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Clinical trials and rare diseases: a way out of a conundrum.

机译:临床试验和罕见疾病:摆脱难题的出路。

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摘要

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.
机译:目前,临床试验往往由个人资助,申请人必须在其资助申请中包括功效计算。但是,如果需要通过试验来评估罕见病的治疗方法,则无法获得常规水平的统计精度。这意味着治疗此类疾病的临床医生仍然一无所知,必须仅根据(可能有偏见的)观察研究经验和轶事来做出判断。由于一些公正的证据显然比没有证据更好,因此这种情况不应继续下去。但是,即使处理方式大不相同,常规(频繁)的置信度限制也不大可能排除无效结果。贝叶斯方法利用所有可用数据来计算可直接推断给临床实践的概率。因此,供资机构应为小型试验的全部经费提供经费,这些试验不需要预定的结局,并且需要进行标准的大型研究。

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