Macabeo Bérengère, Quenéchdu Arthur, Aballéa Samuel, François Clément, Boyer Laurent, Laramée Philippe
Department of Public Health, Aix-Marseille University, 13005 Marseille, France.
Pierre Fabre Laboratories, 92100 Paris, France.
J Mark Access Health Policy. 2024 Apr 16;12(2):58-80. doi: 10.3390/jmahp12020006. eCollection 2024 Jun.
Health technology assessment (HTA) agencies express a clear preference for randomized controlled trials when assessing the comparative efficacy of two or more treatments. However, an indirect treatment comparison (ITC) is often necessary where a direct comparison is unavailable or, in some cases, not possible. Numerous ITC techniques are described in the literature. A systematic literature review (SLR) was conducted to identify all the relevant literature on existing ITC techniques, provide a comprehensive description of each technique and evaluate their strengths and limitations from an HTA perspective in order to develop guidance on the most appropriate method to use in different scenarios.
Electronic database searches of Embase and PubMed, as well as grey literature searches, were conducted on 15 November 2021. Eligible articles were peer-reviewed papers that specifically described the methods used for different ITC techniques and were written in English. The review was performed in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines.
A total of 73 articles were included in the SLR, reporting on seven different ITC techniques. All reported techniques were forms of adjusted ITC. Network meta-analysis (NMA) was the most frequently described technique (in 79.5% of the included articles), followed by matching-adjusted indirect comparison (MAIC) (30.1%), network meta-regression (24.7%), the Bucher method (23.3%), simulated treatment comparison (STC) (21.9%), propensity score matching (4.1%) and inverse probability of treatment weighting (4.1%). The appropriate choice of ITC technique is critical and should be based on the feasibility of a connected network, the evidence of heterogeneity between and within studies, the overall number of relevant studies and the availability of individual patient-level data (IPD). MAIC and STC were found to be common techniques in the case of single-arm studies, which are increasingly being conducted in oncology and rare diseases, whilst the Bucher method and NMA provide suitable options where no IPD is available.
ITCs can provide alternative evidence where direct comparative evidence may be missing. ITCs are currently considered by HTA agencies on a case-by-case basis; however, their acceptability remains low. Clearer international consensus and guidance on the methods to use for different ITC techniques is needed to improve the quality of ITCs submitted to HTA agencies. ITC techniques continue to evolve quickly, and more efficient techniques may become available in the future.
卫生技术评估(HTA)机构在评估两种或更多治疗方法的比较疗效时,明确倾向于采用随机对照试验。然而,在无法进行直接比较或在某些情况下不可能进行直接比较时,间接治疗比较(ITC)往往是必要的。文献中描述了多种ITC技术。进行了一项系统文献综述(SLR),以识别关于现有ITC技术的所有相关文献,全面描述每种技术,并从HTA角度评估其优势和局限性,以便为在不同场景中使用最合适的方法制定指南。
于2021年11月15日对Embase和PubMed进行电子数据库检索,以及灰色文献检索。符合条件的文章为专门描述不同ITC技术所用方法的同行评审论文,且为英文撰写。该综述按照系统评价和Meta分析的首选报告项目(PRISMA)指南进行。
SLR共纳入73篇文章,报道了七种不同的ITC技术。所有报道的技术均为调整后的ITC形式。网络Meta分析(NMA)是描述最频繁的技术(在所纳入文章的79.5%中),其次是匹配调整间接比较(MAIC)(30.1%)、网络Meta回归(24.7%)、布彻方法(23.3%)、模拟治疗比较(STC)(21.9%)、倾向得分匹配(4.1%)和治疗权重逆概率(4.1%)。ITC技术的恰当选择至关重要,应基于连通网络的可行性、研究间和研究内异质性的证据、相关研究的总数以及个体患者水平数据(IPD)的可用性。在单臂研究中,MAIC和STC是常用技术,单臂研究在肿瘤学和罕见病领域越来越普遍,而在没有IPD的情况下,布彻方法和NMA提供了合适的选择。
在可能缺少直接比较证据的情况下,ITC可以提供替代证据。目前HTA机构逐案考虑ITC;然而,其可接受性仍然较低。需要就不同ITC技术的使用方法达成更明确的国际共识和指南,以提高提交给HTA机构的ITC的质量。ITC技术继续快速发展,未来可能会有更高效的技术出现。