Institute of Ophthalmology and Visual Science, Rutgers-New Jersey Medical School, Rutgers University, Newark, New Jersey, USA.
PharmaLogic Development, Inc., San Rafael, California, USA.
J Ocul Pharmacol Ther. 2021 Nov;37(9):495-501. doi: 10.1089/jop.2021.0059. Epub 2021 Sep 3.
N-of-1 trials are randomized, prospective, controlled, multiple crossover trials in a single patient. Effects of one or more treatments are studied by following individual patients who receive alternative treatments (eg, therapeutic intervention). Such trials may provide a path to assess treatments for rare diseases with rigor equal to or greater than that afforded by parallel group randomized clinical trials provided that the condition is reasonably stable during the trial and has a sign/symptom that responds reversibly to the therapy and that can be measured repeatedly. In this article, the authors propose that N-of-1 trials may improve the feasibility and affordability of clinical trials for patients with rare inherited retinal diseases.
N-of-1 试验是在单个患者中进行的随机、前瞻性、对照、多次交叉试验。通过随访接受不同治疗（例如治疗干预）的个体患者来研究一种或多种治疗方法的效果。此类试验可能为评估罕见疾病的治疗方法提供一种途径，其严格程度与平行组随机临床试验相当或更高，前提是在试验期间病情相对稳定，并且存在对治疗有可逆反应且可重复测量的体征/症状。在本文中，作者提出 N-of-1 试验可能会提高罕见遗传性视网膜疾病患者临床试验的可行性和可负担性。
