比较有效性研究中的方法学问题：临床试验。

Methodological issues in comparative effectiveness research: clinical trials.

机构信息

VA Cooperative Studies Program Coordinating Center, US Department of Veterans Affairs, West Haven, Connecticut, USA.

出版信息

Am J Med. 2010 Dec;123(12 Suppl 1):e8-15. doi: 10.1016/j.amjmed.2010.10.003.

DOI:10.1016/j.amjmed.2010.10.003

Abstract

The US Department of Veterans Affairs (VA) Cooperative Studies Program has been conducting comparative effectiveness clinical trials for nearly 4 decades in many disease areas, including cardiovascular disease/surgery, diabetes mellitus, mental health, neurologic disorders, cancer, infectious diseases, and rheumatoid arthritis. The features that have made this program advantageous for conducting comparative effectiveness clinical trials are described along with methodological considerations for future trials based on lessons learned from its experience conducting these types of studies. Some of the lessons learned involve managing risk factors, clinical equipoise, patient preferences, evolving technology, the use of usual care as a comparator and pharmaceutical issues related to study drug blinding. These issues are not unique to the VA but can play an important role in enabling valid comparisons between treatments that may have differences in delivery or mechanisms of action and could affect the execution and feasibility of conducting a clinical trial with a comparative effectiveness aim. We also outline some future directions for comparative effectiveness clinical trials.

摘要

美国退伍军人事务部（VA）合作研究计划在近 40 年来一直在许多疾病领域开展以比较效果为目的的临床试验，包括心血管疾病/手术、糖尿病、心理健康、神经障碍、癌症、传染病和类风湿关节炎。本文介绍了使该计划有利于开展以比较效果为目的的临床试验的特点，并根据从开展这类研究中吸取的经验教训，对未来试验的方法学考虑因素进行了讨论。从经验中学到的一些内容涉及管理风险因素、临床均衡、患者偏好、技术发展、将常规护理作为对照以及与研究药物设盲相关的制药问题。这些问题不仅是 VA 特有的，它们在促进具有不同给药途径或作用机制的治疗方法之间的有效比较方面发挥着重要作用，可能会影响具有比较效果目的的临床试验的实施和可行性。我们还概述了一些未来的比较效果临床试验方向。