Trading off risks and Benefits for Cystic Fibrosis treatments

  • Research type

    Research Study

  • Full title

    Trading off risks and benefits for cystic fibrosis treatments in an era of stratified medicine.

  • IRAS ID

    163770

  • Contact name

    Frank Kee

  • Contact email

    f.kee@qub.ac.uk

  • Duration of Study in the UK

    1 years, 0 months, 1 days

  • Research summary

    Cystic fibrosis (CF) is a serious life threatening autosomal recessive disorder which is thought to affect some 80,000 individuals worldwide. Individuals with cystic fibrosis experience severe breathing difficulties and serious lung infections. Despite rigorous research there is no cure for cystic fibrosis. CF is primarily treated with antibiotics. Recently, however, a double blind randomized control trial has shown that cystic fibrosis patients who have a specific genetic mutation respond significantly better to a new treatment than those individuals who do not have the specific genetic profile, signalling the emergence of a stratified medicine approach for his condition.

    Thus the proposed study aims to investigate whether individuals with cystic fibrosis are willing to accept the risks of side effects for a treatment which is more tailored to their medical needs. We will investigate this by presenting them with a series of clinical vignettes (a vignette is simply a summary of key pieces of information on a patient with CF). In each vignette, participants will be provided with options for two possible treatments (treatment A & treatment B). Each treatment will differ in the potential severity of risks and benefits and participants will simply be asked to choose, in a thought experiment, which of these two treatments they themselves would prefer, if these were the only two treatments available to them.

  • REC name

    HSC REC B

  • REC reference

    15/NI/0053

  • Date of REC Opinion

    19 Mar 2015

  • REC opinion

    Favourable Opinion