Off-label prescribing in ophthalmology: a retrospective analysis

Off-label practice is often needed in ophthalmology, due to the limited number of drugs approved to treat several ocular diseases and to manage challenging ocular surgeries. The aim was to analyze the off-label drug prescriptions issued from January 2014 to December 2024 at the Department of Ophthalmology of the University Hospital of Catania (Italy). 233 off-label prescriptions, comprising 13 drugs, were delivered to treat 225 patients affected by ocular disorders. Results of the present study showed a raise of off-label prescriptions in 2015 and 2016 mainly due to mitomycin C use in trabeculectomy, representing 34.3% of the total off-label drug prescriptions in the reference period. After a decrease in 2017-2018, there was an increasing trend in the number of off-label issued prescriptions until 2024. The main contributors were anti-vascular endothelial growth factor (VEGF) drugs such as bevacizumab, ranibizumab and aflibercept, which represent 25.3%, 9.0% and 4.3% of off-label prescriptions, respectively. Additionally, 5-fluorouracil and dexamethasone were prescribed off-label, each representing 7.3% of the total off-label prescriptions, to mainly handle conjunctival carcinoma and various macular edema, respectively. Alteplase (6.9%), voriconazole (2.1%), verteporfin (1.3%), and foscarnet (0.9%), together with amphotericin B (0.4%), ganciclovir (0.4%), as well as rituximab (0.4%) were off-label prescribed to manage other specific ocular conditions. These results suggest that off-label practice is evolving in the field of ophthalmology. Collaboration among regulatory authorities, clinicians, marketing authorization holders, and patients is essential, in order to develop standardized, evidence-based international guidelines about off-label use, to improve outcome of patients with unmet medical needs.