A Drug to Prevent Pediatric Myopia-What Would it Take?

Because of the epidemic of myopia with both its short-term and long-term effects, we desperately need ways to slow myopic progression. In this study which was part of a myopia prevention symposium, the author answers the following question: Assuming that researchers did come up with a pharmacological treatment to slow myopic progression-what would it take to obtain regulatory approval from the United States Food and Drug Administration (FDA)? Previous publicly available information (a 2003 FDA advisory committee on this topic, International Conference on Harmonisation guidances on drug development, and published articles) as well as the author's experience is used. Development pathways including preclinical safety, chemistry, manufacturing and controls, and early- and late-stage clinical studies are presented. In particular, challenges for the conduct of multi-year controlled double-masked studies are presented. As any treatment would have to be chronic, prophylactic, and pediatric, there are a host of concerns as to efficacy, safety, and benefit/risk (therapeutic index). Although more challenging than some short-term indications in ophthalmology, nonetheless the pharmaceutical community is investing in seemingly equally challenging conditions such as retinal degeneration. The author looks forward to working with colleagues in academia and industry to evaluate and develop novel therapies to slow the development of myopia.