现实世界研究生态圈的构建与发展

现实世界证据（real-world evidence，RWE）由现实世界数据（real-world data，RWD）产生，经现实世界研究（real-world study，RWS）转化而来。科学的RWE可为决策提供参考依据，其产生有赖于系统性地构建和发展良性生态圈。本文梳理了RWD、RWS在产生RWE中的角色及转化机制，试图探讨构建现实世界研究良性生态系统的必要性及构成要素，并简要展望生态圈的发展趋势、机遇及挑战。     

产生现实世界证据的现实世界数据应用现状及展望

现实世界数据（real-world data，RWD）在医药卫生领域的应用日益广泛。在RWD转化为现实世界证据（real-world evidence，RWE）的过程中也涌现了大量问题，其中主要关注点在于RWD相关的数据质量和数据标准问题。本文着眼于RWD来源、数据合规性，讨论其数据标准及其质量控制问题，并对其未来可能的发展途径作一展望。     

对中医药领域开展循证医学研究的几点看法

近年来，随着循证医学在世界范围内的迅速发展，国内外学者对中医药学是否需要用循证医学的标准来评价，以及如何发展循证的中医药学进行了广泛而热烈地讨论。我们的观点是：中医药学需要循证医学；循证医学认可的证据并不仅仅是大样本的随机双盲对照试验和系统评价；中医药学的理论体系与临床诊疗有许多不同于现代医学的特点，需要针对这些特点进行方法学的创新性研究。本文根据作者近年来在中医药领域进行方法学培训与临床研究的经验，从完善临床试验流程、规范临床研究报告、系统评价当前的中医药临床研究、开展方法学研究、以及证据管理和资源优化整合等五个方面对如何在中医药领域进一步开展循证医学研究进行了讨论。     

效果-实施双轨设计的分类和应用

将证据转化为卫生保健服务,需要经过3个转化过程“临床前研究-临床研究-实施性研究”,耗时漫长且常常出现转化不足。为加快证据转化为日常医疗实践和卫生政策的进程,融合了临床有效性研究和实施性研究的“效果-实施双轨设计”应运而生。本文介绍效果-实施双轨设计的概念、分类及其应用,为研究人员应用此方法开展研究提供参考。     

真实世界证据

真实世界证据是在现实医疗环境中,通过优化分析来源于多种途径的数据而获得的证据。其与传统临床研究证据的本质区别在于获取数据的环境,而非研究方法或试验设计。与传统随机对照临床试验证据相比,真实世界证据具有结果外推性好、研究效率高等优势,可广泛应用于医疗产品有效性和安全性评价、临床实践过程分析等领域。近年来,随着信息技术的飞速发展和医学循证需求的持续增加,真实世界研究愈发受到人们重视,发展前景良好。     

在神经科领域应用循证医学

因为在神经科领域也存在以下问题： (1)一些有证据证明有效的医疗措施没有及时推广 [如脑卒中单位 (stroke unit)],而一些尚无证据证明有效的措施却被广泛使用 (例如在缺血性脑卒中急性期，国外常使用肝素，国内常静脉使用尼莫地平等 )； (2)以病人为中心的、高质量的临床研究证据不多，大量临床研究的质量有待提高； (3)第一线的临床医生获取最新最佳研究证据困难等等。因此神经科的临床研究者有责任去进行高质量的研究，为神经疾病的临床决策提供真实可靠的科学依据，并使之容易获得；神经科的临床医生有责任去应用高质量的研究结果，使自…     

循证医学(evidence based medicine,EBM)

循证医学 (遵循证据的医学 )指医生对病人的诊断、治疗预防、康复和其他决策应建立在当前最佳临床研究证据、临床专业知识技能及病人的需求三者有机结合的基础之上。 最佳临床研究证据指与临床密切相关的研究，包括对诊断试验准确性和精确性的研究；对预后因素预测强度的研究；对治疗、康复和预防措施效果及安全性的研究等等。新的、更好的证据常常推翻和代替以前的证据。医学研究非常活跃，很少有永恒不变的“真理”，现代临床医生应终生学习，随时更新知识，跟踪本领域最新研究进展，才能保证为病人提供高质量的医疗服务。临床专业知识技…     

知识转化的概念及理论框架

知识转化是将临床研究证据或以研究证据为依据的临床实践指南运用于临床实践当中,改善临床实践,促进执行者行为改变,提高患者服务质量。知识转化在国外发展迅速,而国内对知识转化的认识刚起步。为了促进知识转化在我国的发展,本文将重点阐述知识转化的概念及理论框架等内容,探讨其对临床实践的重要价值。     

