National Institute for Health and Clinical Excellence, London, UK.
Clin Trials. 2012 Aug;9(4):436-46. doi: 10.1177/1740774512450097. Epub 2012 Jul 2.
There is a growing appreciation that our current approach to clinical research leaves important gaps in evidence from the perspective of patients, clinicians, and payers wishing to make evidence-based clinical and health policy decisions. This has been a major driver in the rapid increase in interest in comparative effectiveness research (CER), which aims to compare the benefits, risks, and sometimes costs of alternative health-care interventions in 'the real world'. While a broad range of experimental and nonexperimental methods will be used in conducting CER studies, many important questions are likely to require experimental approaches - that is, randomized controlled trials (RCTs). Concerns about the generalizability, feasibility, and cost of RCTs have been frequently articulated in CER method discussions. Pragmatic RCTs (or 'pRCTs') are intended to maintain the internal validity of RCTs while being designed and implemented in ways that would better address the demand for evidence about real-world risks and benefits for informing clinical and health policy decisions. While the level of interest and activity in conducting pRCTs is increasing, many challenges remain for their routine use. This article discusses those challenges and offers some potential ways forward.
人们越来越认识到，从希望做出基于证据的临床和卫生政策决策的患者、临床医生和支付者的角度来看，我们目前的临床研究方法存在重要的证据缺口。这是人们对比较效果研究（CER）兴趣迅速增加的主要驱动因素之一，CER 的目的是比较替代医疗干预措施在“真实世界”中的益处、风险，有时还包括成本。虽然在进行 CER 研究时将使用广泛的实验和非实验方法，但许多重要问题可能需要采用实验方法，即随机对照试验（RCT）。在 CER 方法讨论中，人们经常表达对 RCT 普遍性、可行性和成本的担忧。实用随机对照试验（或“pRCT”）旨在在设计和实施时保持 RCT 的内部有效性，同时以更好地满足对真实世界风险和益处的证据的需求，以告知临床和卫生政策决策。虽然人们对进行 pRCT 的兴趣和活动水平在不断提高，但在常规使用方面仍存在许多挑战。本文讨论了这些挑战，并提出了一些潜在的前进方向。
