Knight Cancer Institute.
Division of Hematology Oncology, Knight Cancer Institute.
J Natl Compr Canc Netw. 2020 Feb;18(2):161-167. doi: 10.6004/jnccn.2019.7349.
Noninferiority (NI) trials should help identify interventions that offer some benefit (eg, lower financial costs, more tolerable, or less invasive) without sacrificing noticeable effectiveness, and researchers should adhere to appropriate standards in the conduct and reporting of methods. This study describes the characteristics of a systematic sampling of NI studies from an updated search of recent published oncology trials.
We performed a cross-sectional analysis of NI research published between 2014 and 2018 in the top 3 medical journals and top 3 oncology journals. We estimated the percentage of NI trials in oncology that report informative details of study, such as justification for conducting NI trial, justification of NI margin, analysis population, and alpha level.
There were 94 NI studies and 104 comparisons, and 59.6% (n=62) of comparisons declared NI. The median NI margin of comparisons reporting an odds or hazard ratio was 1.3 (1.05-3.2; n=64). Twenty-three percent (n=22) of studies did not provide a justification for conducting a NI study; 54.3% (n=51) of studies did not provide a justification of the margin they used in their study. Only approximately 46% (n=43) of comparisons used both an intention-to-treat (ITT) and per-protocol (PP) analysis, and 37.3% (n=35) of studies used a one-sided alpha level of >.025. There is notable variation in key elements of the conduct and reporting of NI trials, including the NI margin, the alpha level, and the population analyzed. Furthermore, a high number of studies do not provide justification for conducting a NI study or the margin used for determining NI.
These results suggest that there is room for improvement in the reporting and conduct of NI trials in oncology.
非劣效性 (NI) 试验应有助于确定具有一定益处（例如，降低财务成本、更可耐受或侵入性更小）的干预措施，同时不会牺牲显著的疗效，并且研究人员应在方法的实施和报告中遵守适当的标准。本研究描述了从最近发表的肿瘤学试验的更新搜索中系统抽样的 NI 研究的特征。
我们对 2014 年至 2018 年期间在顶级 3 家医学期刊和顶级 3 家肿瘤学期刊上发表的 NI 研究进行了横断面分析。我们估计了肿瘤学中报告 NI 试验的信息量的研究百分比，例如进行 NI 试验的理由、NI 边界的理由、分析人群和 alpha 水平。
共有 94 项 NI 研究和 104 项比较，其中 59.6%（n=62）的比较宣布为 NI。报告比值比或风险比的比较的中位数 NI 边界为 1.3（1.05-3.2；n=64）。23%（n=22）的研究未提供进行 NI 研究的理由；54.3%（n=51）的研究未提供他们在研究中使用的边界的理由。只有大约 46%（n=43）的比较同时使用了意向治疗（ITT）和按方案（PP）分析，并且 37.3%（n=35）的研究使用了单侧 alpha 水平>0.025。NI 试验的实施和报告的关键要素存在明显差异，包括 NI 边界、alpha 水平和分析人群。此外，许多研究未提供进行 NI 研究或确定 NI 所用边界的理由。
这些结果表明，在肿瘤学中，NI 试验的报告和实施还有改进的空间。
