Clinical Trial Evidence and Outcomes in Wet AMD


Diana Do, MD, discusses outcomes of interest for anti-VEGF agents, clinical evidence supporting the treat-and-extend approach, and strategies to overcome financial barriers in wet AMD.

Diana Do, MD: When we’re looking at evidence from wet AMD [age-related macular degeneration], the most important clinical parameter that we use to guide whether an agent is effective is currently the visual acuity. In all these phase 3 clinical trials, the primary end point of these studies is the best-corrected visual acuity obtained over a 1-year period. The FDA accepts that outcome as its main point of determining whether an agent is efficacious. Of course, there are also secondary end points that are evaluated, such as retinal thickness or quality-of-life measures. But the most important end point is visual acuity.

In many of these clinical trials, patient reported outcomes such as quality of life is also important. Quality of life is obviously affected by the patient’s visual acuity. If a patient does not receive an effective therapy for their age-related macular degeneration, then the vision declines and the quality of life also declines. Therefore, quality of life and patient reported outcomes, is almost a surrogate marker for how their vision is doing over time with their therapy.

Over the past decade, there have been many randomized clinical trials that have evaluated the treat-and-extend regimen for wet age-related macular degeneration. The vast majority of these clinical trials have shown that the treat-and-extend regimen is noninferior to fixed monthly or every-2-month dosing of FDA-approved intravitreal VEGF inhibitors. For example, the ALTAIR study, which was conducted overseas, evaluated a treat-and-extend regimen of aflibercept compared with aflibercept given every 2 months on a fixed regimen. In this clinical trial, eyes that were randomized to treat and extend, had noninferior visual acuity outcomes compared with fixed dosing. In addition, there was a slight reduction in the number of treatments given over a 1 to 2-year period. In addition, other clinical trials have evaluated the treat-and-extend regimen for ranibizumab and showed similar results of noninferior visual acuity gains and a slight reduction in intravitreal injections.

For the treatment of wet age-related macular degeneration, many of these intravitreal VEGF inhibitors that are FDA approved are expensive. With proper insurance coverage, there are no significant financial barriers for patients who have adequate coverage. Of course, many patients who don’t have significant medical insurance will be treated by off-label bevacizumab, which is very cost-effective for the treatment of this ocular disease. If there’s more open access to these medicines and adequate coverage, most retina specialists will elect to choose the FDA-approved medicines, which might have slight efficacious benefits and more durability benefits compared with off label bevacizumab.

Transcript edited for clarity.

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