Oklahoma State University Center for Health Sciences, Department of Biomedical Sciences, 1111 W 17th Street, Tulsa, OK, 74107, United States.
Oklahoma State University Center for Health Sciences, Department of Psychiatry and Behavioral Sciences, Tulsa, OK, 74107, United States.
Crit Rev Oncol Hematol. 2020 May;149:102938. doi: 10.1016/j.critrevonc.2020.102938. Epub 2020 Mar 5.
Noninferiority trials can show that new treatments with slightly less efficacy are safer, cheaper, or easier to administer. However, the conclusions of noninferiority trials depend on robust methodology.
We conducted a 6 year cross-sectional investigation of the methodological quality of oncology noninferiority trials published in the top 10 oncology journals. Four key quality criteria were investigated.
Nonefficacy benefits of the new treatment were stated in 88/110 (80.0 %) trials. Justification for the noninferiority margin was provided in 79/110 (71.8 %) trials. Authors most often used previous data as justification for the chosen margin (n = 42). In 15 noninferiority trials the percent preserved effect could be calculated and the median effect preserved was 56.8 %.
The oncology noninferiority trials included in our study had key methodological shortcomings, counterbalanced by a clear delineation of expected nonefficacy benefits of the new treatment.
非劣效性试验可以表明，疗效略低但更安全、更便宜或更易于管理的新疗法。然而，非劣效性试验的结论取决于稳健的方法。
我们对发表在顶级 10 家肿瘤学期刊上的肿瘤学非劣效性试验的方法学质量进行了为期 6 年的横断面调查。调查了 4 个关键的质量标准。
88/110（80.0%）项试验中陈述了新治疗方法的无效益处。79/110（71.8%）项试验中提供了非劣效性边界的依据。作者最常使用先前的数据作为选择边界的依据（n=42）。在 15 项非劣效性试验中，可以计算出保留效应的百分比，中位数保留效应为 56.8%。
我们研究中纳入的肿瘤学非劣效性试验存在关键的方法学缺陷，但新治疗方法预期的无效益处有明确的说明。
