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

• Published in: Journal of medical economics Vol. 23; no. 3; pp. 287 - 296
• Main Authors: Holekamp, Nancy, Duff, Steven B., Rajput, Yamina, Garmo, Vincent
• Format: Journal Article
• Language: English
• Published: England Taylor & Francis 03.03.2020
• Subjects: Activities of Daily Living; aflibercept; Aged; Aged, 80 and over; Angiogenesis Inhibitors - economics; Angiogenesis Inhibitors - therapeutic use; Anti-vascular endothelial growth factor; Cost-Benefit Analysis; cost-effectiveness analysis; Diabetes Complications - drug therapy; diabetic macular edema; Female; Health Expenditures - statistics & numerical data; Health Resources - economics; Health Resources - statistics & numerical data; health-related quality of life; Humans; Macular Edema - drug therapy; Male; Medicare - statistics & numerical data; Patient Acceptance of Health Care - statistics & numerical data; Quality-Adjusted Life Years; ranibizumab; Ranibizumab - economics; Ranibizumab - therapeutic use; Receptors, Vascular Endothelial Growth Factor - antagonists & inhibitors; Receptors, Vascular Endothelial Growth Factor - therapeutic use; Recombinant Fusion Proteins - economics; Recombinant Fusion Proteins - therapeutic use; Severity of Illness Index; United States; Visual Acuity; United States; I19; Anti-vascular endothelial growth factor; aflibercept; I11; health-related quality of life; cost-effectiveness analysis; ranibizumab; diabetic macular edema
• ISSN: 1369-6998; 1941-837X
• DOI: 10.1080/13696998.2019.1666855

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 vs ranibizumab for all patients, but could have been cost-effective in certain patient sub-groups if the 1-year results were extrapolated out to 10 years. The present study evaluated the cost-effectiveness of US 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 US 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 vs ranibizumab in the full cohort ($44,423 vs $34,529; 1.476 vs 1.466), 20/40 or better VA sub-group ($40,854 vs $31,897; 1.517 vs 1.519), and 20/50 or worse VA sub-group ($48,214 vs $37,246; 1.433 vs 1.412), respectively. Incremental cost-effectiveness ratios in the full cohort and 20/50 or worse VA sub-group 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 analyzed 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 vs ranibizumab for patients with DME, regardless of baseline vision.