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Real-World Data Shown to Match Eye Disease Trial Results

Fundus photograph

Fundus photograph of a healthy right eye (OptometrusPrime, Wikimedia Commons)

19 Jan. 2023. Research findings show data in patients’ electronic health records can be collected and analyzed to replicate results from late-stage clinical trials of an eye disease biologic drug. Results of the study conducted by a team from Stanford University and life science and health analytics company Verana Health in San Francisco, appear in the 1 Jan. issue of the journal Ophthalmic Surgery, Lasers and Imaging or OSLI Retina.

Verana Health collects and analyzes real-world health data from databases the company says represent some 90 million patients generated by de-identified records from 20,000 health care providers and medical associations in ophthalmology, neurology, and urology. The company says its analytical engine uses artificial intelligence to transform structured and unstructured data in electronic records to produce curated disease-specific data modules. Verana also provides its analytical software as a service to generate real-world medical evidence, assist in clinical trials, and manage data in medical registries.

In the study, a team from Stanford and Verana Health tested a data set from de-identified health records of patients with neovascular or so-called wet age-related macular degeneration or AMD, a common eye disease among older individuals where the retina deteriorates, leading to loss of visual acuity. The retina is a layer of light-sensitive tissue in the back of the eye, where images are focused and converted to electrical impulses sent through the optic nerve to the brain. With wet AMD, abnormal growth of blood vessels occur under the retina that bleed or leak, causing distortion in the retina’s structure.

In the previous decade, a number of new treatments for wet AMD emerged that control the abnormal development of blood vessels in the eye. These new drugs block a protein called vascular endothelial growth factor or VEGF that encourages blood vessel growth. Among the new drugs authorized by FDA to control VEGF for eye diseases are aflibercept, a biologic drug marketed as Eylea by Regeneron and ranibizumab, marketed as Lucentis by Genentech. Both drugs are injected periodically into the eyes.

Health data records from eye patient registry

Regeneron tested aflibercept in two late-stage clinical trials, first among wet AMD patients in the U.S. and Canada from 2007 to 2011, and in Europe, Asia, South America, and Australia from 2008 to 2011. In both trials, participants were randomly assigned to receive aflibercept , an experimental drug at the time, or a then-current treatment ranibizumab for comparison.

The Stanford/Verana Health team drew on health data of patients in the Intelligent Research in Sight or IRIS Registry maintained by American Academy of Ophthalmology. The researchers sought records of patients receiving injections of aflibercept or ranibizumab from Jan. 2013 through Dec. 2018. From that population, the team sought to match patients in the IRIS registry to eligibility and treatment conditions in the clinical trials.

Results show of the 90,900 patients with IRIS registry records meeting the clinical trial eligibility criteria, 4,457 or about five percent also matched the treatment group requirements. Of those patients meeting both eligibility and treatment group criteria, 90 percent maintained vision after one year. And, differences among the treatment groups — those taking aflibercept monthly or bi-monthly, or ranibizumab monthly — were not large enough for statistical reliability.

Even though the percentage of registry patients meeting both eligibility and treatment criteria was only five percent of the original cohort, the authors conclude this test of real-world data generates results replicating the endpoints in the original clinical trials. Plus, Verana Health believes these real-world data can support submissions to regulatory agencies, reducing time and cost of bringing new drugs and medical devices to market.

“This was a great pilot experiment to show that this type of analysis was possible,” says Stanford University ophthalmology professor and the paper’s senior author Theodore Leng in a Verana Health statement. “The next step is to determine whether we can replicate results in other trials and develop confidence in real-world data as a foundation from which we can scale.”

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