Götte Heiko, Donica Margarita, Mordenti Giacomo
a Global Biostatistics, Merck KGaA , Darmstadt , Germany.
J Biopharm Stat. 2015;25(5):1020-38. doi: 10.1080/10543406.2014.929583. Epub 2014 Jun 10.
Choice of target population is an essential part at the design stage of clinical trials. Data from earlier clinical development might suggest that the treatment is more effective in a subpopulation, but there might not be enough evidence to restrict the target population upfront. Adaptive designs allow modification of the target population based on interim data. Decision for modification should be based on objective decision rules. The presented decision rules maximize the weighted probability of correct interim decisions based on prior assumptions. Evaluation of decision rules in the planning phase can improve probabilities of correct interim decision and power.
目标人群的选择是临床试验设计阶段的一个重要部分。早期临床开发的数据可能表明该治疗在一个亚组中更有效，但可能没有足够的证据预先限制目标人群。适应性设计允许根据中期数据修改目标人群。修改的决策应基于客观的决策规则。所提出的决策规则基于先验假设最大化正确中期决策的加权概率。在规划阶段对决策规则进行评估可以提高正确中期决策的概率和检验效能。
