A health state utility valuation study to assess the impact of treatment mode of administration in Gaucher disease
- PDF / 570,791 Bytes
- 7 Pages / 595.276 x 790.866 pts Page_size
- 48 Downloads / 130 Views
RESEARCH
Open Access
A health state utility valuation study to assess the impact of treatment mode of administration in Gaucher disease Monica Hadi1, Paul Swinburn1, Luba Nalysnyk2*, Alaa Hamed2 and Atul Mehta3
Abstract Background: 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. Methods: 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). Results: 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). Conclusions: 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. Keywords: Utility, United Kingdom, Gaucher disease, Treatment mode of administration
Background Over the past decades, treatments with similar efficacy have been developed in several indications with the main differences being in the mode of administration, intravenous infusion, subcutaneous injection, oral intake [1, 2]. Gaucher disease, a rare, genetic lysosomal storage disorder caused by a deficiency in the enzyme acid beta-glucosidase, is an example of a disease for which treatment exists with different modes of administration; intravenously-administered enzyme replacement therapy and orally-administered substrate reduction therapy. Gaucher disease affects patients of all ages and is one of the most common lysosomal storage diseases with a worldwide prevalence of approximately 1/ * Correspondence: [email protected] 2 Sanofi Genzyme, 50 Binney Street, Cambridge, MA 02142, USA Full list of author information is available at the end of the article
100,000 and approximately 1/855 in the Ashkenazi Jewish population [3, 4]. There are three
Data Loading...
