Abstract
Purpose :
We surveyed industry and academic leaders to determine the most predictive functional endpoint for evaluating therapies for geographic atrophy. There are many endpoints to evaluate the efficacy of potential therapeutics for geographic atrophy. It is unclear which endpoint can be used to best predict an intervention's ability to improve the quality of life of those with geographic atrophy.
Methods :
We surveyed executives and directors at biotechnology companies and academic centers in North America and Europe convened during the 2023 Dry Age-Related Macular Degeneration Therapeutic Development Summit, about endpoints for geographic atrophy. The survey was conducted with online polling software (FeedBeo) and taken on personal handheld devices.
Results :
Fourteen people participated in the survey. Of them, 64% were pharmaceutical professionals, 18% practicing physicians, 9% academic researchers, and 9% were from non-profit organizations. More than half of the participants from the pharmaceutical industry had sponsored a trial in Dry Age-Related Macular Degeneration or geographic atrophy. When asked which functional endpoints they had previously seen in geographic atrophy trials, Best Corrected Visual Acuity was the most frequent answer (33%). Microperimetry was the top response to the question of which functional endpoint could best predict an intervention's ability to improve the quality of life of those living with geographic atrophy, chosen by 36% of respondents (Table 1), followed by Best Corrected Visual Acuity (BCVA) (18%), Low Luminance Visual Acuity (LLVA) (18%), Low Luminance Deficit (LLD), (9%), multifocal electroretinogram (9%), and dark adaptation (9%); non selected were reading speed or patient-reported outcomes.
Conclusions :
Microperimetry was the top response of surveyed industry and academic leaders as most promising functional endpoint for evaluating treatments in geographic atrophy. Although challenging to perform both by site personnel and patients, prioritizing microperimetry might be helpful. Interestingly, reading speed and patient-reported outcomes, which are typically integrated in clinical trials, were not selected as relevant functional endpoints. Limitations of this research is its small sample size and its demographics composed in majority of pharmaceutical professionals.
This abstract was presented at the 2024 ARVO Annual Meeting, held in Seattle, WA, May 5-9, 2024.