■这项研究的目的是评估一种疾病模型的表面有效性,该模型评估了ataluren治疗无义突变Duchenne肌营养不良(nmDMD)的成本效益。
■这是一项Delphi小组研究,由具有治疗nmDMD第一手经验的医生组成。对以前未经验证的模型数据寻求共识,包括使用健康效用指数(HUI)测量的患者健康状况和生活质量,死亡率,非正式护理,以及四个州的早期ataluren治疗的预期益处:(1)门诊,(2)非门诊,还不需要通风支持,(3)非卧床,夜间通风支持,(4)非卧床,全时通风支持。
■来自五个国家的九位专家参加了德尔菲小组。经过三轮小组讨论,所有问题都获得了共识(除了两个关于手功能[灵巧]的HUI问题)。州(1)的共识HUI衍生公用事业在最佳支持性护理(BSC)之上是ataluren的1.0000,仅BSC的0.7337。状态(2)的相应估计值为0.3179和0.2672,状态(3)为0.1643和0.0913,状态(4)为-0.0732和-0.1163。各州的共识死亡率(1),(2),(3)为4%,13%,33%,州(4)的预期寿命被同意为3年。小组成员进一步同意,两名非正式护理人员通常为nmDMD患者提供日常护理/支持。并且在2岁和5岁时开始使用Ataluren进行治疗,预计将使步行丧失延迟额外的2年,并开始额外3年的夜间和全职通风支持,分别。
■主要限制涉及Delphi面板的大小,主要由疾病的稀有性支配。
■这项研究证实了ataluren成本效益模型基础的关键临床参数和假设的表面有效性。
UNASSIGNED: The objective of this
study was to assess the face validity of a disease model evaluating the cost-effectiveness of ataluren for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
UNASSIGNED: This was a Delphi panel
study comprising of physicians with first-hand experience of ataluren for the treatment of nmDMD. Consensus was sought for previously unvalidated model data, including patient health status and quality of life measured using the Health Utility Index (HUI), mortality, informal caregiving, and the expected benefit of early ataluren treatment across four states: (1) ambulatory, (2) non-ambulatory, not yet requiring ventilation support, (3) non-ambulatory, night-time ventilation support, and (4) non-ambulatory, full-time ventilation support.
UNASSIGNED: Nine experts from five countries participated in the Delphi panel. Consensus was obtained for all questions after three panel rounds (except for two HUI-questions concerning hand function [dexterity]). Consensus HUI-derived utilities for state (1) were 1.0000 for ataluren on top of best supportive care (BSC) and 0.7337 for BSC alone. Corresponding estimates for state (2) were 0.3179 and 0.2672, for state (3) 0.1643 and 0.0913, and for state (4) -0.0732 and -0.1163. Consensus mortality rates for states (1), (2), and (3) were 4%, 13%, and 33%, and life expectancy in state (4) was agreed to be 3 years. Panelists further agreed that two informal caregivers typically provide day-to-day care/support to patients with nmDMD, and that starting treatment with ataluren at 2 versus 5 years of age would be expected to delay loss of ambulation by an additional 2 years, and initiation of night-time and full-time ventilation support by an additional 3 years, respectively.
UNASSIGNED: The main limitation concerns the size of the Delphi panel, govern primarily by the rarity of the disease.
UNASSIGNED: This
study confirms the face validity of key clinical parameters and assumptions underlying the ataluren cost-effectiveness model.