Hemkens Lars G, Contopoulos-Ioannidis Despina G, Ioannidis John P A
Stanford Prevention Research Center, Department of Medicine (Hemkens, Ioannidis), Stanford University School of Medicine, Stanford, Calif.; Basel Institute for Clinical Epidemiology and Biostatistics (Hemkens), University Hospital Basel, Basel, Switzerland; Department of Pediatrics, Division of Infectious Diseases (Contopoulos-Ioannidis), Stanford University School of Medicine, Stanford, Calif.; Palo Alto Medical Foundation Research Institute (Contopoulos-Ioannidis), Palo Alto, Calif.; Department of Health Research and Policy (Ioannidis), Stanford University School of Medicine; Meta-Research Innovation Center at Stanford (METRICS) (Ioannidis), Stanford, Calif.
CMAJ Open. 2016 Apr 6;4(2):E132-40. doi: 10.9778/cmajo.20150036. eCollection 2016 Apr-Jun.
Studies that use routinely collected health data (RCD studies) are advocated to complement evidence from randomized controlled trials (RCTs) for comparative effectiveness research and to inform health care decisions when RCTs would be unfeasible. We aimed to evaluate the current use of routinely collected health data to complement RCT evidence.
We searched PubMed for RCD studies published to 2010 that evaluated the comparative effectiveness of medical treatments on mortality using propensity scores. We identified RCTs of the same treatment comparisons and evaluated how frequently the RCD studies analyzed treatments that had not been compared previously in randomized trials. When RCTs did exist, we noted the claimed motivations for each RCD study. We also analyzed the citation impact of the RCD studies.
Of 337 eligible RCD studies identified, 231 (68.5%) analyzed treatments that had already been compared in RCTs. The study investigators rarely claimed that it would be unethical (6/337) or difficult (18/337) to perform RCTs on the same question. Evidence from RCTs was mentioned or cited by authors of 213 RCD studies. The most common motivations for conducting the RCD studies were alleged limited generalizability of trial results to the "real world" (37.6%), evaluation of specific outcomes (31.9%) or specific populations (23.5%), and inconclusive or inconsistent evidence from randomized trials (25.8%). Studies evaluating "real world" effects had the lowest citation impact.
Most of the RCD studies we identified explored comparative treatment effects that had already been investigated in RCTs. The objective of such studies needs to shift more toward answering pivotal questions that are not supported by trial evidence or for which RCTs would be unfeasible.
有人主张使用常规收集的健康数据的研究（RCD研究）来补充随机对照试验（RCT）的证据，以进行比较效果研究，并在RCT不可行时为医疗保健决策提供信息。我们旨在评估当前常规收集的健康数据对RCT证据的补充作用。
我们在PubMed中搜索截至2010年发表的使用倾向评分评估医学治疗对死亡率的比较效果的RCD研究。我们确定了相同治疗比较的RCT，并评估了RCD研究分析随机试验中先前未比较过的治疗的频率。当存在RCT时，我们记录了每项RCD研究声称的动机。我们还分析了RCD研究的引文影响力。
在确定的337项符合条件的RCD研究中，231项（68.5%）分析了在RCT中已经比较过的治疗。研究调查人员很少声称就同一问题进行RCT是不道德的（6/337）或困难的（18/337）。213项RCD研究的作者提到或引用了RCT的证据。进行RCD研究最常见的动机是声称试验结果对“现实世界”的普遍适用性有限（37.6%）、评估特定结局（31.9%）或特定人群（23.5%），以及随机试验的证据不确定或不一致（25.8%）。评估“现实世界”效应的研究的引文影响力最低。
我们确定的大多数RCD研究探讨了在RCT中已经研究过的比较治疗效果。此类研究的目标需要更多地转向回答试验证据不支持或RCT不可行的关键问题。
