In the current study, we propose an approach for selection of a model that is transferable to a specific decision-making context (in this case, the Netherlands), using the case of rheumatoid arthritis (RA). The objectives of this study were (a) to perform a systematic literature review to identify existing health economic evaluation models for economic evaluation of disease-modifying antirheumatic drugs (DMARDs) in RA; and (b) to test the appropriateness of a stepwise model-selection process.

Methods

First, we searched Medline and Embase to identify relevant studies in the English language, published between 1 January 2002 and 31 August 2012. From the included studies, all unique models were identified. Second, we applied a multi-step approach to model selection. Models that did not meet all minimal methodological and structural requirements based on the Outcome Measures in Rheumatology (OMERACT) criteria were excluded. Next, models were assessed on the basis of their fit when transferred to the Dutch health care setting. The criteria for model fit were transferability factors, as published by Welte et al., after exclusion of those that were deemed transferable by simple adaptation. Finally, the remaining models underwent a general quality check using the Philips checklist. Models showing good fit and high quality were considered to be transferable to the Dutch health care setting, using simple adaptation.

Results

The systematic literature search identified 498 articles, which included 33 unique health economic evaluation models. Only six models passed the minimal methodological and structural requirements. Two of these models had an imperfect transferability fit to the Dutch health care setting, according to the Welte method. The remaining four models were, according to the Philips method, of good quality and were expected to be transferable by a simple adaptation.

Conclusion

This study introduces a stepwise approach for selecting health economic evaluation models that are transferable by a simple adaptation. The approach seems feasible and can be applied in various therapeutic areas, provided that the minimal methodological and structural requirements are defined accordingly. Availability of health economic evaluation models coupled with structured model selection could improve the efficiency, quality and comparability of health economic research.

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Notes

  1. Some models use survival statistics based on observational data to predict the subjects’ time on treatment. We also considered this a suitable approach for modelling health state transitions (i.e. treatment discontinuation).

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Disclosures

The author contributions to this manuscript were as follows:

HvH: study rationale and design, execution of systematic literature search, study selection, execution of stepwise selection, interpretation and reflection, writing of the manuscript.

JS: study rationale and design, study selection, verification of stepwise selection, interpretation and reflection, review of the manuscript, overall guarantor of the study.

ATD: study selection, feedback on study execution and interpretation, review of the manuscript.

AB: feedback on study execution and interpretation, review of the manuscript.

HvH is employed by AstraZeneca and affiliated with the Erasmus University Rotterdam Institute of Health Policy & Management by means of a PhD hospitality agreement. JS’s supervision of HvH is compensated by AstraZeneca on the basis of an unrestricted supervision grant to his institution. In the field of rheumatoid arthritis, JS has received research grants from Pfizer. AB has received research grants from Merck, AbbVie and Amgen. HvH, JS, ATD and AB declare no conflict of interest regarding the topics studied and the results presented in this publication.

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Authors and Affiliations

  1. Institute of Health Policy and Management, Erasmus University Rotterdam, Burgemeester Oudlaan 50, 3062 PA, Rotterdam, The Netherlands

    H. G. M. van Haalen & J. L. Severens

  2. AstraZeneca, Zoetermeer, The Netherlands

    H. G. M. van Haalen

  3. Division of Rheumatology, Department of Internal Medicine, Maastricht University Medical Center and Caphri School for Public Health and Primary Care, P. Debyelaan 25, 6229 HX, Maastricht, The Netherlands

    A. Tran-Duy & A. Boonen

  4. Department of Clinical Epidemiology and Medical Technology Assessment, Maastricht University Medical Center, Maastricht, The Netherlands

    A. Tran-Duy

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  1. H. G. M. van Haalen
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  3. A. Tran-Duy
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van Haalen, H.G.M., Severens, J.L., Tran-Duy, A. et al. How to Select the Right Cost-Effectiveness Model?. PharmacoEconomics 32, 429–442 (2014). https://doi.org/10.1007/s40273-014-0139-9

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