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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.

Author information

1
Newcastle upon Tyne Hospitals NHS Foundation Trust, Newcastle upon Tyne, UK. Electronic address: James.Talks@nuth.nhs.uk.
2
Universitary Hospital of Montpellier, Montpellier, France.
3
Universitäts-Augenklinik Bonn, Bonn, Germany.
4
Hacettepe University, Ankara, Turkey.
5
Kagoshima University Graduate School of Medical and Dental Sciences, Kagoshima, Japan.
6
University of São Paulo, São Paulo, Brazil.
7
Sydney West Retina, Sydney, Australia.
8
Singapore National Eye Centre, Duke-NUS Medical School, National University of Singapore, Singapore.
9
Service d'ophtalmologie, CHU Bordeaux, Bordeaux, France; University of Bordeaux Population Health Research Center, team LEHA, UMR 1219, F-33000 Bordeaux, France.

Abstract

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.

KEYWORDS:

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

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