Intravitreal Ranibizumab for the Treatment of Visual Impairment Due to Choroidal Neovascularization Associated with Rare Diseases: Cost-Effectiveness in the UK

  • Grant McCarthy
  • Elisabetta Fenu
  • Natalie BennettEmail author
  • Chrissy Almond
Original Research



This study sought to determine the cost-effectiveness of intravitreal ranibizumab compared with best supportive care (BSC; considered to be no active treatment) for the treatment of visual impairment due to choroidal neovascularization (CNV) associated with causes other than neovascular age-related macular degeneration (nAMD) and pathologic myopia (PM) in a UK setting.


An individual patient-level simulation model was developed to estimate the lifetime costs and quality-adjusted life years (QALYs) of ranibizumab vs. BSC. Regression analyses, performed on patient-level data collected within the pivotal phase III MINERVA trial, modelled visual acuity (VA) progression while patients remained on treatment. Patient utilities were modelled as a function of VA in both eyes and resource use estimates were based on trial data or the literature. Costs were evaluated from the perspective of the UK National Health Service and personal social services, with future costs and health outcomes discounted at 3.5% per annum. Sensitivity and scenario analyses were conducted.


The incremental cost-effectiveness ratio for intravitreal ranibizumab was £1363 per QALY compared to BSC and was associated with an incremental benefit of 1.06 QALYs and an incremental cost of £1444 per patient. Drug and administration costs of intravitreal ranibizumab were offset by the prevention of the development of blindness and its associated costs, while the increase in benefits was driven by a reduction in mortality risk and an improved health-related quality of life attributed to an improvement in VA. The findings were robust to a range of sensitivity analyses and ranibizumab consistently remained cost-effective at a willingness-to-pay threshold of £20,000–30,000 per QALY gained for all sensitivity analyses.


Intravitreal ranibizumab is a highly cost-effective intervention for the treatment of CNV due to causes other than nAMD and PM as it delivers substantial QALY gains to patients while making cost savings vs. BSC.


Novartis Pharmaceuticals UK Ltd.


Choroidal neovascularization Cost-effectiveness Economic evaluation Ophthalmology Ranibizumab VEGF 




This study was funded by Novartis Pharmaceuticals UK Ltd, Surrey, UK. Funding for the article processing charges was provided by Novartis Pharmaceuticals UK Ltd. All authors had full access to all of the data in this study and take complete responsibility for the integrity of the data and accuracy of the data analysis.

Editorial Assistance

Editorial assistance in the preparation of this manuscript was provided by Dr. Amanda Prowse, Lochside Medical Communications. Support for this assistance was funded by Novartis Pharmaceuticals UK Ltd, Surrey, UK.


All named authors meet the International Committee of Medical Journal Editors (ICMJE) criteria for authorship for this article, take responsibility for the integrity of the work as a whole, and have given their approval for this version to be published.


Grant McCarthy is an employee of BresMed Health Solutions which was contracted and paid by Novartis to develop the cost-effectiveness model. Elisabetta Fenu is an employee of BresMed Health Solutions which was contracted and paid by Novartis to develop the cost-effectiveness model. Natalie Bennett is an employee at Novartis Pharmaceuticals UK Ltd. Chrissy Almond is an employee of BresMed Health Solutions which was contracted and paid by Novartis to develop the cost-effectiveness model. The authors did not receive direct payment as a result of this work outside of their normal salary payments.

Compliance with Ethics Guidelines

This study is based on previously conducted studies and does not involve any new studies of human participants or animal subjects performed by any of the authors. This study (a cost-effectiveness analysis) did not require ethical approval as it did not involve human participants or animal subjects.

Data Availability

The datasets generated during and/or analyzed during the current study are available from the corresponding author on reasonable request.

Supplementary material

12325_2019_894_MOESM1_ESM.pdf (236 kb)
Supplementary material 1 (PDF 236 kb)


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Copyright information

© Springer Healthcare Ltd., part of Springer Nature 2019

Authors and Affiliations

  • Grant McCarthy
    • 1
  • Elisabetta Fenu
    • 1
  • Natalie Bennett
    • 2
    Email author
  • Chrissy Almond
    • 1
  1. 1.BresMed Health Solutions LtdSheffieldUK
  2. 2.Novartis Pharmaceuticals UK LimitedCamberleyUK

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