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01.12.2018 | Research | Ausgabe 1/2018 Open Access

Orphanet Journal of Rare Diseases 1/2018

A health state utility valuation study to assess the impact of treatment mode of administration in Gaucher disease

Orphanet Journal of Rare Diseases > Ausgabe 1/2018
Monica Hadi, Paul Swinburn, Luba Nalysnyk, Alaa Hamed, Atul Mehta
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Electronic supplementary material

The online version of this article (https://​doi.​org/​10.​1186/​s13023-018-0903-6) contains supplementary material, which is available to authorized users.



This study aimed to obtain UK societal-based utility values for health states related to treatment mode of administration using Gaucher disease as the background condition.


A review of relevant literature and expert clinical input informed the development of five health states characterising the impact of Gaucher disease and its management on patients’ lives. A base-state characterising the “controlled disease” was developed as well as four subsequent health states which varied in description of the method (intravenous versus oral) and frequency of treatment administration. Health state utilities were obtained using the time trade-off (TTO) method via face-to-face interviews with 100 members from the UK general population. Before the valuation exercise, participants provided informed consent, completed a demographic form and the EQ-5D, and ranked the health states from best to worst on a 0–100 visual analogue scale (VAS).


Mean age of the participants (n = 100) was 35 years and 66% were female. Participants reported high EQ-5D VAS (86.1) and index scores (0.95) indicating very good health status. The “controlled disease” state had the highest mean TTO-derived utility value (0.89). There was only a marginal reduction in utility for the generic state for “Oral treatment” (0.85), while the reduction was more pronounced for the generic state for “Intravenous treatment” (0.73).


The findings suggest that the avoidance of the need for intravenous treatment administration is associated with a notable positive increase in health-related quality of life. Patient benefit arising from less invasive treatment could be an important consideration when undertaking economic evaluation of future therapies for Gaucher disease.
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