Department of Medicine and Surgery, University of Insubria, Varese, Italy.
Department of Medicine and Cardiopulmonary Rehabilitation, Maugeri Care and Research Institute, IRCCS Tradate, Varese, Italy.
Pharmaceut Med. 2020 Jun;34(3):159-165. doi: 10.1007/s40290-020-00334-z.
Superiority trials are conducted to test the hypothesis that a treatment or strategy A is superior to (i.e., better than) treatment strategy B in reducing the impact of disease. However, A may be considerably safer, more convenient, or cheaper than B. These features may make A more attractive than B even if the burden of disease is reduced comparably by the two treatments, or even a bit worse by A over B. In this context, non-inferiority trials have become increasingly popular to test the hypothesis that a new treatment is not 'unacceptably worse' than an active comparator by more than a predefined non-inferiority margin. Non-inferiority trials have unique design features and methodology and require a different analysis than traditional superiority trials. The main aim of this overview is to analyze the role of non-inferiority trials in the development of new treatments, involving some scientific, statistical, and practical considerations. We elucidate some aspects of non-inferiority trials that contribute to the validity of the results. The unique design features and methodology of non-inferiority trials are summarized and key findings to consider when evaluating a non-inferiority trial are illustrated.
优势试验旨在检验一种治疗或策略 A 优于（即优于）策略 B 降低疾病影响的假设。然而，A 可能比 B 更安全、更方便或更便宜。即使两种治疗方法减轻疾病负担的程度相当，或者 A 比 B 略差，这些特征可能使 A 比 B 更具吸引力。在这种情况下，非劣效性试验越来越受欢迎，旨在检验新的治疗方法不比活性对照药物差得“无法接受”的假设，其差值超过预设的非劣效性边界。非劣效性试验具有独特的设计特点和方法，需要与传统的优势性试验不同的分析。本综述的主要目的是分析非劣效性试验在新治疗方法发展中的作用，涉及一些科学、统计和实际考虑因素。我们阐明了一些有助于结果有效性的非劣效性试验的方面。总结了非劣效性试验的独特设计特点和方法，并说明了在评估非劣效性试验时需要考虑的关键发现。
