OBJECTIVES: To summarise the methods currently used to evaluate the cost-effectiveness of anti-vascular endothelial growth factor (VEGF) agents used in the treatment of retinal diseases.

METHODS: A systematic literature review (SLR) of cost-effectiveness studies and a targeted review of health technology assessment (HTA) submissions for anti-VEGF agents used in the treatment of wet age-related macular degeneration (wAMD), diabetic macular edema (DME), and macular edema secondary to branch or central retinal vein occlusion (ME/BRVO and ME/CRVO) that were published between 2011 and 2017 were undertaken. The HTA bodies included were the CADTH, NICE, PBAC, and SMC.

RESULTS: The SLR identified 30 publications describing cost-effectiveness of anti-VEGF agents; 18 in wAMD, 10 in DME, and 2 in ME/BRVO and ME/CRVO. Among the 22 publications in which the model structure was described, 19 reported a Markov structure whilst 3 publications, all in wAMD, described a patient-level simulation (PLS) approach. Twenty-nine of 30 publications reported a cost-utility analysis (CUA); 1 publication presented a cost-effectiveness analysis. In addition to the presentation of quality-adjusted life years (QALYs), 2 of the CUAs presented months or years of blindness avoided. In the target HTA review, 23 submissions meeting the inclusion criteria were identified; these models adopted a Markov approach, comprising health states defined by best-corrected visual acuity (BCVA) and death.

CONCLUSIONS: Severe visual impairment has been shown to be a determinant of reduced health-related quality of life (HRQoL) and excess mortality. Economic models in retinal conditions model the change in BCVA of patients and the relative improvement of BCVA achieved through different treatments to determine their cost utility/cost-effectiveness. Markov models are the preferred modeling approach although recent analyses are using PLS. Within the potential challenges identified, modelling the impact of HRQoL and excess mortality associated with bilateral vision seems to be the most difficult to manage.