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抗肿瘤药物临床试验适应性设计的应用

Application of Adaptive Designs Used in Clinical Trials of Anti-Cancer Drugs

  • 摘要: 抗肿瘤药物临床试验具有周期长、风险高、投入大及疗效评估复杂等特点。传统试验设计进度缓慢、成本高,增加了研究的不确定性。随着政策引导与临床试验方法学的发展,适应性设计(Adaptive design)应运而生。该设计凭借其灵活性,能够缩短试验时间,更高效识别有效药物与疗法,从而提升新药研发的效率与成功率。本文基于抗肿瘤药物临床试验的特点,系统综述当前所采用的适应性设计,结合具体研发案例,比较分析不同适应性设计方法的特性与差异,探讨其面临的问题与挑战,以期为抗肿瘤药物临床试验提供方法学参考。

     

    Abstract: Clinical trials of anti-cancer drugs are characterized by long cycles, high risks, large investments, complex efficacy evaluations, and numerous influencing factors. Traditional experimental design is slow in progress and high in cost, which to some extent increases the uncertainty of research. With the guidance of domestic and international policies and the development of clinical trial methodologies, adaptive design has emerged. Flexible adaptive designs can shorten the clinical trial time of anti-cancer drugs, which is conducive to the discovery of truly effective anti-cancer drugs and therapies, thereby increasing the efficiency and success rate of new drug research and development. Starting from the characteristics of clinical trials of anti-cancer drugs, this article reviews the adaptive designs adopted in current clinical trials, summarizes the characteristics and differences of various adaptive design methods by introducing specific research and development cases, and discusses the problems and challenges faced. This work aims to provide methodological references for clinical trials of anti-cancer drugs.

     

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