Statistical approaches to group sequential monitoring of postmarket safety surveillance data: current state of the art for use in the Mini-Sentinel pilot
Statistical approaches to group sequential monitoring of postmarket safety surveillance data: current state of the art for use in the Mini-Sentinel pilot
复制标题
DOI：
10.1002/pds.2320
复制
发表时间：
2012-01-01
影响因子：
2.6
通讯作者：
Nelson, Jennifer C.
中科院分区：
文献类型：
--
作者：
Cook, Andrea J.;Tiwari, Ram C.;Nelson, Jennifer C.
目的：本文描述了目前可用的统计方法，用于预先指定的安全结果的上市后监测，使用前瞻性事件用户并发对照队列设计和现有的电子医疗保健数据。方法以美国食品药品监督管理局的迷你哨点试点为例，提供积极的上市后监测设置的动机。提出了四种序列监测统计方法，包括Lan-Demets误差花费法、以二项MaxSPRT为特例的匹配似然比检验统计方法、分层的条件序列抽样方法和基于置换的广义估计方程回归方法。提供了关于每种方法的假设、限制和优点的信息，包括每种方法如何定义顺序监视边界，使用什么测试统计量，以及它对罕见事件或频繁测试设置的鲁棒性如何。结果提出了一个假设的例子，说明如何将这些方法应用于比较感兴趣的医疗产品（药物A）与同时控制的药物（药物B）的数据，包括提供可用于监测此类药物的信息类型。版权所有：John Wiley & Sons， Ltd
Purpose This manuscript describes the current statistical methodology available for active postmarket surveillance of pre-specified safety outcomes using a prospective incident user concurrent control cohort design with existing electronic healthcare data. Methods Motivation of the active postmarket surveillance setting is provided using the Food and Drug Administration's Mini-Sentinel Pilot as an example. Four sequential monitoring statistical methods are presented including the Lan-Demets error spending approach, a matched likelihood ratio test statistic approach with the binomial MaxSPRT as a special case, the conditional sequential sampling procedure with stratification, and a generalized estimating equation regression approach using permutation. Information on the assumptions, limitations, and advantages of each approach is provided, including how each method defines sequential monitoring boundaries, what test statistic is used, and how robust it is to settings of rare events or frequent testing. Results A hypothetical example of how the approaches could be applied to data comparing a medical product of interest, drug A, to a concurrent control drug, drug B, is presented including providing the type of information one would have available for monitoring such drugs. Copyright (C) 2012 John Wiley & Sons, Ltd.