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

Abstract

BackgroundThis 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.MethodsA 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).ResultsMean 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).ConclusionsThe 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.Electronic supplementary materialThe online version of this article (10.1186/s13023-018-0903-6) contains supplementary material, which is available to authorized users.

Quality of life and psychological functioning of pediatric and young adult patients with Gaucher disease, type 1

“…Of importance, over half of our young adult sample was taking oral eliglustat at the time of data collection, while in earlier studies participants were exclusively treated with ERT. This distinction is noteworthy since recent research has suggested that treatment with oral eliglustat offers important advantages for quality of life (e.g., Hadi et al, ; Sechi et al, ). In our study, young adults who were taking eliglustat described improvement in their symptoms and their quality of life.…”

“…Treatment with ERT has been associated with significant symptom reduction and improvement in HRQoL across a variety of domains. Some researchers have suggested that treatment with SRT offers benefits for quality of life relative to ERT due to greater convenience associated with oral versus intravenous administration (e.g., Elstein et al, ; Hadi, Swinburn, Nalysnyk, Hamed, & Mehta, ; Sechi, Dardis, & Bembi, ). Despite treatment however, patients with GD1 report persistent difficulty coping with the diagnosis, adverse effects of pain on daily activities, mood changes and anxiety, and a negative outlook on life (Packman, Crosbie, Behnken, Eudy, & Packman, ).…”

Vignette-Based Utilities: Usefulness, Limitations, and Methodological Recommendations

“…45,46,48,61 As with AEs, the vignette approach can isolate the utility impact of a specific treatment process attribute by holding all aspects of a health state constant except for this attribute. Process utilities estimated with vignettes have been included in CUAs conducted for HTA submissions, 38,41,62 and favorable HTA impressions of costeffectiveness suggest that these process utilities can be useful model inputs. [63][64][65][66]…”

Increasing respondent engagement in composite time trade-off tasks by imposing three minimum trade-offs to improve data quality

“…The EQ-5D-5L is a measure of health that covers five dimensions-mobility, self-care, usual activities, pain/discomfort, and anxiety/depression-using five levels of severity-no, slight, moderate, severe, and extreme problems/unable to [16]. The measure describes 3125 health states (5 5 = 3125) and is used across many healthcare settings. A misery score can be calculated by summing the dimension-level responses for a crude estimate of health state severity ranging from 5 to 25.…”

“…The TTO is often used to elicit preferences for health states in the development of value sets for measures of health like the EQ-5D [2,3]. Because the TTO can be difficult to understand, valuation studies using the TTO have historically been conducted using resourceand time-intensive face-to-face studies to ensure respondent comprehension [4,5].…”