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

Journal Article (Journal Article;Review)

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.

Full Text

Duke Authors

Cited Authors

  • Talks, J; Daien, V; Finger, RP; Eldem, B; Sakamoto, T; Cardillo, JA; Mitchell, P; Wong, TY; Korobelnik, J-F

Published Date

  • September 2019

Published In

Volume / Issue

  • 64 / 5

Start / End Page

  • 707 - 719

PubMed ID

  • 30797883

Electronic International Standard Serial Number (EISSN)

  • 1879-3304

Digital Object Identifier (DOI)

  • 10.1016/j.survophthal.2019.02.008


  • eng

Conference Location

  • United States