背景:卫生技术评估(HTA)机构在评估两种或更多种治疗方法的比较疗效时,对随机对照试验表现出明显的偏好。然而,间接治疗比较(ITC)通常是必要的,如果直接比较是不可用的,或者,在某些情况下,不可能。文献中描述了许多ITC技术。进行了系统的文献综述(SLR),以确定有关现有ITC技术的所有相关文献,提供每种技术的全面描述,并从HTA的角度评估它们的优势和局限性,以便为在不同场景中使用的最合适的方法制定指导。
方法:Embase和PubMed的电子数据库搜索,以及灰色文献搜索,于2021年11月15日进行。符合条件的文章是经过同行评审的论文,这些论文专门描述了用于不同ITC技术的方法,并用英语编写。审查是根据系统审查和荟萃分析(PRISMA)指南的首选报告项目进行的。
结果:SLR共收录了73篇文章,报告七种不同的ITC技术。所有报道的技术都是经调整的ITC形式。网络荟萃分析(NMA)是描述最频繁的技术(在79.5%的文章中),其次是匹配调整间接比较(MAIC)(30.1%),网络元回归(24.7%),Bucher方法(23.3%),模拟处理比较(STC)(21.9%),倾向评分匹配(4.1%)和治疗加权的逆概率(4.1%).ITC技术的适当选择至关重要,应基于连接网络的可行性,研究之间和研究内异质性的证据,相关研究的总数和个体患者水平数据(IPD)的可用性。MAIC和STC被发现是单臂研究的常见技术,越来越多地在肿瘤学和罕见疾病中进行,而Bucher方法和NMA在没有IPD可用的情况下提供合适的选项。
结论:ITC可以提供替代证据,其中可能缺少直接的比较证据。ITC目前由HTA机构逐案审议;然而,他们的可接受性仍然很低。需要就不同ITC技术的使用方法达成更清晰的国际共识和指导,以提高提交给HTA机构的ITC的质量。ITC技术继续快速发展,和更有效的技术可能会在未来变得可用。
BACKGROUND: 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.
METHODS: 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.
RESULTS: 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.
CONCLUSIONS: 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.