Personalised medicine in cystic fibrosis is unaffordable

Paediatr Respir Rev. 2014 Jun;15 Suppl 1:2-5. doi: 10.1016/j.prrv.2014.04.003. Epub 2014 Apr 13.


Personalised medicine refers to a tailored approach to treatment of an individual based on molecular analysis of genes, proteins or metabolites, and commonly involves a companion diagnostic test. It usually applies to small subsets of patients, often with rare diseases. In cystic fibrosis (CF), the best example is the CFTR (CF transmembrane conductance regulator) potentiator, ivacaftor, relevant to the 5% of cystic fibrosis patients with the p.Gly551Asp gene mutation. However the cost of personalised medicine is too high, making it unaffordable in the long term for many healthcare systems. Society needs to find a way to make personalised medicine affordable in order to not deny life-changing treatments from patients.

Keywords: Cystic fibrosis; Ivacaftor; Personalised medicine.

Publication types

  • Review

MeSH terms

  • Adult
  • Child
  • Costs and Cost Analysis
  • Cystic Fibrosis / economics*
  • Cystic Fibrosis / therapy*
  • Humans
  • Precision Medicine / economics*