REDUCTION IN BURDEN OF ILLNESS - A NEW EFFICACY MEASURE FOR PREVENTION TRIALS
REDUCTION IN BURDEN OF ILLNESS - A NEW EFFICACY MEASURE FOR PREVENTION TRIALS
复制标题
DOI：
10.1002/sim.4780131803
复制
发表时间：
1994-09-30
影响因子：
2
通讯作者：
HEYSE, JF
中科院分区：
文献类型：
--
作者：
CHANG, MN;GUESS, HA;HEYSE, JF
开发了一种新的有效性测量方法，用于可能影响疾病发生率和疾病严重程度的干预措施的预防试验。我们为每个事件病例分配一个严重性评分，并将每个治疗组内所有事件病例的严重性评分相加，以创建每个治疗组的疾病负担评分。通过两个随机化治疗组中每例随机化受试者的疾病负担之间的差异评价疗效。由于每个疾病负担评分中的被加数是一个随机变量，因此标准分析方法不能直接应用。疾病负担评分净减少的渐近分布和抽样性质是针对设计为在固定随访时间后或发生固定数量病例后停止的试验得出的。我们说明的方法，从临床试验的人轮状病毒疫苗的数据。
A new efficacy measure is developed for use in prevention trials of interventions which may affect both disease incidence and disease severity. We assign a severity score to each incident case and sum severity scores over all incident cases within each treatment group to create a burden-of-illness score for each treatment group. Efficacy is evaluated by the difference between the burden-of-illness per randomized subject in the two randomized treatment groups. Since the numbers of summands in each burden-of-illness score is a random variable, standard methods of analysis are not directly applicable. The asymptotic distribution and sampling properties of the net reduction in the burden-of-illness score are derived for trials designed to stop either after a fixed length of follow-up or after the occurrence of a fixed number of cases. We illustrate the method with data from a clinical trial of a human rotavirus vaccine.