From randomised controlled trials to real-world data: Clinical evidence to guide management of diabetic macular oedema

Pierre-Henry Gabrielle; Hemal Mehta; Daniel Barthelmes; Vincent Daien; Vuong Nguyen; Mark C Gillies; Catherine P Creuzot-Garcher

doi:10.1016/j.preteyeres.2023.101219

From randomised controlled trials to real-world data: Clinical evidence to guide management of diabetic macular oedema

Prog Retin Eye Res. 2023 Nov:97:101219. doi: 10.1016/j.preteyeres.2023.101219. Epub 2023 Oct 26.

Authors

Pierre-Henry Gabrielle¹, Hemal Mehta², Daniel Barthelmes³, Vincent Daien⁴, Vuong Nguyen⁵, Mark C Gillies⁵, Catherine P Creuzot-Garcher⁶

Affiliations

¹ Department of Ophthalmology, Dijon University Hospital, Dijon, Burgundy, France; The Save Sight Institute, Sydney Medical School, The University of Sydney, Sydney, New South Wales, Australia.
² The Save Sight Institute, Sydney Medical School, The University of Sydney, Sydney, New South Wales, Australia; Ophthalmology Department, Royal Free London NHS Foundation Trust, London, United Kingdom.
³ The Save Sight Institute, Sydney Medical School, The University of Sydney, Sydney, New South Wales, Australia; Department of Ophthalmology, University Hospital and University of Zurich, Zurich, Switzerland.
⁴ The Save Sight Institute, Sydney Medical School, The University of Sydney, Sydney, New South Wales, Australia; Department of Ophthalmology, Montpellier University Hospital, Montpellier, France; Institute for Neurosciences of Montpellier, Univ Montpellier, INSERM, Montpellier, France.
⁵ The Save Sight Institute, Sydney Medical School, The University of Sydney, Sydney, New South Wales, Australia.
⁶ Department of Ophthalmology, Dijon University Hospital, Dijon, Burgundy, France. Electronic address: catherine.creuzot-garcher@chu-dijon.fr.

PMID: 37898362
DOI: 10.1016/j.preteyeres.2023.101219

Abstract

Randomised clinical trials (RCTs) are generally considered the gold-standard for providing scientific evidence for treatments' effectiveness and safety but their findings may not always be generalisable to the broader population treated in routine clinical practice. RCTs include highly selected patient populations that fit specific inclusion and exclusion criteria. Although they may have a lower level of certainty than RCTs on the evidence hierarchy, real-world data (RWD), such as observational studies, registries and databases, provide real-world evidence (RWE) that can complement RCTs. For example, RWE may help satisfy requirements for a new indication of an already approved drug and help us better understand long-term treatment effectiveness, safety and patterns of use in clinical practice. Many countries have set up registries, observational studies and databases containing information on patients with retinal diseases, such as diabetic macular oedema (DMO). These DMO RWD have produced significant clinical evidence in the past decade that has changed the management of DMO. RWD and medico-administrative databases are a useful resource to identify low frequency safety signals. They often have long-term follow-up with a large number of patients and minimal exclusion criteria. We will discuss improvements in healthcare information exchange technologies, such as blockchain technology and FHIR (Fast Healthcare Interoperability Resources), which will connect and extend databases already available. These registries can be linked with existing or emerging retinal imaging modalities using artificial intelligence to aid diagnosis, treatment decisions and provide prognostic information. The results of RCTs and RWE are combined to provide evidence-based guidelines.

Keywords: Database; Diabetic macular oedema; Randomized clinical trials; Real-world data; Real-world evidence; Registry.

Publication types

Review

MeSH terms

Diabetes Mellitus*
Diabetic Retinopathy* / diagnosis
Diabetic Retinopathy* / therapy
Humans
Laser Coagulation / methods
Macular Edema* / diagnosis
Macular Edema* / therapy
Randomized Controlled Trials as Topic
Retina