McEwin Eliza J, Hooimeyer Ashleigh, Mintzes Barbara J
Charles Perkins Centre and School of Pharmacy, Faculty of Medicine and Health, The University of Sydney, Sydney, New South Wales, Australia.
Pharmacoepidemiol Drug Saf. 2025 Jan;34(1):e70093. doi: 10.1002/pds.70093.
Cancer medicines usually have uncertain efficacy and safety profiles when they are first approved by medicines regulators because this evidence usually emerges post-market. Little is known about the extent to which post-market evidence is evaluated and integrated into evidence review processes in regulatory and clinical contexts.
The objective of this scoping review is to examine the literature on how post-market evidence on benefits and harms is evaluated and integrated in regulatory decisions and guidance for clinical decision-making.
This scoping review focussed on the organisations that review cancer medicines and post-market evidence for their benefits and harms. It examined all regulatory or clinical contexts in which this post-market evidence might be included in evidence review processes for evaluation then integration into regulatory or clinical contexts. Four electronic databases were searched. Titles and abstracts were screened for all retrieved references followed by full-text screening by two independent reviewers according to pre-specified inclusion criteria.
In total, 28 studies met inclusion criteria. These included 31 assessments by medicines regulators, four by clinical practice guideline developers and two by health technology assessment agencies. Half of the studies evaluated clinical outcomes for benefit or harms (e.g., overall survival, serious adverse events). We found that more published literature evaluated and integrated post-market evidence for benefits and harms of cancer medicines in regulatory than in clinical situations, such as treatment guidelines and health technology assessments. In these studies, post-market evidence for harms seemed to be integrated more often than for benefits. And the studies showed a gap: only some of the evaluated post-market evidence was subsequently integrated in both regulatory and clinical situations.
Overall, these findings raise important questions around the availability, accessibility, and assessment of post-market evidence for benefits and harms of cancer medicines so that it can be used by health professionals working in cancer services and by people with cancer.
癌症药物在首次获得药品监管机构批准时，其疗效和安全性通常是不确定的，因为这类证据通常是在上市后才出现。对于上市后证据在监管和临床环境中被评估并纳入证据审查流程的程度，人们了解甚少。
本范围综述的目的是研究关于癌症药物上市后益处和危害的证据如何在监管决策以及临床决策指导中进行评估和整合的文献。
本范围综述聚焦于审查癌症药物及其上市后益处和危害证据的组织。它考察了所有可能将此类上市后证据纳入证据审查流程以进行评估，然后整合到监管或临床环境中的监管或临床环境。检索了四个电子数据库。对所有检索到的参考文献进行标题和摘要筛选，随后由两名独立评审员根据预先指定的纳入标准进行全文筛选。
总共有28项研究符合纳入标准。其中包括药品监管机构进行的31项评估、临床实践指南制定者进行的4项评估以及卫生技术评估机构进行的2项评估。一半的研究评估了益处或危害的临床结局（例如总生存期、严重不良事件）。我们发现，与治疗指南和卫生技术评估等临床情况相比，更多已发表的文献在监管方面评估并整合了癌症药物上市后益处和危害的证据。在这些研究中，上市后危害证据似乎比益处证据更常被整合。并且研究显示存在一个差距：只有部分评估的上市后证据随后在监管和临床情况中都得到了整合。
总体而言，这些发现围绕癌症药物上市后益处和危害证据的可得性、可及性和评估提出了重要问题，以便癌症服务领域的卫生专业人员和癌症患者能够使用这些证据。
