Saiyed M M, Ong P S, Chew L
Department of Pharmacy, Faculty of Science, National University Singapore, Singapore.
J Clin Pharm Ther. 2017 Jun;42(3):251-258. doi: 10.1111/jcpt.12507. Epub 2017 Feb 5.
The off-label use of medicines is widespread in several diseases. This type of prescribing practice is particularly more acute in oncology. However, the suitability of anticancer medications for off-label use remains an issue of controversy, due to uncertainty around the clinical benefits and potential toxicities, limited evidence to support clinical decision-making, increased out-of-pocket costs for patients and ethical concerns around the lack of informed consent. Currently, data pertaining to the global prevalence of off-label use in cancer therapy are lacking. The aim of this review was to provide an overview of off-label drug use prevalence in oncology.
A systematic literature search was performed in PubMed as per the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines from 1975 to 2016. Studies assessing the prevalence of off-label use of anticancer drugs were included.
Of the 199 eligible papers retrieved, 23 studies were included in this systematic literature review. Off-label drug use in inpatients ranged from 18% to 41%. Among adult patients with cancer, 13%-71% received a minimum of one off-label chemotherapy. The main reasons for off-label drug use were 'drug unapproved for specific tumour' and 'modified drug applications'. Among adults, metastatic cancers and palliative care patients received the most off-label drugs. The off-label drug use unsupported by standard treatment guidelines or drug compendia was in the range of 7%-31%.
Off-label drug use in cancer therapy is commonly practised but outcomes could vary significantly. Hence, greater scrutiny and robust clinical guidance is needed to establish the favourable benefit-risk ratio for patients at the time of prescribing at each level of oncology care to facilitate rational off-label prescribing.
药品的超说明书用药在多种疾病中广泛存在。这种处方行为在肿瘤学领域尤为突出。然而，由于临床获益和潜在毒性存在不确定性、支持临床决策的证据有限、患者自付费用增加以及缺乏知情同意引发的伦理问题，抗癌药物超说明书用药的适用性仍是一个有争议的问题。目前，缺乏有关癌症治疗中超说明书用药全球流行率的数据。本综述的目的是概述肿瘤学中超说明书用药的流行情况。
根据系统评价和Meta分析的首选报告项目指南，于1975年至2016年在PubMed上进行了系统的文献检索。纳入评估抗癌药物超说明书用药流行率的研究。
在检索到的199篇符合条件的论文中，23项研究纳入了本系统文献综述。住院患者的超说明书用药率在18%至41%之间。在成年癌症患者中，13% - 71%至少接受过一次超说明书化疗。超说明书用药的主要原因是“药物未获特定肿瘤批准”和“药物应用调整”。在成年人中，转移性癌症患者和姑息治疗患者接受超说明书用药最多。未得到标准治疗指南或药品手册支持的超说明书用药率在7%至31%之间。
癌症治疗中的超说明书用药很常见，但结果可能差异很大。因此，需要更严格的审查和强有力的临床指导，以便在肿瘤护理的各个层面开处方时为患者确定有利的获益风险比，以促进合理的超说明书用药。
