Targeted Clinical Trials

摘要: Randomized clinical trials (RCTs) are the most reliable scientific tool for identification of safe and effective therapies cancer. Indeed, it can be argued that such indispensible this purpose. The design analysis these generally aimed at differences in overall population parameters. compare outcome measures as response rates, progression-free survival (PFS), disease-free survival, (OS) patients assumed to representative under study, determined by specified eligibility criteria on trial. Although no assumption homogeneity studied or relative effects treatment is required, major conclusions and, new agents, subsequent regulatory approvals based population. For example, a conclusion might some therapy better than standard with respect PFS, if observed hazard ratio significantly less unity (in favor therapy), even “average” benefit other scales (e.g., difference PFS medians) quite small. This legitimate reasonable approach, one has over time yielded advances cancer treatments. However, sufficiently broad, desirable feature traditional RCTs, unrecognized heterogeneity may have substantial effect power trial (Betensky et al. 2002; Zhang 2006). there reasons suspect study only subset any due solely largely results subset.If true reliably identified beforehand, benefits could accrue targeting rather broader one. Benefits include more efficient trials, avoidance unnecessary who will not from treatment, faster drug development times, so on. concept “personalized” medicine predicated existence exploitable patient differences. Of course, population, assuming exists, easy, but critical importance (George 2008).