Implications of “not me” drugs for health systems: lessons from age related macular degeneration

BMJ 2012; 344 doi: http://dx.doi.org/10.1136/bmj.e2941 (Published 1 May 2012)
Cite this as: BMJ 2012;344:e2941

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  1. Robert J Campbell, assistant professor1,
  2. Irfan A Dhalla, assistant professor2,
  3. Sudeep S Gill, associate professor3,
  4. Chaim M Bell, associate professor2
  1. 1Department of Ophthalmology, Hotel Dieu Hospital and Queen’s University, 166 Brock Street, Kingston, Ontario, Canada K7L 5G2
  2. 2Department of Medicine, St Michael’s Hospital and the University of Toronto, Toronto, Ontario, Canada
  3. 3Division of Geriatric Medicine, St Mary’s of the Lake Hospital and Queen’s University, Kingston
  1. Correspondence to: R J Campbell rob.campbell{at}queensu.ca
  • Accepted 16 February 2012

The debate surrounding the use of new drugs for age related macular degeneration has sparked worldwide controversy. Robert Campbell and colleagues highlight how differing funding systems have affected use of these drugs in different countries and suggest how to deal with similar challenges in future

The journey of a new drug from laboratory to market is long and expensive, and particularly treacherous for a drug with a novel mechanism of action. Only a small proportion of truly innovative new drugs find their way into our therapeutic armamentarium, and the success of these innovative drugs often inspires the proliferation of “me-too” drugs—products developed to target a pathway of known viability.1 w1

Recently, however, two drugs sharing a novel mechanism, ranibizumab (Lucentis) and bevacizumab (Avastin), have been licensed for different indications, and the manufacturer is trying to keep their uses distinct. Although both drugs have been shown to be effective for the treatment of age related macular degeneration (AMD), a leading cause of blindness in high income countries, only ranibizumab has been approved by the US Food and Drug Administration for this indication.2 Both drugs were developed by Genentech, which was acquired by Roche in 2009, with Novartis holding the licence to sell ranibizumab outside of the US. However, ranibizumab is much more expensive than bevacizumab when used to treat AMD. The companies’ active discouragement of the use of bevacizumab for macular degeneration—creating a “not me” drug—has focused attention on important aspects of drug policy. In particular, how differing health financing systems have contributed to the wide variation in uptake of ranibizumab and bevacizumab for AMD among high income countries.

Pharmacology of new drugs

Most cases of severe vision loss in AMD are caused by the proliferation of abnormal blood vessels beneath and within the retina.2 w2 Both ranibizumab and bevacizumab are monoclonal …

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