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Model-Based Cost-Effectiveness Analyses for the Treatment of Chronic Lymphocytic Leukaemia: A Review of Methods to Model Disease Outcomes and Estimate Utility

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Abstract

Assessing the economic value of treatments for chronic lymphocytic leukaemia (CLL) is necessary to support healthcare decision makers; however, it poses a number of challenges. This paper reviews economic models of CLL treatment to learn the lessons from this experience and support ongoing model efforts. A search of databases and submissions to key health technology assessment agencies identified nine models. The modelling approaches adopted across these studies were fairly similar, with most models adopting a cohort Markov structure, though one example of a discrete event simulation was identified. While the cohort Markov approach has been acceptable to the National Institute for Health and Care Excellence, the review identifies a number of key uncertainties with these models, including the extrapolation of survival outcomes beyond the period observed by the trial, the effectiveness of second-line therapies, and estimates of health state utility. Further work is required to overcome these uncertainties, including comprehensive sensitivity analysis, systematic review of the evidence on the natural progression of CLL, and the collection of longer-term trial and registry data.

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Acknowledgments

This study was funded by Boehringer Ingelheim (BI) in Germany. This funding source had no role in study design, data collection, analysis, interpretation or writing of the report. The authors would like to thank Ciaran McCarron for his support on the contribution and coordination of the clinical input required for this study.

Conflicts of interest

The authors declare that they have no conflicts of interest to disclose regarding this study. Two of the authors (James Gordon and Ingolf Griebsch) are employed by Boehringer Ingelheim (BI). BI have a product for CLL in development but none on the market. The funding for the review was provided to Evidera by BI. Evidera regularly consult for BI on a range of health economics and outcomes research projects.

Author contributions

All authors were involved in the study concept and design, interpretation of data, and critical revision of the manuscript for important intellectual content. Kevin Marsh, Peng Xu and Panos Orfanos were responsible for the acquisition and analysis of data, and the drafting of the manuscript. Kevin Marsh supervised all the study and acts as guarantor for the content of the paper.

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Authors and Affiliations

  1. Evidera, Metro Building, 6th Floor, 1 Butterwick, W6 8DL, London, UK

    Kevin Marsh, Peng Xu & Panagiotis Orfanos

  2. Boehringer Ingelheim Pharma GmbH, Binger Str. 173, 55216, Ingelheim am Rhein, Germany

    James Gordon & Ingolf Griebsch

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  1. Kevin Marsh
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  2. Peng Xu
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  3. Panagiotis Orfanos
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Correspondence to Kevin Marsh.

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P. Xu was an Evidera employee until the submission of the manuscript.

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Marsh, K., Xu, P., Orfanos, P. et al. Model-Based Cost-Effectiveness Analyses for the Treatment of Chronic Lymphocytic Leukaemia: A Review of Methods to Model Disease Outcomes and Estimate Utility. PharmacoEconomics 32, 981–993 (2014). https://doi.org/10.1007/s40273-014-0187-1

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