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

Abstract

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.

Keywords:

• Anti-vascular endothelial growth factor
• aflibercept
• cost-effectiveness analysis
• diabetic macular edema
• health-related quality of life
• ranibizumab

JEL classification codes:

• I11
• I19

Transparency

Declaration of funding

Genentech, Inc., South San Francisco, CA, provided support for the study and participated in the design and conduct of the study, and data collection, management, and interpretation. Third-party writing assistance was provided by Amy Lindsay, PhD, of Envision Pharma Group and funded by Genentech, Inc.

Declaration of financial/other interests

NH is a consultant for Alimera, Allegro, Allergan, BioTime, Gemini, Genentech, Inc., Katalyst, Novartis, Regeneron, and Roche. SBD is a consultant for Genentech, Inc. YR and VG are employees of Genentech, Inc., and hold stock/stock options in Roche. The peer reviewers on this manuscript have received an honorarium from JME for their review work. In addition, a reviewer on this manuscript discloses their role as a consultant for Genentech, Inc., and Novartis, while another reviewer discloses their role as a consultant for Bayer and Novartis. The reviewers have no other relevant financial relationships or otherwise to disclose.

Author contributions

All authors were involved in the conception and design of the study, the analysis and interpretation of the data, and critical review and revision of the paper for intellectual content. All authors provided final approval of the version to be published and agree to be accountable for all aspects of the work.

Acknowledgements

Funding was provided by Genentech, Inc., a member of the Roche Group, for the study and third-party writing assistance, which was provided by Amy Lindsay, PhD, of Envision Pharma Group.

Previous presentations

Portions of these data were presented at the Association for Research in Vision and Ophthalmology Annual Meeting; May 7–11, 2017; Baltimore, MD.