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Original Investigation
October 24, 2019

Five-Year Cost-effectiveness of Intravitreous Ranibizumab Therapy vs Panretinal Photocoagulation for Treating Proliferative Diabetic Retinopathy: A Secondary Analysis of a Randomized Clinical Trial

Author Affiliations
  • 1Department of Health Management and Policy, University of Michigan School of Public Health, Ann Arbor
  • 2Department of Industrial and Operations Engineering, University of Michigan College of Engineering, Ann Arbor
  • 3Institute for Healthcare Policy & Innovation, University of Michigan, Ann Arbor
  • 4Department of Ophthalmology and Visual Sciences, University of Michigan Medical School, Ann Arbor
  • 5Jaeb Center for Health Research, Tampa, Florida
  • 6Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, Maryland
  • 7Editor, JAMA Ophthalmology
  • 8Feinberg School of Medicine, Northwestern University, Chicago, Illinois
  • 9Beetham Eye Institute, Joslin Diabetes Center, Boston, Massachusetts
  • 10Department of Ophthalmology, Harvard Medical School, Boston, Massachusetts
  • 11CME Editor, JAMA Ophthalmology
JAMA Ophthalmol. Published online October 24, 2019. doi:https://doi.org/10.1001/jamaophthalmol.2019.4284
Key Points

Question  What is the incremental cost-effectiveness ratio of therapy with ranibizumab, 0.5 mg, compared with panretinal photocoagulation at 5- and 10-year horizons for treating patients diagnosed with proliferative diabetic retinopathy?

Findings  This preplanned secondary analysis of a randomized clinical trial found that the estimated 10-year incremental cost-effectiveness ratio of the ranibizumab group compared with panretinal photocoagulation for those without center-involved diabetic macular edema at baseline is $742 202 per quality-adjusted life-year, and for those with baseline center-involved diabetic macular edema, $63 930 per quality-adjusted life-year.

Meaning  This study’s findings suggest that, at the 5- and 10-year horizons, therapy with ranibizumab, 0.5 mg, may be within the frequently cited range considered cost-effective in the United States for treating patients with proliferative diabetic retinopathy and with baseline center-involved diabetic macular edema and vision loss but not for those without.

Abstract

Importance  The DRCR Retina Network Protocol S randomized clinical trial suggested that the mean visual acuity of eyes with proliferative diabetic retinopathy (PDR) treated with ranibizumab is not worse at 5 years than that of eyes treated with panretinal photocoagulation (PRP). Moreover, the ranibizumab group had fewer new cases of diabetic macular edema (DME) with vision loss or vitrectomy but had 4 times the number of injections and 3 times the number of visits. Although 2-year cost-effectiveness results of Protocol S were previously identified, incorporating 5-year data from Protocol S could alter the longer-term cost-effectiveness of the treatment strategies from the perspective of the health care system.

Objective  To evaluate 5- and 10-year cost-effectiveness of therapy with ranibizumab, 0.5 mg, compared with PRP for treating PDR.

Design, Setting, and Participants  A preplanned secondary analysis of the Protocol S randomized clinical trial using efficacy, safety, and resource utilization data through 5 years of follow-up for 213 adults diagnosed with PDR and simulating results through 10 years.

Interventions  Intravitreous ranibizumab, 0.5 mg, at baseline and as frequently as every 4 weeks based on a structured retreatment protocol vs PRP at baseline for PDR; eyes in both groups could receive ranibizumab for concomitant DME with vision loss.

Main Outcomes and Measures  Incremental cost-effectiveness ratios (ICERs) of ranibizumab therapy compared with PRP were evaluated for those with and without center-involved DME (CI-DME) and vision loss (Snellen equivalent, 20/32 or worse) at baseline.

Results  The study included 213 adults with a mean (SD) age of 53 (12) years, of whom 92 (43%) were women and 155 (73%) were white. The ICER of the ranibizumab group compared with PRP for patients without CI-DME at baseline was $582 268 per quality-adjusted life-year (QALY) at 5 years and $742 202/QALY at 10 years. For patients with baseline CI-DME, ICERs were $65 576/QALY at 5 years and $63 930/QALY at 10 years.

Conclusions and Relevance  This study suggests that during 5 to 10 years of treatment, ranibizumab, 0.5 mg, as given in the studied trial compared with PRP may be within the frequently cited range considered cost-effective in the United States for eyes presenting with PDR and vision-impairing CI-DME, but not for those with PDR but without vision-impairing CI-DME. Substantial reductions in anti–vascular endothelial growth factor cost may make the ranibizumab therapy cost-effective within this range even for patients without baseline CI-DME.

Trial Registration  ClinicalTrials.gov identifier: NCT01489189

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