Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2020 Jan 1;138(1):40-47.
doi: 10.1001/jamaophthalmol.2019.4557.

Economic Value of Anti-Vascular Endothelial Growth Factor Treatment for Patients With Wet Age-Related Macular Degeneration in the United States

Karen Mulligan  1   2 , Seth A Seabury  3   2 , Pravin U Dugel  4   5 , Jill F Blim  6 , Dana P Goldman  1   3   2 , Mark S Humayun  5   7
Affiliations

Economic Value of Anti-Vascular Endothelial Growth Factor Treatment for Patients With Wet Age-Related Macular Degeneration in the United States

Karen Mulligan et al. JAMA Ophthalmol. .

Erratum in

  • Error in the Author Affiliation.
    [No authors listed] [No authors listed] JAMA Ophthalmol. 2020 Feb 1;138(2):223. doi: 10.1001/jamaophthalmol.2019.5606. JAMA Ophthalmol. 2020. PMID: 31855249 Free PMC article. No abstract available.

Abstract

Importance: Anti-vascular endothelial growth factor (anti-VEGF) is a breakthrough treatment for wet age-related macular degeneration (wAMD), the most common cause of blindness in western countries. Anti-VEGF treatment prevents vision loss and has been shown to produce vision gains lasting as long as 5 years. Although this treatment is costly, the benefits associated with vision gains are large.

Objective: To estimate the economic value of benefits, costs for patients with wAMD, and societal value in the United States generated from vision improvement associated with anti-VEGF treatment.

Design, setting, and participants: This economic evaluation study used data from the published literature to simulate vision outcomes for a cohort of 168 820 patients with wAMD aged 65 years or older and to translate them into economic variables. Data were collected and analyzed from March 2018 to November 2018.

Main outcomes and measures: Main outcomes included patient benefits, costs, and societal value. Each outcome was estimated for a newly diagnosed cohort and the full population across 5 years, with a focus on year 3 as the primary outcome because data beyond that point may be less representative of the general population. Drug costs were the weighted mean across anti-VEGF therapies. Two current treatment scenarios were considered: less frequent injections (mean [SD], 8.2 [1.6] injections annually) and more frequent injections (mean [range], 10.5 [6.8-13.1] injections annually). The 2 treatment innovation scenarios, improved adherence and best case, had the same vision outcomes as the current treatment scenarios had but included more patients treated from higher initiation and lower discontinuation.

Results: The study population included 168 820 patients aged 65 years at the time of diagnosis with wAMD. The underlying clinical trials that were used to parameterize the model did not stratify visual acuity outcomes or treatment frequency by sex; therefore, the model parameters could not be stratified by sex. The current treatment scenario of less frequent injections generated $1.1 billion for the full population in year 1 and $5.1 billion in year 3, whereas the scenario of more frequent injections generated $1.6 billion (year 1) and $8.2 billion (year 3). Three-year benefits ranged from $7.3 billion to $11.4 billion in the improved adherence scenario and from $9.7 billion to $15.0 billion if 100% of the patients initiated anti-VEGF treatment and the discontinuation rates were 6% per year or equivalent to clinical trial discontinuation (best-case scenario). Societal value (patient benefits net of treatment cost) ranged from $0.9 billion to $3.0 billion across 3 years in the current treatment scenarios and from $0.9 billion to $4.3 billion in the treatment innovation scenarios.

Conclusions and relevance: This study's findings suggest that improved vision associated with anti-VEGF treatment may provide economic value to patients and society if the outcomes match published outcomes data used in these analyses; however, future innovations that increase treatment utilization may result in added economic benefit.

