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Cost-Effectiveness of Treatment of Diabetic Macular Edema

Suzann Pershing, MD, MS; Eva A. Enns, MS, PhD; Brian Matesic, BS; Douglas K. Owens, MD, MS; and Jeremy D. Goldhaber-Fiebert, PhD
[+] Article, Author, and Disclosure Information

From Stanford Health Policy, Center for Health Policy and Center for Primary Care and Outcomes Research, Stanford University, Stanford, California; Byers Eye Institute at Stanford University, Stanford University School of Medicine, and Veterans Affairs Palo Alto Health Care System, Palo Alto, California; and University of Minnesota School of Public Health, Minneapolis, Minnesota.

This material was presented at the 34th Annual Meeting of the Society of Medical Decision Making, Phoenix, Arizona, 17–19 October 2012.

Disclaimer: The contents of this article are solely the responsibility of the authors and do not necessarily represent the views of the National Institutes of Health, Agency for Healthcare Research and Quality, or U.S. Department of Veterans Affairs. Discussion of DRCR.net data, as cited in this publication, is by the authors and is in no way affiliated or endorsed by DRCR.net. Additional data provided by DRCR.net is not an indication that DRCR.net has made any statement on the validity of these analyses or interpretations.

Acknowledgment: The authors thank Dr. Mark S. Blumenkranz for guidance and manuscript review.

Financial Support: By grant T32-HS000028 from the Agency for Healthcare Research and Quality and a National Institutes of Health National Institute on Aging Career Development Award (K01 AG037593-01A1; Dr. Goldhaber-Fiebert, principal investigator). Dr. Owens was supported by the U.S. Department of Veterans Affairs. This research was also supported in part by the Office of the Dean, Stanford Medical School, Stanford Society of Physician Scholars.

Potential Conflicts of Interest: Disclosures can be viewed at www.acponline.org/authors/icmje/ConflictOfInterestForms.do?msNum=M13-0768.

Reproducible Research Statement: Study protocol, statistical code, and data set: Available from Dr. Pershing (e-mail, pershing@stanford.edu).

Requests for Single Reprints: Suzann Pershing, MD, MS, 2452 Watson Court, Palo Alto, CA 94303; e-mail, pershing@stanford.edu.

Current Author Addresses: Dr. Pershing: 2452 Watson Court, Palo Alto, CA 94303.

Dr. Enns: University of Minnesota, Division of Health Policy and Management, MMC 729 Mayo, Campus Mail Code 8729A, 420 Delaware Street SE, Minneapolis, MN 55455.

Mr. Matesic: 316 Grant Avenue, Palo Alto, CA 94306.

Dr. Owens and Goldhaber-Fiebert: Stanford University, Center for Health Policy and Center for Primary Care and Outcomes Research, 117 Encina Commons, Stanford, CA 94305.

Author Contributions: Conception and design: S. Pershing, J.D. Goldhaber-Fiebert, D.K. Owens.

Analysis and interpretation of the data: S. Pershing, E.A. Enns, J.D. Goldhaber-Fiebert.

Drafting of the article: S. Pershing,

Critical revision of the article for important intellectual content: S. Pershing, E.A. Enns, J.D. Goldhaber-Fiebert, D.K. Owens.

Final approval of the article: S. Pershing, E.A. Enns, J.D. Goldhaber-Fiebert, D.K. Owens, B. Matesic.

Provision of study materials or patients:

Statistical expertise: S. Pershing, E.A. Enns, J.D. Goldhaber-Fiebert.

Obtaining of funding:

Administrative, technical, or logistic support:

Collection and assembly of data: S. Pershing.

Ann Intern Med. 2014;160(1):18-29. doi:10.7326/M13-0768
Text Size: A A A

Background: Macular edema is the most common cause of vision loss among patients with diabetes.

Objective: To determine the cost-effectiveness of different treatments of diabetic macular edema (DME).

Design: Markov model.

Data Sources: Published literature and expert opinion.

Target Population: Patients with clinically significant DME.

Time Horizon: Lifetime.

Perspective: Societal.

Intervention: Laser treatment, intraocular injections of triamcinolone or a vascular endothelial growth factor (VEGF) inhibitor, or a combination of both.

Outcome Measures: Discounted costs, gains in quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs).

Results of Base-Case Analysis: All treatments except laser monotherapy substantially reduced costs, and all treatments except triamcinolone monotherapy increased QALYs. Laser treatment plus a VEGF inhibitor achieved the greatest benefit, gaining 0.56 QALYs at a cost of $6975 for an ICER of $12 410 per QALY compared with laser treatment plus triamcinolone. Monotherapy with a VEGF inhibitor achieved similar outcomes to combination therapy with laser treatment plus a VEGF inhibitor. Laser monotherapy and triamcinolone monotherapy were less effective and more costly than combination therapy.

Results of Sensitivity Analysis: VEGF inhibitor monotherapy was sometimes preferred over laser treatment plus a VEGF inhibitor, depending on the reduction in quality of life with loss of visual acuity. When the VEGF inhibitor bevacizumab was as effective as ranibizumab, it was preferable because of its lower cost.

Limitation: Long-term outcome data for treated and untreated diseases are limited.

Conclusion: The most effective treatment of DME is VEGF inhibitor injections with or without laser treatment. This therapy compares favorably with cost-effective interventions for other conditions.

Primary Funding Source: Agency for Healthcare Research and Quality.


Grahic Jump Location
Figure 1.

Markov model schematic.

The 6 alternatives to the right of the decision node (square box) represent the 6 strategies for comparison, each progressing within the Markov model. The shaded boxes represent the Markov model transitions for progression of diabetic macular edema. Visual acuity categories 1 through 6 represent states of visual acuity (Table 1). Solid arrows represent possible worsening (progression) within a given month, and dashed arrows represent the potential for improvement or progression within a given month while receiving treatment. On-treatment states are subject to risk for complications (arterial thromboembolic events, glaucoma, cataracts, and other major or minor complications). In the base case, treatment was stopped after 1 y for all strategies or sooner if an arterial thromboembolic event or severe glaucoma occurred. VEGF = vascular endothelial growth factor.* Off or after treatment.

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Figure 2.

One-year treatment and lifetime treatment cost-effectiveness frontiers.

Discounted lifetime costs and QALYs associated with the 6 strategies. The top panel represents the main analysis, with 1 y of treatment with lifetime follow-up. The bottom panel represents lifetime treatment with 3 VEGF inhibitor injections per year (monotherapy or combination therapy), 1 triamcinolone injection per year (monotherapy or combination therapy), 1 laser treatment every other year in laser monotherapy, and no additional laser treatments in combination therapy strategies. The solid black line indicates the cost-effectiveness frontier, which represents the most cost-effective series of strategies (achieving the greatest relative benefit for the lowest cost). The ICERs are indicated in dollars per QALY, representing the cost of additional effectiveness relative to the next best strategy (this figure compares the preferred strategy of laser therapy plus VEGF inhibitors with laser therapy plus triamcinolone, with an ICER of $12 410/QALY with 1 y of treatment and an ICER of $26 477/QALY with treatment over a lifetime). The strategies that form the cost-effectiveness frontier (laser therapy plus triamcinolone and laser therapy plus VEGF inhibitors, each depicted with a solid black circle) dominate those to the right of the frontier (gray circles) because they are more cost-effective and cost less (strong dominance) or have a better cost-effectiveness ratio (weak dominance). ICER = incremental cost-effectiveness ratio; QALY = quality-adjusted life-year; VEGF = vascular endothelial growth factor.

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