Patient-Centered Outcomes, Mapi, London, UK.
Sanofi Genzyme, 50 Binney Street, Cambridge, MA, 02142, USA.
Orphanet J Rare Dis. 2018 Sep 10;13(1):159. doi: 10.1186/s13023-018-0903-6.
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.
本研究旨在获得与治疗方式相关的戈谢氏病健康状态的英国基于社会的效用值。
对相关文献的回顾和专家临床意见为五种健康状态的发展提供了信息，这些健康状态描述了戈谢氏病及其管理对患者生活的影响。还制定了一个基础状态来描述“控制疾病”，以及随后的四个健康状态，这些状态在描述方法（静脉内与口服）和治疗给药频率方面有所不同。使用时间权衡（TTO）方法通过对 100 名英国普通人群进行面对面访谈获得健康状态效用值。在估值练习之前，参与者提供了知情同意书，完成了人口统计学表格和 EQ-5D，并在 0-100 视觉模拟量表（VAS）上对健康状态进行了从最佳到最差的排名。
参与者（n=100）的平均年龄为 35 岁，66%为女性。参与者报告了很高的 EQ-5D VAS（86.1）和指数得分（0.95），表明他们的健康状况非常好。“控制疾病”状态的 TTO 衍生效用值最高（0.89）。对于“口服治疗”的通用状态，效用值仅略有下降（0.85），而对于“静脉内治疗”的通用状态，效用值下降更为明显（0.73）。
研究结果表明，避免需要静脉内治疗管理与显著提高健康相关生活质量相关。当对未来戈谢氏病治疗的经济评估时，治疗方法的侵入性较小带来的患者获益可能是一个重要的考虑因素。
