Centre for Health Economics, University of York, York, UK.
Pharmacoeconomics. 2012 Jan;30(1):35-46. doi: 10.2165/11594280-000000000-00000.
The National Institute for Health and Clinical Excellence (NICE) invited the manufacturer of dronedarone (Multaq®, Sanofi-Aventis Limited, UK) to submit evidence on the clinical and cost effectiveness of the anti-arrhythmic drug (AAD) for the treatment of atrial fibrillation (AF) and atrial flutter, as part of the Institute's single technology appraisal (STA) process. The Centre for Reviews and Dissemination and the Centre for Health Economics, both at the University of York, were commissioned to act as the independent Evidence Review Group (ERG). This article provides a description of the company submission, the ERG review and NICE's subsequent decisions regarding the use of dronedarone within the UK NHS. The ERG review comprised a critique of the submitted evidence on the clinical effectiveness and cost effectiveness of dronedarone. The ERG examined the search strategy used to obtain relevant evidence, the selection of studies included in the assessment, outcome measures chosen and statistical methods employed. The ERG also validated the manufacturer's decision analytic model and used it to explore the robustness of the cost-effectiveness results to key assumptions. The main clinical effectiveness evidence supporting the use of dronedarone as a treatment for AF came from four randomized controlled trials. These trials were compared with a broader set of trials examining the effectiveness of other AADs for AF: amiodarone, sotalol and class 1c agents (flecainide and propafenone). The evidence suggested that all AADs decreased the recurrence of AF but dronedarone had the smallest effect. A mixed treatment comparison analysis of the trials showed that dronedarone was associated with a lower risk of all-cause mortality than other AADs, but this was highly uncertain. There was limited evidence to assess the effect of dronedarone on stroke, and no statistically significant differences between dronedarone and other AADs were found for treatment discontinuation. From the evidence presented by the manufacturer, dronedarone appeared highly cost effective in each of the population groups examined compared with using standard baseline therapy alone as first-line treatment, or compared with sotalol or amiodarone as first-line AAD, with incremental cost-effectiveness ratios (ICERs) well below £20,000 per QALY gained. The ICER for dronedarone relative to class 1c agents was around £19,000 per QALY. Although the evidence presented by the manufacturer indicated that dronedarone was cost effective, the estimates of treatment effect relative to other AADs and safety in the longer term were highly uncertain. The NICE Appraisal Committee in its preliminary guidance did not recommend the use of dronedarone for AF. However, following the response from a large number of consultees and commentators, NICE revised its preliminary guidance to allow the use of the drug in a specific subgroup of AF patients with additional cardiovascular risk factors.
国家卫生与临床优化研究所(NICE)邀请多非利特(Multaq ® ,赛诺菲-安万特英国有限公司)的制造商提交有关抗心律失常药物(AAD)治疗心房颤动(AF)和心房扑动的临床和成本效益的证据,作为研究所单一技术评估(STA)过程的一部分。约克大学的评论与传播中心和卫生经济学中心受委托作为独立的证据审查小组(ERG)。本文介绍了公司提交的内容、ERG 审查以及 NICE 随后对多非利特在英国国民保健制度中的使用做出的决定。ERG 审查包括对多非利特临床疗效和成本效益的提交证据进行批判性评估。ERG 检查了用于获取相关证据的搜索策略、评估中纳入的研究选择、选择的结果衡量标准和使用的统计方法。ERG 还验证了制造商的决策分析模型,并使用该模型探索了关键假设对成本效益结果的稳健性。支持多非利特作为 AF 治疗药物的主要临床疗效证据来自四项随机对照试验。这些试验与一组更广泛的试验进行了比较,这些试验评估了其他 AAD 对 AF 的有效性:胺碘酮、索他洛尔和 1c 类药物(氟卡尼和普罗帕酮)。证据表明,所有 AAD 都降低了 AF 的复发率,但多非利特的效果最小。对试验的混合治疗比较分析表明,与其他 AAD 相比,多非利特与全因死亡率降低相关,但这一结果高度不确定。评估多非利特对中风影响的证据有限,也没有发现多非利特与其他 AAD 相比在停药方面存在统计学上的显著差异。根据制造商提交的证据,与单独使用标准基线治疗作为一线治疗相比,与索他洛尔或胺碘酮作为一线 AAD 相比,多非利特在每个检查的人群中都具有很高的成本效益,增量成本效益比(ICER)远低于每获得 1 个质量调整生命年(QALY) 20,000 英镑以下。多非利特与 1c 类药物的 ICER 约为每获得 1 个 QALY 19,000 英镑。尽管制造商提交的证据表明多非利特具有成本效益,但相对于其他 AAD 的治疗效果和长期安全性的估计高度不确定。NICE 评估委员会在其初步指导意见中不建议将多非利特用于 AF。然而,在收到大量顾问和评论员的回应后,NICE 修改了其初步指导意见,允许在具有额外心血管风险因素的 AF 患者的特定亚组中使用该药物。