Sankoh Abdul J, D'Agostino Ralph B, Huque Mohammad F
Global Biostatistics, Aventis Pharmaceuticals, P. O. Box 6890, 200 Crossing Boulevard, BX2-300E, Bridgewater, NJ 08807, U.S.A.
Stat Med. 2003 Oct 30;22(20):3133-50. doi: 10.1002/sim.1557.
The ideal approach for the design and analysis of clinical trials is to select a single primary endpoint that provides a complete characterization of the disease under study and permits an efficient evaluation of the effect of a test drug. However, this is often not possible for a number of diseases or clinical trials. This paper examines some practical clinical decision-making scenarios for the selection and analysis of efficacy outcome measures in clinical trials with inherent multiplicity components.
临床试验设计与分析的理想方法是选择一个单一的主要终点，该终点能全面表征所研究的疾病，并能有效评估受试药物的效果。然而，对于许多疾病或临床试验而言，这往往是不可能的。本文探讨了一些实际的临床决策场景，用于在具有内在多重性成分的临床试验中选择和分析疗效结局指标。
