Affiliation:
1. Scientific Centre for Expert Evaluation of Medicinal Products,
Abstract
Until now, there have been no effective treatments for some ophthalmic diseases that have high social significance. Development of therapeutic approaches to such diseases may be complicated due to challenges in diagnosis and selection of clinical trial endpoints. The aim of the study was to analyse current approaches to selection of endpoints in clinical trials of ophthalmic drugs. Clinical efficacy studies of new medicinal products use surrogate endpoints in addition to clinical endpoints. However, currently used surrogate endpoints are not always relevant and do not fully reflect changes in the status of patients with chronic or progressive diseases. The study analysed published approaches to the selection of endpoints in clinical studies of ophthalmic drugs intended for the treatment of glaucoma, uveitis, dry eye syndrome, and age-related macular degeneration. It was demonstrated that the choice of surrogate endpoints in a clinical trial should take into account specific aspects of a particular disease. The assessment of dynamic patterns of changes in visual functions generally requires a complex approach for a comprehensive characterisation of the eye condition in a particular disease. The paper analyses the possibility of using potential surrogate endpoints in studies of the most common eye diseases, and highlights that none of them has been recommended for use in clinical trials or routine clinical practice.