Department of Academic Rheumatology, Weston Education Centre, Denmark Hill Campus, King's College London, London SE5 9RJ, UK; and at the Department of Rheumatology, Whittington Hospital, Magdala Avenue, London N19 5NF, UK.
Leeds Institute of Rheumatic and Musculoskeletal Medicine, Chapel Allerton Hospital; and at the National Institute for Health Research (NIHR) Leeds Musculoskeletal Biomedical Research Unit, Chapel Allerton Hospital, Chapeltown Road, Leeds LS7 4SA, UK.
Nat Rev Rheumatol. 2017 Aug;13(8):503-510. doi: 10.1038/nrrheum.2017.81. Epub 2017 Jun 1.
The beginning of the 21st century saw a biopharmaceutical revolution in the treatment of inflammatory rheumatic diseases, particularly rheumatoid arthritis. The fast-evolving use of biologic therapies highlighted the need to develop registers at national and international levels with the aim of collecting long-term data on patient outcomes. Over the past 15 years, many biologics registers have contributed a wealth of data and provided robust and reliable evidence on the use, effectiveness and safety of these therapies. The unavoidable challenges posed by the continuous introduction of new therapies, particularly with regard to understanding their long-term safety, highlights the importance of learning from experience with established biologic therapies. In this Perspectives article, the role of biologics registers in bridging the evidence gap between efficacy in clinical trials and real-world effectiveness is discussed, with a focus on methodological aspects of registers, their unique features and challenges and their role going forward.
21 世纪初，生物制药在治疗炎症性风湿疾病（尤其是类风湿关节炎）方面掀起了一场革命。生物疗法的快速发展凸显了在国家和国际层面建立登记处的必要性，以便收集关于患者结局的长期数据。在过去的 15 年中，许多生物制剂登记处提供了大量数据，并为这些疗法的使用、有效性和安全性提供了可靠的证据。新疗法的不断引入带来了不可避免的挑战，特别是在了解其长期安全性方面，这凸显了从已确立的生物疗法中吸取经验的重要性。在这篇观点文章中，讨论了生物制剂登记处如何在临床试验中的疗效与真实世界中的有效性之间弥合证据差距，重点介绍了登记处的方法学方面、其独特之处和挑战，以及未来的作用。
