Clinical trials and rare diseases: A way out of a conundrum
Clinical trials and rare diseases: A way out of a conundrum
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DOI：
10.1136/bmj.311.7020.1621
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发表时间：
1995-12-16
影响因子：
105.7
通讯作者：
Braunholtz, D
中科院分区：
文献类型：
--
作者：
Lilford, RJ;Thornton, JG;Braunholtz, D
目前，临床试验往往是单独资助的，申请人必须在他们的拨款申请中包括一个功率计算。然而，如果试验需要评估罕见疾病的治疗，则不太可能前瞻性地获得常规水平的统计学精确度。这意味着治疗此类疾病的临床医生仍然处于无知状态，必须完全基于（可能存在偏见的）观察性研究、经验和轶事来做出判断。既然有一些公正的证据显然比没有好，这种情况不应继续下去。然而，常规（频率论）置信限不太可能排除无效结果，即使治疗方法差异很大。贝叶斯方法利用所有可用的数据来计算可能直接外推到临床实践的概率。因此，资助机构应该资助一系列小型试验，这些试验不需要预先确定目的，同时还应资助标准的大型研究。
Currently, clinical trials tend to be individually funded and applicants must include a power calculation in their grant request. However, conventional levels of statistical precision are unlikely to be obtainable prospectively if the trial is required to evaluate treatment of a rare disease. This means that clinicians treating such diseases remain in ignorance and must form their judgments solely on the basis of (potentially biased) observational studies, experience, and anecdote. Since some unbiased evidence is clearly better than none, this state of affairs should not continue. However, conventional (frequentist) confidence limits are unlikely to exclude a null result, even when treatments differ substantially. Bayesian methods utilise all available data to calculate probabilities that may be extrapolated directly to clinical practice. Funding bodies should therefore fund a repertoire of small trials, which need have no predetermined end, alongside standard larger studies.