Warwick Medical School, University of Warwick, Coventry, United Kingdom.
School of Pharmacy (ISPB)/UMR CNRS 5510 MATEIS/Edouard Herriot Hospital, Pharmacy Department, University of Lyon, Lyon, France.
PLoS One. 2020 Nov 9;15(11):e0241361. doi: 10.1371/journal.pone.0241361. eCollection 2020.
Two randomized controlled trials (RCTs), Mitra-Fr and Coapt, evaluating the benefit of percutaneous repair (PR) for heart failure (HF) patients with severe mitral regurgitation, have led to conflicting results. We aimed to evaluate the impact of these trial results on the cost-effectiveness of PR using effectiveness inputs from the two RCTs.
We developed a time varying Markov type model with three mutually exclusive health states: alive without HF hospitalisation, alive with HF hospitalisation, and dead. Clinically plausible extrapolations beyond observed data were obtained by developing parametric modelling for overall survival and HF hospitalisations using published data from each trial. We adopted the perspective of the French Health System and used a 30-year time horizon. Results were expressed as € / quality-adjusted life year (QALY) gained using utility inputs from literature.
Results are presented using treatment efficacy measures from Mitra-F and Coapt trials respectively. With the Mitra-Fr data, after annual discounting, the base case model generated an incremental 0.00387 QALY at a cost of €25,010, yielding an incremental cost effectiveness ratio (ICER) of €6,467,032 / QALY. The model was sensitive to changes made to model inputs. There was no potential of PR being cost-effective. With the Coapt data, the model generated 1.19 QALY gain at a cost of €26,130 yielding an ICER of €21,918 / QALY and at a threshold of >€50,000/QALY PR had a probability of 1 of being cost-effective.
Cost effectiveness results were conflicting; reconciling differences between trials is a priority and could promote optimal cost effectiveness analyses and resource allocation.
两项评估经皮修复（PR）对严重二尖瓣反流心力衰竭（HF）患者益处的随机对照试验（RCT），Mitra-Fr 和 Coapt，得出了相互矛盾的结果。我们旨在使用来自两项 RCT 的有效性数据来评估这些试验结果对 PR 的成本效益的影响。
我们开发了一个具有三个相互排斥的健康状态的时变马尔可夫模型：无 HF 住院的存活、HF 住院的存活和死亡。通过使用来自每个试验的已发表数据为总体生存和 HF 住院开发参数模型，对观察数据之外的临床合理推断进行了推断。我们采用了法国卫生系统的观点，并使用了 30 年的时间范围。结果使用文献中效用数据表示为每获得一个质量调整生命年（QALY）的欧元 (/QALY)。
使用 Mitra-F 和 Coapt 试验的治疗效果测量结果分别呈现结果。使用 Mitra-Fr 数据，每年折现后，基础模型产生了 0.00387 的增量 QALY，成本为 25,010 欧元，增量成本效益比（ICER）为 6,467,032 欧元/QALY。该模型对模型输入的变化很敏感。PR 没有潜在的成本效益。使用 Coapt 数据，该模型产生了 1.19 的 QALY 增益，成本为 26,130 欧元，ICER 为 21,918 欧元/QALY，在阈值 >50,000 欧元/QALY 时，PR 有 1 的概率具有成本效益。
成本效益结果相互矛盾；协调试验之间的差异是当务之急，这可以促进最佳的成本效益分析和资源分配。