用循证医学指导脑血管病的治疗

循证医学(evidence—based medicine，EBM)是近年来国际临床医学领域迅速兴起的研究热点，其中心思想是负责、明确、明智地利用已有最好证据来决定每个病人的治疗。对于每个病人的临床决策，都应建立在最佳临床研究证据、临床专业知识技能及病人的需求三者有机结合的基础之上。以脑血管病的治疗为例，为遵从循证医学的规律，神经内科医师需进行如下工作。     

我国护理领域证据临床转化研究的范围综述

目的:探讨我国护理领域内证据临床转化研究的现状、所依据的证据质量、研究方法及研究内容。方法:使用"证据转化""证据应用"等检索词在中国生物医学文献数据库、中国知网、万方数据库、维普中文科技期刊数据库中进行检索,应用Arksey和O’Malley的范围综述方法对文献进行分析。结果:共纳入152篇文献,95.4%的文献发表于2015年之后,92.1%的文献发表在护理类期刊上,27.0%的文献提供了系统的检索策略,43.3%的文献进行了方法学质量评价,74.3%的文献均没有对证据的可用性进行评价。证据转化以症状护理为主题的文献最多,占39.5%,87.5%的文献采用了前后对照方法来评价证据转化的效果,所有的研究均构建了综合性、多元化的证据转化策略,包括基于证据(42.1%)和基于障碍因素的干预策略(57.9%)。73.7%的文献采用了多元化指标对效果进行评价,仅有7.2%的文献提出了应维持证据在临床的持续应用。结论:我国护理领域内证据临床转化研究数量上呈现明显的增加趋势,但转化所依据的证据质量及研究方法尚需要加强,应采取策略推动证据在实践的持续应用。     

The Case for Real-world Evidence in the Future of Clinical Research on Chronic Myeloid Leukemia

Purpose

In light of recently published guidelines from the US Food and Drug Administration (FDA) on the communication of real-world data (RWD) and real-world evidence (RWE) to support regulatory decision making, it is important to understand how such data are developed, the limitations of these data, and how to best use RWD to improve patient care. Historically, the use of RWE has been approached with skepticism because of its often-retrospective nature compared with data from conventional randomized controlled trials (RCTs). This review discusses the role and function of RWE and RWD in clinical research. We summarize the types of RWE used in clinical research, outline the challenges and limitations involved with these data, and suggest how these types of analyses can supplement results from clinical trials to foster a more complete understanding of a drug or disease area of interest. In particular, we focus on the role of RWE in investigating chronic myeloid leukemia (CML) and tyrosine kinase inhibitor therapy for CML.

Routinely collected data as real-world evidence for physiotherapy practice

Evidence-based practice is the current undisputed predominant paradigm within medicine and allied health care, particularly in physiotherapy. Despite its potential benefits, over the years various points of criticism have been formulated one of which is the overreliance on randomized clinical trials as the highest level of evidence for treatment effectiveness. In the current era, where the availability of large amounts of clinical data gathered during the course of care delivery is rapidly increasing as well as our ability to access, process, link, and analyze these data in fairly efficient ways, alternative sources to supplement rather than replace evidence from RCTs look promising. In this Editorial, we discuss the opportunities and limitations of these routinely collected data in physiotherapy research and provide several examples from the literature. We conclude that the use of routinely collected data in physiotherapy research has the potential to increasingly contribute to real-world evidence, particularly in musculoskeletal primary care physiotherapy, provided that researchers are aware of methodological limitations and adhere to reporting standards.     

Ustekinumab in psoriatic arthritis: need for studies from real-world evidence

Ustekinumab (UST) is a recently approved drug for the treatment of psoriatic arthritis (PsA). The ACR response rates in randomized clinical trials (RCTs) with this drug have been slightly lower than that reported in RCTs of anti-TNF and anti-IL17 therapies. Therefore, the position that this drug may occupy in the treatment algorithms of PsA is not clear. More information is needed on the true efficacy of this agent under real clinical practice conditions. In this review of real-world evidence studies, it is shown that UST is effective and safe to treat PsA; nevertheless, it is necessary to homogenize the way in which the main outcomes and treatment objectives of these studies are presented.     

