Harvard Clinical Research Institute, Boston, Massachusetts;, †Division of Cardiology, Massachusetts General Hospital, Boston, Massachusetts;, ‡Cardiology Division, Department of Internal Medicine, Hennepin County Medical Center, University of Minnesota, Minneapolis, Minnesota;, §Division of Nephrology, Stanford University School of Medicine, Palo Alto, California;, ‖Duke Clinical Research Institute, Duke University School of Medicine, Durham, North Carolina, ¶Renal Division and Clinical Biometrics, Brigham and Women Hospital, Boston, Massachusetts.
Clin J Am Soc Nephrol. 2013 Dec;8(12):2213-20. doi: 10.2215/CJN.05130513. Epub 2013 Aug 22.
The lack of evidence on the effectiveness and safety of interventions in chronic dialysis patients has been a subject of continuing criticism. New technologies are often introduced into the market without having specifically studied or even included patients with advanced kidney disease. Therefore, the need to generate valid effectiveness and safety data in this vulnerable subpopulation is of utmost importance. The US Food and Drug Administration has recently placed an increased focus on safety surveillance, and sponsors must now meet this additional postmarketing commitment. In patients with ESRD, the unique data collection environment in the United States allows for creative and efficient study designs to meet the needs of patients, providers, and sponsors. The purpose of this manuscript is to review the methodological and practical aspects of the different options for postmarketing study design in this field, with critical appraisal of their advantages and disadvantages.
干预措施在慢性透析患者中的有效性和安全性缺乏证据，这一直是持续批评的主题。新技术经常被引入市场，而没有专门针对晚期肾病患者进行研究，甚至没有将这些患者纳入其中。因此，在这个脆弱的亚人群中生成有效且安全的数据至关重要。美国食品和药物管理局最近更加关注安全性监测，赞助商现在必须满足这一额外的上市后承诺。在美国，终末期肾病患者独特的数据收集环境允许采用创造性和高效的研究设计来满足患者、提供者和赞助商的需求。本文的目的是回顾该领域中不同的上市后研究设计方案的方法学和实际方面，并对其优缺点进行批判性评估。
