ESTIMATING THE UTILITY ASSOCIATED WITH MODE OF TREATMENT ADMINISTRATION IN GAUCHER DISEASE

Author(s)

Hadi M1, Nalysnyk L2, Hamed A2, Swinburn P1
1Mapi Group, London, UK, 2Genzyme, a Sanofi Company, Cambridge, MA, USA

OBJECTIVES: A Phase 3 trial of imiglucerase and eliglustat for Gaucher disease type 1 demonstrated comparable efficacy. However, the differences that exist in their mode of administration may have important considerations in terms of impact on health-related quality of life (HRQL). The study aim was to capture UK societal-based utility values for health states related to treatment mode of administration for Gaucher disease using a Time Trade-Off (TTO) approach. METHODS:  A review of relevant literature and expert clinical input informed the development of five health states. These varied in description of the method and frequency of treatment administration. Members of the UK general public (n=6) took part in pilot interviews to ensure the comprehension and suitability of the health states for use in a valuation exercise. The valuation study was conducted face-to-face by trained interviewers with members of the UK general public who provided informed consent. Participants were asked to complete a brief socio-demographic form, the EQ-5D and then participate in a visual analog scale (VAS) ranking exercise and TTO interview. RESULTS: Mean age of the study participants (n=100) was 35 years, 66% were female, 71% had degree or postgraduate-level education, 66% were employed full-time and 69% were white. Participants reported high VAS (86.13) and EQ-5D index scores (0.95) indicating very good health status. Data revealed that varying mode of administration had a significant impact on health-related quality of life (HRQL) with the “oral treatment” state (VAS 70.9; utility 0.85) valued more positively than the “intravenous treatment” state (VAS 49.9; utility 0.73) suggesting reduced burden for patients. CONCLUSIONS: The findings suggest that the avoidance of the need for intravenous treatment administration is associated with a significant positive increase in HRQL. Patient benefit arising from less invasive treatment could be an important consideration when undertaking economic evaluation of future therapies for Gaucher disease. 

Conference/Value in Health Info

2016-10, ISPOR Europe 2016, Vienna, Austria

Value in Health, Vol. 19, No. 7 (November 2016)

Code

PSY102

Topic

Patient-Centered Research

Topic Subcategory

Health State Utilities

Disease

Rare and Orphan Diseases

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