Bridging new horizons for ophthalmology: State of the art methodologies and outlook

Developing ophthalmic drugs is challenging, in part due to limitations on sampling drug concentrations and biomarker levels at the site of action within the eye. Oftentimes, pre-clinical exposure-PD/efficacy/safety relationships do not necessarily translate into humans. Despite various challenges, there is a vast amount of longitudinal clinical data, real world data, and retinal imaging data available. Hence, there are many unique opportunities in ophthalmology, in which different tools can be used to address critical questions throughout different stages of drug development. In this talk, we will present a few examples on how knowledge, data and analytics can be seamlessly integrated throughout different development stages to help inform ophthalmic drug development. One of the examples is the ophthalmology PK-PD platform model developed to mechanistically describe drug PK and PD based on data from multiple therapeutic programs in nonclinical species and humans. There are also examples of disease progression models developed to describe and predict the natural progression of disease trajectories in the absence of treatment or with treatment. In addition, there are examples of exposure-efficacy models developed using longitudinal clinical data to predict clinical endpoints. We are optimistic that these state-of-the-art methodologies and tools can be integrated effectively and efficiently and offer a new horizon for ophthalmic drug development.