Cost-effectiveness of ranibizumab and aflibercept to treat diabetic macular edema from a U.S. perspective: analysis of 2-year Protocol T data.

Aims: Protocol T (NCT01627249) was a head-to-head study conducted by the Diabetic Retinopathy Clinical Research Network that compared intravitreal aflibercept, bevacizumab, and ranibizumab for the treatment of diabetic macular edema (DME). A cost-effectiveness analysis accompanying the 1-year data of Protocol T revealed that aflibercept was not cost-effective versus ranibizumab for all patients, but could have been cost-effective in certain patient subgroups if the 1-year results were extrapolated out to 10 years. The present study evaluated cost-effectiveness of U.S. Food and Drug Administration-approved anti-vascular endothelial growth factor agents (ranibizumab, aflibercept) for treatment of DME using the 2-year data from Protocol T. Methods: Costs of aflibercept 2.0 mg or ranibizumab 0.3 mg, visual acuity (VA)-related medical costs, and quality-adjusted life-years (QALYs) were simulated for eight VA health states. Treatment, adverse event management, and VA-related healthcare resource costs (2016 U.S. dollars) were based on Medicare reimbursement and published literature. VA-related health utilities were determined using a published algorithm. Patients were stratified by baseline VA: 20/40 or better; 20/50 or worse. Results: Total 2-year costs were higher, and QALYs similar, for aflibercept versus ranibizumab in the full cohort ($44,423 versus $34,529; 1.476 versus 1.466), 20/40 or better VA subgroup ($40,854 versus $31,897; 1.517 versus 1.519), and 20/50 or worse VA subgroup ($48,214 versus $37,246; 1.433 versus 1.412), respectively. Incremental cost-effectiveness ratios in the full cohort and 20/50 or worse VA subgroup were $986,159/QALY and $523,377/QALY, respectively. These decreased to $711,301 and $246,978 when analyses were extrapolated to 10 years. Limitations: Key potential limitations include the fact that VA was the only QALY parameter analysed and the uncertainty surrounding the role of better- and worse-seeing eye VA in overall functional impairment. Conclusions: This analysis suggests that aflibercept is not cost-effective versus ranibizumab for patients with DME, regardless of baseline vision.