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Surv Ophthalmol. 2019 Sep - Oct;64(5):707-719. doi: 10.1016/j.survophthal.2019.02.008. Epub 2019 Feb 22.

The use of real-world evidence for evaluating anti-vascular endothelial growth factor treatment of neovascular age-related macular degeneration.

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Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK. Electronic address:
Universitary Hospital of Montpellier, Montpellier, France.
Universitäts-Augenklinik Bonn, Bonn, Germany.
Hacettepe University, Ankara, Turkey.
Kagoshima University Graduate School of Medical and Dental Sciences, Kagoshima, Japan.
University of São Paulo, São Paulo, Brazil.
Sydney West Retina, Sydney, Australia.
Singapore National Eye Centre, Duke-NUS Medical School, National University of Singapore, Singapore.
Service d'ophtalmologie, CHU Bordeaux, Bordeaux, France; University of Bordeaux Population Health Research Center, team LEHA, UMR 1219, F-33000 Bordeaux, France.


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.


clinical practice; effectiveness; neovascular age-related macular degeneration; noninterventional; ophthalmology; real-life; real-world; retinal disease

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