Tan A R, Swain S M
Cancer Therapeutics Branch, National Cancer Institute, Bethesda, MD 20889, USA.
Semin Oncol. 2001 Oct;28(5 Suppl 16):148-53. doi: 10.1016/s0093-7754(01)90292-1.
The development of molecularly targeted anticancer agents has become an attractive therapeutic strategy. However, evaluation of these novel drugs poses special challenges to laboratory and clinical scientists. These include the creation of appropriate models to assess the effect of the agent on its putative target, the validation of new surrogate endpoints, and the use of effective clinical trial designs. An overview of the difficulties in using a new paradigm of drug development for these target-directed compounds is presented and optimal clinical research methodology to determine their efficacy described.
分子靶向抗癌药物的研发已成为一种颇具吸引力的治疗策略。然而，对这些新型药物进行评估给实验室和临床科学家带来了特殊挑战。这些挑战包括创建合适的模型来评估药物对其假定靶点的作用、验证新的替代终点以及使用有效的临床试验设计。本文概述了将药物研发新范式用于这些靶向化合物时所面临的困难，并描述了确定其疗效的最佳临床研究方法。
