Kravitz Richard L, Duan Naihua, Niedzinski Edmund J, Hay M Cameron, Subramanian Saskia K, Weisner Thomas S
University of California, Davis, USA.
Milbank Q. 2008 Dec;86(4):533-55. doi: 10.1111/j.1468-0009.2008.00533.x.
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.
The authors reviewed the literature and conducted in-depth telephone interviews with leaders in the n-of-1 trial movement.
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.
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.
在可行的情况下，随机、盲法单病例（n=1）试验在确定个体患者的最佳治疗方案方面具有独特的能力。尽管早期人们热情高涨，但到21世纪之交，很少有学术中心定期开展n=1试验。
作者查阅了文献，并对n=1试验领域的领军人物进行了深入的电话访谈。
n=1试验可以通过提高治疗精准度来改善医疗护理。然而，它们尚未得到广泛采用，部分原因是医生认为与所带来的不便相比，其降低不确定性的价值不够高。有限的证据表明，患者一旦了解了n=1试验的益处，可能会接受这种试验。
n=1试验为个性化临床护理和丰富临床研究提供了独特的机会。虽然药物研发、生产和营销方面正在发生的变化最终可能促使制药商和医疗保健支付方支持n=1试验，但目前最有前景的复苏策略是将n=1试验精简到最基本要素，并直接向患者推广。为了优化从这些试验中得出的统计推断，可以使用经验贝叶斯方法将个体患者数据与来自可比患者的汇总数据相结合。
