Groundbreaking research, presented at the Association for Research in Vision and Ophthalmology’s annual meeting in 2021, demonstrated that it is possible to successfully use real world data (RWD) to replicate the primary outcome measures of randomized controlled trials (RCTs) .
The study from Verana Health, a health technology and analytics company, focused on two major RCTs in ophthalmology – the pivotal studies VIEW 1 and VIEW 2 (VIEW 1/2). VIEW 1/2 led to the 2011 release of aflibercept (EYLEA, Regeneron) by the US Food and Drug Administration for the treatment of neovascular age-related macular degeneration (wet AMD) in 2011.
In this electronic health record-based enrollment study, researchers replicated the study design and assessed the results using RWD, which is included in the American Academy of Ophthalmology’s (Intelligent Research in Sight) IRIS registry, the world’s largest clinical specialty database. The same inclusion and exclusion criteria of the VIEW 1/2 studies were successfully applied to the IRIS registry and resulted in the identification of 4,779 patients who could be compared to the 1,632 subjects in the VIEW 1/2 studies. The proportion of eyes that lost fewer than 15 ETDRS letters in the VIEW 1 and VIEW 2 studies was similar to that in the RWD cohort of the IRIS registry, which indicates a proof-of-concept, although the average increases in the letters in the studies were different.
“So far we did not know whether the data and results of the phase III clinical trial were in the [RWD] included in the IRIS register, ”said Dr. Theodore Leng, Verana Health Medical Advisor and Director of Clinical and Translational Research at Stanford Byers Eye Institute, Stanford University Medical School. “We are pleased to note that in this retrospective study, primary outcome measures were reproducible in the IRIS registry. This was the first time that a large-scale phase III was carried out [RCT] in ophthalmology could be replicated with [an RWD] to adjust. This has exciting implications for clinicians, investigators, and drug and device development companies, including the potential to validate study endpoints as a measure of treatment effectiveness. “
Replicating clinical trial results with VIEW 1/2 shows the potential value of RWD in accelerating clinical trials, added Dr. David W. Parke II, CEO of the American Academy of Ophthalmology, added.
Edited by Gary Cramer