The statistical principles of fully adaptive designs are outlined. The options of flexibility and the price to be paid in terms of statistical properties of the test procedures are discussed. It is stressed that controlled inference after major design modifications (changing hypotheses) will include a penalty: Intersections among all the hypotheses considered throughout the trial have to be rejected before testing individual hypotheses. Moreover, feasibility in terms of integrity and persuasiveness of the results achieved after adaptations based on unblinded data is considered as the crucial issue in practice. In the second part, sample size adaptive procedures are considered testing a large number of hypotheses under constraints on total sample size as in genetic studies. The advantage of sequential procedures is sketched for the example of two-stage designs with a pilot phase for screening promising hypotheses (markers) and controlling the false discovery rate. Finally, we turn to the clinical problem how to select markers and estimate a score from limited samples, e.g. for predicting the response to therapy of a future patient. The predictive ability of such scores will be rather poor when investigating a large number of hypotheses and truly large marker effects are lacking. An obvious dilemma will show up: More optimistic selection rules may be superior if in fact effective markers exist, but will produce more nuisance prediction if no effective markers exist compared with more cautious strategies, e.g. aiming at some control of type I error probabilities.

译文

概述了完全自适应设计的统计原理。根据测试程序的统计特性,讨论了灵活性的选择和要支付的价格。需要强调的是,重大设计修改 (改变假设) 后的受控推理将包括一个惩罚: 在测试单个假设之前,必须拒绝整个试验中考虑的所有假设之间的交叉。此外,根据非盲数据进行改编后,在完整性和说服力方面的可行性被认为是实践中的关键问题。在第二部分中,考虑了样本量自适应程序,例如在遗传研究中,在总样本量的约束下测试了大量假设。对于两阶段设计的示例,概述了顺序程序的优势,该阶段具有试点阶段,用于筛选有希望的假设 (标记) 并控制错误发现率。最后,我们转向临床问题如何从有限的样本中选择标记并估计分数,例如用于预测未来患者对治疗的反应。在调查大量假设时,此类分数的预测能力将很差,并且缺乏真正的大标记效应。一个明显的困境将出现: 如果实际上存在有效的标记,则更乐观的选择规则可能会更优越,但是如果与更谨慎的策略相比,没有有效的标记存在,则会产生更多的干扰预测,例如针对I型错误概率的某些控制。

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