Small Clinical Trials: Issues and Challenges
Small Clinical Trials: Issues and Challenges
复制标题
小型临床试验：问题和挑战
DOI：
--
复制
发表时间：
2001
期刊：
影响因子：
--
通讯作者：
E. del Rio
中科院分区：
文献类型：
--
作者：
D. Reitberg;S. Weiss;E. del Rio
临床试验在设计、实施和分析方法方面有很长的历史，建立了良好的、有文件记录的和有效的方法。适当的研究设计包括足够的样本量(N)和统计能力，以及对偏差的控制，以允许对结果进行有意义的解释。临床试验的参与者数量应该始终足够大，以提供对所提出的研究问题的足够准确的答案，但也应该是实现这一目标所必需的最低限度。动力充足的随机临床试验和双盲随机临床试验通常被认为是确定新的治疗干预措施有效性的最权威的研究方法。通过将足够数量的个体分配给小组，例如实验或对照，研究人员可以某种程度上确定地估计或确定给定干预的效果。然而，当临床环境不能提供足够数量的研究对象来进行充分的动力和对照试验时--例如，因为一种罕见的疾病或特殊的患者群体(如宇航员)没有治疗方法，或者因为研究需要患有晚期或严重衰弱或丧失能力的疾病的患者参与--研究人员往往面临使他们能够向前迈进的设计和分析工具不足。为了回应对这种工具的需求，医学研究所的一个委员会被要求评估目前进行小样本临床试验的方法和适当的情况。该委员会的报告《小型临床试验：问题和挑战》中包含的建议描述了在设计和分析涉及小样本个人的试验时可以采取的方法，以获得可靠和有效的结果。
Clinical trials have a long history of well established, documented, and validated methods for design, conduct, and analysis. Appropriate study design includes sufficient sample size (n) and statistical power, and control of bias to allow for a meaningful interpretation of the results. The number of participants in a clinical trial should always be large enough to provide a sufficiently precise answer to the research question posed, but should also be the minimum necessary to achieve this aim. Adequately powered randomized clinical trials and double-blinded, randomized clinical trials are generally regarded as the most authoritative research methods for establishing the efficacy of new therapeutic interventions. By allocating sufficient numbers of individuals to groups, for example, experimental or control, investigators can estimate or determine with some degree of certainty the effect of a given intervention. However, when the clinical context does not provide a sufficient number of research subjects for an adequately powered and controlled trial—for example, because treatments are unavailable for a rare disorder or a unique patient population uch as astronauts, or because studies require the participation of patients with terminal or severely debilitating or incapacitating disorders— researchers are often faced with insufficient design and analysis tools that would allow them to move forward. In response to the need for such tools, an Institute of Medicine committee was asked to assess the current methodologies and appropriate situations for conducting clinical trials with small sample sizes. The committee’s report, Small Clinical Trials: Issues and Challenges, contains recommendations that describe approaches that can be taken in the design and analysis of trials involving a small sample of individuals to obtain reliable and valid results.