Ligthelm Robert J, Borzì Vito, Gumprecht Janusz, Kawamori Ryuzo, Wenying Yang, Valensi Paul
Executive Health Management Clinic, Hoofddorp, Rotterdam, The Netherlands.
Clin Ther. 2007;29 Spec No:1284-92.
In this era of evidence-based medicine, clinicians require a comprehensive range of well-designed studies to support prescribing decisions and patient management. In recent years, data from observational studies have become an increasingly important source of evidence because of improvements in observational-study methods and advances in statistical analysis.
This article reviews the current literature and reports some of the key studies indicating that observational studies can both complement and build on the evidence base established by randomized controlled trials (RCTs).
A literature search using the MEDLINE/ PubMed database (years: 1966-present) was carried out using the search terms observational or observational study(ies), historical control, nonrandomized, and postmarketing surveillance. All references comparing observational studies with randomized controlled trials were obtained and reviewed and were also hand-checked for studies not identified in the database searches.
Observational studies play an important role in investigating treatment outcomes. Data from large observational studies can clarify the tolerability profile of marketed medicines. In particular, observational studies can be of benefit in the study of large, heterogeneous patient populations with complex, chronic diseases such as diabetes mellitus. Observational studies have played a key role in supporting the results of Phase III studies of insulin analogues for the treatment of patients with type 1 and type 2 diabetes. Future observational studies in the field of diabetes such as PREDICTIVE (Predictable Results and Experience in Diabetes through Intensification and Control to Target: an International Variability Evaluation) and IMPROVE will further our understanding of this global pandemic.
Well-designed observational studies can play a key role in supporting the evidence base for drugs and therapies. Current evidence suggests that observational studies can be conducted using the same exacting and rigorous standards as are used for RCTs. The observational study design should be considered as a complementary rather than a rival analytic technique.
在这个循证医学时代，临床医生需要一系列精心设计的研究来支持处方决策和患者管理。近年来，由于观察性研究方法的改进和统计分析的进展，来自观察性研究的数据已成为越来越重要的证据来源。
本文回顾了当前文献，并报告了一些关键研究，这些研究表明观察性研究既可以补充随机对照试验（RCT）建立的证据基础，也可以在此基础上进一步拓展。
使用MEDLINE/PubMed数据库（年份：1966年至今）进行文献检索，检索词为观察性或观察性研究、历史对照、非随机和上市后监测。获取并审查了所有比较观察性研究与随机对照试验的参考文献，还对数据库搜索中未识别的研究进行了人工核对。
观察性研究在调查治疗结果方面发挥着重要作用。大型观察性研究的数据可以阐明上市药品的耐受性概况。特别是，观察性研究在研究患有复杂慢性疾病（如糖尿病）的大型异质性患者群体时可能会有所帮助。观察性研究在支持胰岛素类似物治疗1型和2型糖尿病患者的III期研究结果方面发挥了关键作用。未来在糖尿病领域的观察性研究，如PREDICTIVE（通过强化和控制目标实现糖尿病的可预测结果和经验：一项国际变异性评估）和IMPROVE，将进一步加深我们对这一全球大流行病的理解。
精心设计的观察性研究可以在支持药物和疗法的证据基础方面发挥关键作用。目前的证据表明，观察性研究可以采用与RCT相同严格的标准进行。观察性研究设计应被视为一种补充性而非竞争性的分析技术。
