The estimation of health state utility values in rare diseases: overview of existing techniques

M. Meregaglia; E. Nicod; Mike Drummond

doi:10.1017/S0266462320000665

The estimation of health state utility values in rare diseases: overview of existing techniques

M. Meregaglia, E. Nicod, Mike Drummond

Centre for Health Economics

Research output: Contribution to journal › Article › peer-review

Abstract

There are several techniques for estimating health state utility values, each of which presents pros and cons in the context of rare diseases (RDs). Direct approaches (e.g. standard gamble and time trade-off) may be too demanding for patients with RDs, since most of them affect young children or cause cognitive impairment. The alternatives are using “vignettes” that describe hypothetical health states for the general public, which may not reflect the heterogeneous manifestations of RDs, or multi-attribute utility instruments (i.e. indirect techniques), such as EQ-5D, which may be less sensitive in capturing the specificities of RDs. The “rule of rescue” approach is a promising alternative in RDs, since it prioritizes identifiable patients with life-threatening or disabling conditions. However, it raises measurement challenges and ethical issues. Furthermore, the literature reports on relevant implications of choosing a technique over others for health technology assessment, which should be considered in relation to individual RDs.

Original language	English
Pages (from-to)	469-473
Number of pages	5
Journal	International Journal of Technology Assessment in Health Care
Volume	36
Issue number	5
DOIs	https://doi.org/10.1017/S0266462320000665
Publication status	Published - 25 Sept 2020

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10.1017/S0266462320000665

Cite this

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abstract = "There are several techniques for estimating health state utility values, each of which presents pros and cons in the context of rare diseases (RDs). Direct approaches (e.g. standard gamble and time trade-off) may be too demanding for patients with RDs, since most of them affect young children or cause cognitive impairment. The alternatives are using “vignettes” that describe hypothetical health states for the general public, which may not reflect the heterogeneous manifestations of RDs, or multi-attribute utility instruments (i.e. indirect techniques), such as EQ-5D, which may be less sensitive in capturing the specificities of RDs. The “rule of rescue” approach is a promising alternative in RDs, since it prioritizes identifiable patients with life-threatening or disabling conditions. However, it raises measurement challenges and ethical issues. Furthermore, the literature reports on relevant implications of choosing a technique over others for health technology assessment, which should be considered in relation to individual RDs.",

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