PubMed Disclaimer

Conflict of interest statement

Conflict of Interest Disclosures: Drs Mulligan and Seabury reported receiving unrestricted gifts from the American Society of Retina Specialists (ASRS) during the conduct of the study and receiving personal fees from Precision Health Economics outside the submitted work. Dr Dugel reported receiving personal fees from Alcon, Novartis, Genentech, Roche, Bausch, Allergan, Regenxbio, Oxurion, Clearside Biomedical, Aerpio, Opthea, Spark, Graybug Vision, Zeiss Group, Beyeonics, PanOptica, Chengdu Kanghong Biotech, SciFluor, Boehringer Ingelheim, Kodiak, Oculis SA, Eyepoint, Aerie, Pieris, Gemini, Ionis, Reneuron, Merck, Daiichi Sankyo, AsclepiX, Fox Kiser, Arctic Vision, Nan Fung Group, and Allegro outside the submitted work; and serving on scientific advisory boards for Allergan, Genentech, Roche, Regeneron, Novartis, and Oxurion. Dr Goldman reported receiving an unrestricted gift from the ASRS and other funds from the University of Southern California (Leonard D. Schaeffer Center for Health Policy & Economics) during the conduct of the study; receiving personal fees and consulting fees from Precision Health Economics LLC outside the submitted work; owning (<1%) in the parent company, Precision for Medicine; receiving speaker fees or honoraria unrelated to this work from Allergan, Amgen, Avanir, Cedars-Sinai Medical Center, Celgene, Claremont College, Columbia University, FAIR Health, LA Biomed, Novartis, Novo Nordisk, Stanford University, University of Arizona, University of California, University of Chicago, and Western University College of Pharmacy; serving as a paid scientific advisor to the Aspen Institute, Congressional Budget Office, Fred Hutchinson Cancer Institute, and ACADIA Pharmaceuticals; and serving as the Director of the Leonard D. Schaeffer Center for Health Policy & Economics, which is supported by grants and gifts from individuals, corporations, and associations; by government grants and contracts; and by private foundations (see https://healthpolicy.usc.edu for specific information related to funding sources). Dr Humayun reported receiving consulting fees and honorarium from Allergan outside the submitted work; receiving consulting fees from and holding equity in MTTR outside the submitted work; receiving consulting fees from Outlook Therapeutics outside the submitted work; receiving consulting fees, patents, and royalties, and being an equity owner for Replenish outside the submitted work; and serving as the co-director of the USC Roski Eye Institute, which receives restricted support from Research to Prevent Blindness.

Figures

Figure 1.
Figure 1.. Patient Benefits, Costs, and Societal Value Associated With Anti–Vascular Endothelial Growth Factor (VEGF) Treatment Compared With No Treatment
The figure shows the benefits and costs for current treatment scenarios for the full population. Societal value is calculated as patient benefits net of treatment costs. Population benefits and costs assume that new incident cohorts enter the model each year. Future values are discounted at a rate of 3%.
Figure 2.
Figure 2.. Value of Treatment Innovation Compared With No Treatment
The current treatment scenarios, less and more frequent injections, assume that 65% of the patients initiate anti–vascular endothelial growth factor (VEGF) treatment and 50% discontinue it each year. Improved adherence assumes 80% uptake and 17% discontinuation in year 1 (50% by year 5). The best-case scenario assumes 100% uptake and 6% discontinuation annually. Future values are discounted at 3%.
See this image and copyright information in PMC

Comment in

References

    1. Prevent blindness Living with AMD. https://www.preventblindness.org/living-amd. Accessed December 17, 2018.
    1. Wittenborn J, Rein D Cost of Vision Problems: The Economic Burden of Vision Loss and Eye Disorders in the United States. https://www.google.com/url?sa=t&rct=j&q=&esrc=s&source=web&cd=3&cad=rja&.... Published 2013. Accessed October 10, 2019.
    1. Duffy M. New research: two potential treatments for dry macular degeneration and geographic atrophy. VisionAware website. https://www.visionaware.org/blog/visionaware-blog/new-research-two-poten.... Published October 2017. Accessed February 1, 2019.
    1. National Eye Institute Age-related macular degeneration (AMD) data and statistics. https://nei.nih.gov/learn-about-eye-health/resources-for-health-educator.... Accessed April 13, 2018.
    1. Peden MC, Suñer IJ, Hammer ME, Grizzard WS. Long-term outcomes in eyes receiving fixed-interval dosing of anti-vascular endothelial growth factor agents for wet age-related macular degeneration. Ophthalmology. 2015;122(4):803-808. doi:10.1016/j.ophtha.2014.11.018 - DOI - PubMed

Publication types

MeSH terms

Substances