Wolfson Lara J, Walker Anna, Hettle Robert, Lu Xiaoyan, Kambili Chrispin, Murungi Andrew, Knerer Gerhart
Janssen Pharmaceutica NV, Beerse, Belgium.
HERON Commercialization, London, United Kingdom.
PLoS One. 2015 Mar 20;10(3):e0120763. doi: 10.1371/journal.pone.0120763. eCollection 2015.
To evaluate the cost-effectiveness of adding bedaquiline to a background regimen (BR) of drugs for multidrug-resistant tuberculosis (MDR-TB) in the United Kingdom (UK).
A cohort-based Markov model was developed to estimate the incremental cost-effectiveness ratio of bedaquiline plus BR (BBR) versus BR alone (BR) in the treatment of MDR-TB, over a 10-year time horizon. A National Health Service (NHS) and personal social services perspective was considered. Cost-effectiveness was evaluated in terms of Quality-Adjusted Life Years (QALYs) and Disability-Adjusted Life Years (DALYs). Data were sourced from a phase II, placebo-controlled trial, NHS reference costs, and the literature; the US list price of bedaquiline was used and converted to pounds (£18,800). Costs and effectiveness were discounted at a rate of 3.5% per annum. Probabilistic and deterministic sensitivity analysis was conducted.
The total discounted cost per patient (pp) on BBR was £106,487, compared with £117,922 for BR. The total discounted QALYs pp were 5.16 for BBR and 4.01 for BR. The addition of bedaquiline to a BR resulted in a cost-saving of £11,434 and an additional 1.14 QALYs pp over a 10-year period, and is therefore considered to be the dominant (less costly and more effective) strategy over BR. BBR remained dominant in the majority of sensitivity analyses, with a 81% probability of being dominant versus BR in the probabilistic analysis.
In the UK, bedaquiline is likely to be cost-effective and cost-saving, compared with the current MDR-TB standard of care under a range of scenarios. Cost-savings over a 10-year period were realized from reductions in length of hospitalization, which offset the bedaquiline drug costs. The cost-benefit conclusions held after several sensitivity analyses, thus validating assumptions made, and suggesting that the results would hold even if the actual price of bedaquiline in the UK were higher than in the US.
评估在英国，将贝达喹啉添加到耐多药结核病（MDR-TB）背景治疗方案（BR）中的成本效益。
建立了一个基于队列的马尔可夫模型，以估计在10年时间范围内，贝达喹啉加BR（BBR）与单独使用BR治疗MDR-TB的增量成本效益比。采用了英国国家医疗服务体系（NHS）和个人社会服务的视角。从质量调整生命年（QALYs）和伤残调整生命年（DALYs）方面评估成本效益。数据来源于一项II期安慰剂对照试验、NHS参考成本以及文献；使用了贝达喹啉的美国标价并换算为英镑（18,800英镑）。成本和效益按每年3.5%的贴现率进行贴现。进行了概率性和确定性敏感性分析。
BBR方案下每位患者的总贴现成本为106,487英镑，而BR方案为117,922英镑。BBR方案下每位患者的总贴现QALYs为5.16，BR方案为4.01。在BR基础上加用贝达喹啉在10年期间可节省成本11,434英镑，且每位患者可额外获得1.14个QALYs，因此被认为是优于BR的主导策略（成本更低且效果更佳）。在大多数敏感性分析中，BBR仍为主导策略，在概率分析中相对于BR占优的概率为81%。
在英国，与当前耐多药结核病的标准治疗方案相比，在一系列情景下，贝达喹啉可能具有成本效益且能节省成本。10年期间因住院时间缩短实现了成本节省，抵消了贝达喹啉的药物成本。经过多次敏感性分析后，成本效益结论依然成立，从而验证了所作假设，并表明即使贝达喹啉在英国的实际价格高于美国，结果依然成立。
