Methodological quality of oncology noninferiority clinical trials

Abstract

Introduction: 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.

Methods: 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.

Results: 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 %.

Conclusions: 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.

Keywords: Clinical trial; Epidemiology; Noninferiority trial; Oncology; Quality.