PDF(Pubmed)
Abstract：
UNASSIGNED: Ideally, real-world data (RWD) collected to generate real-world evidence (RWE) should lead to impact on the care and health of real-world patients. Deriving from care in which clinicians and patients try various treatments to inform therapeutic decisions, N-of-1 trials bring scientific methods to real-world practice.
UNASSIGNED: These single-patient crossover trials generate RWD and RWE by giving individual patients various treatments in a double-blinded way in sequential periods to determine the most effective treatment for a given patient.
UNASSIGNED: This approach is most often used for patients with chronic, relatively stable conditions that provide the opportunity to make comparisons over multiple treatment periods, termed Type 1 N-of-1 trials. These are most helpful when there is heterogeneity of treatment effects among patients and no a priori best option. N-of-1 trials also can be done for patients with rare diseases, potentially testing only one treatment, to generate evidence for personalized treatment decisions, designated as Type 2 N-of-1 trials. With both types, in addition to informing individual\'s treatments, when uniform protocols are used for multiple patients with the same condition, the data collected in the individual N-of-1 trials can be aggregated to provide RWD/RWE to inform more general use of the treatments. Thereby, N-of-1 trials can provide RWE for the care of individuals and for populations.
UNASSIGNED: To fulfill this potential, we believe N-of-1 trials should be built into our current healthcare ecosystem. To this end, we are building the needed infrastructure and engaging the stakeholders who should receive value from this approach.
UNASSIGNED: These single-patient crossover trials generate RWD and RWE by giving individual patients various treatments in a double-blinded way in sequential periods to determine the most effective treatment for a given patient.
UNASSIGNED: This approach is most often used for patients with chronic, relatively stable conditions that provide the opportunity to make comparisons over multiple treatment periods, termed Type 1 N-of-1 trials. These are most helpful when there is heterogeneity of treatment effects among patients and no a priori best option. N-of-1 trials also can be done for patients with rare diseases, potentially testing only one treatment, to generate evidence for personalized treatment decisions, designated as Type 2 N-of-1 trials. With both types, in addition to informing individual\'s treatments, when uniform protocols are used for multiple patients with the same condition, the data collected in the individual N-of-1 trials can be aggregated to provide RWD/RWE to inform more general use of the treatments. Thereby, N-of-1 trials can provide RWE for the care of individuals and for populations.
UNASSIGNED: To fulfill this potential, we believe N-of-1 trials should be built into our current healthcare ecosystem. To this end, we are building the needed infrastructure and engaging the stakeholders who should receive value from this approach.
摘要：
■理想情况下，收集真实世界数据（RWD）以生成真实世界证据（RWE）应导致对真实世界患者的护理和健康的影响。源于临床医生和患者尝试各种治疗来告知治疗决策的护理，N-of-1试验为现实世界的实践带来了科学的方法。
■这些单患者交叉试验通过在连续周期中以双盲方式为个体患者提供各种治疗来产生RWD和RWE，以确定给定患者的最有效治疗。
■这种方法最常用于慢性病患者，相对稳定的条件，提供了在多个治疗期间进行比较的机会，称为1型N-of-1试验。当患者之间存在治疗效果的异质性并且没有先验的最佳选择时，这些是最有帮助的。N-of-1试验也可以对罕见疾病患者进行，可能只测试一种治疗方法，为个性化治疗决策提供证据，指定为2型N-of-1试验。这两种类型,除了告知个人的治疗，当统一的协议被用于具有相同条件的多个患者，可以汇总在单个N-of-1试验中收集的数据,以提供RWD/RWE,从而为更普遍的治疗方法提供依据.因此，N-of-1试验可以为个体和人群的护理提供RWE。
■为了实现这一潜力，我们认为N-of-1试验应该纳入我们当前的医疗生态系统.为此,我们正在建设所需的基础设施，并让应该从这种方法中获得价值的利益相关者参与进来。
■这些单患者交叉试验通过在连续周期中以双盲方式为个体患者提供各种治疗来产生RWD和RWE，以确定给定患者的最有效治疗。
■这种方法最常用于慢性病患者，相对稳定的条件，提供了在多个治疗期间进行比较的机会，称为1型N-of-1试验。当患者之间存在治疗效果的异质性并且没有先验的最佳选择时，这些是最有帮助的。N-of-1试验也可以对罕见疾病患者进行，可能只测试一种治疗方法，为个性化治疗决策提供证据，指定为2型N-of-1试验。这两种类型,除了告知个人的治疗，当统一的协议被用于具有相同条件的多个患者，可以汇总在单个N-of-1试验中收集的数据,以提供RWD/RWE,从而为更普遍的治疗方法提供依据.因此，N-of-1试验可以为个体和人群的护理提供RWE。
■为了实现这一潜力，我们认为N-of-1试验应该纳入我们当前的医疗生态系统.为此,我们正在建设所需的基础设施，并让应该从这种方法中获得价值的利益相关者参与进来。
