What Ever Happened to N-of-1 Trials? Insiders' Perspectives and a Look to the Future
What Ever Happened to N-of-1 Trials? Insiders' Perspectives and a Look to the Future
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DOI：
10.1111/j.1468-0009.2008.00533.x
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发表时间：
2008-12-01
影响因子：
6.6
通讯作者：
Weisner, Thomas S.
中科院分区：
文献类型：
--
作者：
Kravitz, Richard L.;Duan, Naihua;Weisner, Thomas S.
上下文：当可行，随机，盲人单患者（N-OF-1）试验具有独特的能力时，可以在单个患者中建立最佳治疗方法。尽管有早期的热情，但到二十一世纪初，很少有学术中心定期进行N-1-1-1试验。 1审判运动。调查：N-1-1试验可以通过提高治疗精度来改善护理。但是，它们并未被广泛采用，部分原因是医生不充分珍视他们产生的不确定性的降低与给他们带来的不便。有限的证据表明，一旦理解好处，患者可能会接受N-1-1试验。结论：N-1-1试验提供了一个独特的机会来个性化临床护理并丰富临床研究。尽管药物发现，制造和营销的持续变化最终可能会刺激药品制造商和医疗保健付款人来支持N-1-1-1试验，但目前最有希望的复苏策略是将N-1-1-1试验剥离为其必需品并进行营销直接给患者。为了优化这些试验的统计推断，经验贝叶斯方法可用于将单个患者数据与可比较患者的骨料数据结合在一起。
Context: When feasible, randomized, blinded single-patient (n-of-1) trials are uniquely capable of establishing the best treatment in an individual patient. Despite early enthusiasm, by the turn of the twenty-first century, few academic centers were conducting n-of-1 trials on a regular basis.Methods: The authors reviewed the literature and conducted in-depth telephone interviews with leaders in the n-of-1 trial movement.Findings: N-of-1 trials can improve care by increasing therapeutic precision. However, they have not been widely adopted, in part because physicians do not sufficiently value the reduction in uncertainty they yield weighed against the inconvenience they impose. Limited evidence suggests that patients may be receptive to n-of-1 trials once they understand the benefits.Conclusions: N-of-1 trials offer a unique opportunity to individualize clinical care and enrich clinical research. While ongoing changes in drug discovery, manufacture, and marketing may ultimately spur pharmaceutical makers and health care payers to support n-of-1 trials, at present the most promising resuscitation strategy is stripping n-of-1 trials to their essentials and marketing them directly to patients. In order to optimize statistical inference from these trials, empirical Bayes methods can be used to combine individual patient data with aggregate data from comparable patients.