Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK.
Universitary Hospital of Montpellier, Montpellier, France.
Surv Ophthalmol. 2019 Sep-Oct;64(5):707-719. doi: 10.1016/j.survophthal.2019.02.008. Epub 2019 Feb 22.
Randomized controlled trials are the gold standard in medical research, providing evidence of the efficacy of a treatment in well-defined patient populations. By contrast, real-world studies explore the effectiveness of treatments in routine clinical practice, often with diverse patient populations. Although both randomized controlled trials and real-world studies contribute to the understanding of the benefits and risks of therapies, they generate different types of data and serve complementary purposes. Real-world studies evaluating the management of neovascular age-related macular degeneration have shown that visual outcomes achieved with anti-vascular endothelial growth factor in clinical practice often differ from those derived from clinical trials, highlighting the importance of assessing such outcomes in real-world studies. Benefits include finding variations in treatment provision, leading to: service improvements; the understanding of the need for continued and higher than previously provided treatment frequency; and new treatment regimens such as treat-and-extend. There is potential for the scope of real-world studies to be expanded to include other patient outcomes, such as quality of life, thus providing decision-makers with additional information to complement the data collected in randomized controlled trials. Physicians, patients, and regulators stand to gain much from further development and the conduct of real-world studies. We provide an overview of the importance of real-world evidence in the management of neovascular age-related macular degeneration with anti-vascular endothelial growth factor therapy, describe sources of real-world evidence, and assess the relative strengths and limitations of randomized controlled trials and real-world studies.
随机对照试验是医学研究的金标准，为明确界定的患者群体中治疗效果提供了证据。相比之下，真实世界研究则在常规临床实践中探索治疗的有效性，通常涉及不同的患者群体。尽管随机对照试验和真实世界研究都有助于了解治疗的益处和风险，但它们产生的数据类型不同，目的也不同。评估抗血管内皮生长因子治疗新生血管性年龄相关性黄斑变性管理的真实世界研究表明，临床实践中抗血管内皮生长因子治疗的视觉结果通常与临床试验得出的结果不同，这凸显了在真实世界研究中评估此类结果的重要性。益处包括发现治疗提供方面的差异，从而导致：服务改进；了解需要持续和高于以前提供的治疗频率的必要性；以及新的治疗方案，如治疗和延长。真实世界研究的范围有可能扩大到包括其他患者结果，如生活质量，从而为决策者提供补充随机对照试验数据的额外信息。医生、患者和监管机构将从真实世界研究的进一步发展和实施中受益匪浅。我们概述了在抗血管内皮生长因子治疗新生血管性年龄相关性黄斑变性管理中真实世界证据的重要性，描述了真实世界证据的来源，并评估了随机对照试验和真实世界研究的相对优势和局限性。
