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Hot off the breath: ‘I've a cost for’—the 64 million dollar question
  1. Andrew Bush1,
  2. Nicholas J Simmonds2
  1. 1Department of Paediatric Respiratory Medicine, Imperial College and Royal Brompton Harefield NHS Foundation Trust, Sydney Street, London, UK
  2. 2Department of Cystic Fibrosis, Imperial College and Royal Brompton Harefield NHS Foundation Trust, Sydney Street, London, UK
  1. Correspondence to Professor Andrew Bush, Department of Paediatric Respiratory Medicine, Imperial College and Royal Brompton Harefield NHS Foundation Trust, Sydney Street, London SW3 6NP, UK; a.bush{at}imperial.ac.uk

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On 12 January 2012, the US Food and Drug Administration (FDA) licensed Ivacaftor for use in patients with cystic fibrosis (CF) aged 6 years and over, who carry at least one copy of the class 111 mutation G551D. The cost per patient year in the USA will be a staggering US$294 000. Given that patients with G551D account for around 5% of the total CF population, and assuming that the price will be similar in the UK, if these patients are to receive this medication, there will be a hole in someone's budget to the extent of £60 million, because the one absolute certainty is that the government will not be making any more money available to cover the costs of this medication. To give context—the total national budget for CF care is of the order of £110 million. This is certainly a ‘wow-factor’ price; is it a wow-factor medication? What are the ethics of having a 50% hike in the CF drug budget driven by 5% of the population? And where do we go from here?

The history of CF treatment has been by any standards a major success story. Median survival has risen from less than a year in 1938 to a predicted value for current newborns of around 50 years.1 This has arisen from advances in the multidisciplinary treatment of the condition, and latterly with earlier diagnosis through newborn screening. Although standard treatment is increasingly successful, it leads to considerable burdens on the patients and their families, and largely comprises firefighting, namely treating the downstream consequences of the CF transmembrane regulator (CFTR) gene dysfunction, such as airway infection. However, Ivacaftor represents a stupendous paradigm shift, …

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Footnotes

  • Competing interests None.

  • Provenance and peer review Commissioned; internally peer reviewed.