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The Role of Condition-Specific Preference-Based Measures in Health Technology Assessment

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Abstract

A condition-specific preference-based measure (CSPBM) is a measure of health-related quality of life (HRQOL) that is specific to a certain condition or disease and that can be used to obtain the quality adjustment weight of the quality-adjusted life-year (QALY) for use in economic models. This article provides an overview of the role and the development of CSPBMs, and presents a description of existing CSPBMs in the literature. The article also provides an overview of the psychometric properties of CSPBMs in comparison with generic preference-based measures (generic PBMs), and considers the advantages and disadvantages of CSPBMs in comparison with generic PBMs. CSPBMs typically include dimensions that are important for that condition but may not be important across all patient groups. There are a large number of CSPBMs across a wide range of conditions, and these vary from covering a wide range of dimensions to more symptomatic or uni-dimensional measures. Psychometric evidence is limited but suggests that CSPBMs offer an advantage in more accurate measurement of milder health states. The mean change and standard deviation can differ for CSPBMs and generic PBMs, and this may impact on incremental cost-effectiveness ratios. CSPBMs have a useful role in HTA where a generic PBM is not appropriate, sensitive or responsive. However, due to issues of comparability across different patient groups and interventions, their usage in health technology assessment is often limited to conditions where it is inappropriate to use a generic PBM or sensitivity analyses.

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Fig. 1

Modified from Brazier et al. [1]

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Acknowledgements

The authors would like to thank Prof. Jon Karnon, PhD, of The University of Adelaide and Dr Andrew Lloyd, PhD, of Bladen Associates Ltd for their editorial review.

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Authors and Affiliations

  1. School of Health and Related Research (ScHARR), University of Sheffield, Regent Court, 30 Regent Street, Sheffield, S1 4DA, UK

    Donna Rowen, John Brazier & Roberta Ara

  2. Takeda Pharmaceuticals International AG, Thurgauerstrasse 130, 8152, Glattpark-Opfikon (Zurich), Switzerland

    Ismail Azzabi Zouraq

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  1. Donna Rowen
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  2. John Brazier
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  3. Roberta Ara
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  4. Ismail Azzabi Zouraq
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DR reviewed the literature and wrote the first draft of the manuscript and subsequent versions. JEB, RA and IA contributed to draft and final versions of the manuscript.

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Correspondence to Donna Rowen.

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Funding

This study was funded by an unrestricted grant from Takeda Pharmaceuticals International AG.

Conflict of interest

Ismail Azzabi Zouraq is employed by Takeda Pharmaceuticals International AG. Donna Rowen, John Brazier and Roberta Ara have no conflicts of interest.

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This article is published in a special edition journal supplement wholly funded by Takeda Pharmaceutical International AG, Zurich, Switzerland.

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Rowen, D., Brazier, J., Ara, R. et al. The Role of Condition-Specific Preference-Based Measures in Health Technology Assessment. PharmacoEconomics 35 (Suppl 1), 33–41 (2017). https://doi.org/10.1007/s40273-017-0546-9

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