The off-label use of medication: the latest on the Avastin - Lucentis debacle

Med Law. 2013 Mar;32(1):65-77.

Abstract

The Avastin - Lucentis debacle illustrates the ethical, policy and legal dilemmas encountered with the off-label use of medication. Ophthalmologists are using intra-ocular injections of Avastin (off-label) to treat age-related macular degeneration (AMD). The off-label use of Avastin is controversial because there are anti-VEGF drugs on the market, authorized for AMD, such as Lucentis. Lucentis is however extremely expensive and costs approximately 50 times more than Avastin. Ophthalmologists did not have the backing of randomized controlled trials, or the blessing of the manufacturer of Avastin. In May 2011 the results from the first year of the Comparison of Age-Related Macular Degeneration Treatments Trials (CATT), a large, randomized clinical trial comparing Lucentis and Avastin, were released. The two-year results were published in April 2012. Respecting efficacy, the results of the drugs were the same. The study was not statistically powerful enough to identify meaningful differences in systemic drug-related adverse events and long-term safety. Ongoing trials in other parts of the world might or might not bring clarity in future. Currently there is conflict on the Avastin-issue between cost-conscious health authorities in EU Member States and the EU drug regulators. There are examples of cost-cutting solutions by health authorities, which risk undermining the fundamental principles of the regulatory framework. In the meantime risk is being shouldered by patients and doctors.

MeSH terms

  • Angiogenesis Inhibitors / therapeutic use
  • Antibodies, Monoclonal, Humanized / therapeutic use
  • Bevacizumab
  • Clinical Trials as Topic
  • European Union
  • Humans
  • Macular Degeneration / drug therapy
  • Off-Label Use / ethics*
  • Off-Label Use / legislation & jurisprudence*
  • Ranibizumab

Substances

  • Angiogenesis Inhibitors
  • Antibodies, Monoclonal, Humanized
  • Bevacizumab
  • Ranibizumab