Effectiveness of omalizumab in adolescent and adult patients with chronic idiopathic/spontaneous urticaria: a systematic review of ‘real-world’ evidence

Introduction: Chronic idiopathic/spontaneous urticaria (CIU/CSU) is a dermatological condition characterized by itchy wheals and/or angioedema of continuous or intermittent duration of ≥6 weeks with a high burden of disease and impact on quality of life. Omalizumab is a recombinant humanized monoclonal antibody that inhibits the binding of IgE to high affinity receptors, and is approved for the CIU/CSU indication. The objective of this systematic review was to evaluate and synthesize the evidence on the real-world effectiveness of omalizumab in CIU/CSU in daily clinical practice.

Areas covered: This review of 84 observational effectiveness studies covers treatments (dosing, medication use), clinical outcomes (treatment response, disease activity, quality of life), and safety.

Expert opinion: The clinical outcomes observed across studies underscore the real-world effectiveness of omalizumab in the management of CIU/CSU. Continued treatment may assist patients showing an initial response to achieve a complete treatment response. Response rates are aligned with observed changes in disease activity, symptom experience, and quality of life, and this across subtypes of CIU/CSU. The positive therapeutic profile is complemented by a positive safety profile. The real-world evidence summarized here points convincingly at the high degree of effectiveness of omalizumab in the treatment of CIU/CSU in daily clinical practice.     

Lessons learned in translating research evidence on early intervention programs into clinical care

There is research evidence describing quality early intervention programs that can promote positive child health and behavior and enhance parenting behaviors and parent-child interactions. Two of the programs with persuasive research evidence are home visitation and parent training. Despite the evidence, it seems that few of these strategies have been translated to real-world clinical settings. This article reviews an evidence-based practice framework that highlights the influence of the research evidence, patient preferences, provider expertise, setting-specific contextual factors, and important role of a facilitator. Examples from one particular early intervention project that implemented a standardized parent training program via home visiting the families of 2- to 6-year-old children help explicate the facilitators and pitfalls in translating best practices into the real-world settings. Strategies that can lead to successful implementation of early intervention child health and parenting best practices in real-world clinical settings are recommended.     

Drug Discontinuation in Studies Including a Switch From an Originator to a Biosimilar Monoclonal Antibody: A Systematic Literature Review

Purpose

In observational studies of patients switched from stable treatment with an originator monoclonal antibody (mAb) to a biosimilar, higher rates of biosimilar discontinuation versus those observed in blinded switching studies have been reported. Because this observation relates to the real-world setting, it has been suggested that switching outside of clinical trials may be associated with nocebo effects. However, real-world data on drug discontinuation and nocebo effects after switching to mAb biosimilars remain limited. This systematic review collated information from switching studies regarding discontinuation rates of biosimilar mAbs and investigated the subjectivity of reasons for discontinuation to determine the impact of potential nocebo responses.

基于真实世界数据的疾病管理研究

近年来, 随着信息技术快速发展和对循证临床实践和决策的需求急剧增加, 真实世界数据在医疗健康领域的作用日益受到重视。基于真实世界数据的疾病管理研究已成为重要发展方向, 其核心是针对疾病负担、诊断、临床预防和治疗、预后管理等多个方面的疾病管理问题, 在严谨的、预先设定的设计基础上, 基于海量、多源和高质量数据开展科学的数据分析和处理, 最终将基于这些研究证据形成疾病管理的指南、路径和技术规范, 从而提高医疗质量和安全。本文系统概述了基于真实世界数据开展疾病管理研究的一些重要问题。     

Implementing evidence in clinical practice: the ‘therapies’ dilemma

Evidence-based practice involves the application of the best available evidence, often from research findings, into the clinical setting to ensure best practice. This paper discusses some of the issues faced by allied health therapists when attempting to integrate research evidence into their own clinical practice. Of particular concern in allied health research are the methodological flaws, which may seriously affect the capacity of the allied health therapist to use the evidence in their clinical practice. Single therapy intervention research, or research based on an occasion of service does not replicate the model of clinical reasoning that underpins the clinical process, nor the notion of an episode of care that characterizes the allied health therapies. Other issues such as the relevance of outcome measures used in allied health research to potential stakeholders, and the differences between therapy diagnostic criteria and research diagnostic criteria used will also adversely affect the capacity for therapists to use the research evidence. It is imperative that research paradigms begin to address the therapy paradigms, allowing research evidence to reflect the real-world situation. Allied health therapists must take a pro-active stance in the process, through undertaking organized reflective practice and forging links with researchers. Barriers associated with the implementation of evidence-based practice in the allied health professions may be reduced with the publishing of relevant clinically directed research.