OBJECTIVES : To characterize the techniques used to derive health-state utilities (HSU) in the cost-utility studies of ophthalmic drugs.

METHODS : A systematic review was performed in Pubmed/Embase until October 2019. Cost-utility studies evaluating ophthalmic drugs were included. The therapeutic area, the technique to derive HSU and the sources of HSU were extracted. It was analysed whether the HSU and the other parameters of the cost-utility studies were collected from the same or different populations. The techniques to derive HSU used in the cost-utility studies and the ones recommended by the country-specific economic evaluation guidelines were compared.

RESULTS : Seventy cost-utility studies were included. Most (n=39; 55.7%) assessed drugs indicated in age-related macular degeneration. Forty-three (61.4%) used direct techniques to derive HSU, 19 (27.1%) used indirect techniques, 1 (1.4%) used direct and indirect techniques and seven (10.0%) used other techniques. Twelve (17.1%) studies collected the HSU and the other study parameters from the same population: nine (12.9%) retrieved utility data from experimental studies, two (2.9%) from observational and one (1.4%) from other sources. Forty-eight (68.6%) studies collected the HSU and the other study parameters from different populations: eight (11.4%) retrieved utility data from experimental studies, 33 (47.1%) from observational, one (1.4%) from both experimental and observational and six (8.6%) from other sources. It was not possible to identify the population from whom data were obtained in 10 (14.3%) studies. Eleven (15.7%) studies followed the recommendations of guidelines regarding the techniques to derive HSU, 21 (30.0%) did not follow and for 38 (54.3%) studies, it was not possible to assess.

CONCLUSIONS : The techniques used to derive HSU significantly varied across the cost-utility studies of ophthalmic drugs. Guidance to conduct cost-utility studies in specific diseases, such as ophthalmology, should be developed and followed to enhance the role of the cost-utility study as a decision-making tool.