Clinical Evidence to Guide the Management of Diabetic Macular Oedema
abstract
This abstract is available on the publisher's site.
Access this abstract now Full Text Available for ClinicalKey SubscribersRandomised 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.
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From randomised controlled trials to real-world data: Clinical evidence to guide management of diabetic macular oedema
Prog Retin Eye Res 2023 Nov 01;97(14)101219, PH Gabrielle, H Mehta, D Barthelmes, V Daien, V Nguyen, MC Gillies, CP Creuzot-GarcherFrom MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.
This review article showed that real-world evidence has provided valuable insights into the optimal management of diabetic macular oedema (DMO). A key insight from real-world data (RWD) was the effectiveness and safety of key intravitreal drugs, such as VEGF inhibitors and steroids, in routine clinical practice for the treatment of patients with DMO. However, the authors noted that, "even if treatment outcomes have significantly improved with time, related to better knowledge and change in practice patterns, a ceiling effect appears to have been reached in what can be achieved in terms of effectiveness using approved therapies for DMO in the real world, which remains inferior to that observed in randomised clinical trials." As we all recognise, there may be more variability in how treatment is administered in real-world settings, resulting in poorer results, as patients who are sicker and have more comorbidities may not receive the same amount of treatment and monitoring as those in clinical trials.
The authors pointed out that real-world evidence shows that earlier treatment and intensive and personalised dosing, such as a treat-and-extend regimen, can refine long-term outcomes in patients with DMO while optimising the number of injections and visits. Novel targeted therapeutics or promising new longer-acting drug agents or devices, such as the port delivery system, are under investigation. RWD will confirm whether they are effective in daily practice and provide valuable safety data regarding rare ocular adverse events.
The authors concluded by noting that, "RWD analysis will benefit from new powerful technologies, such as artificial intelligence, big data, and software that supports dataset linkage, which will enhance the ability to analyse RWD to aid diagnosis and treatment decisions and provide prognostic information to further improve the management of DME."