Do current clinical trials in cystic fibrosis match the priorities of patients and clinicans? A systematic review

J Cyst Fibros. 2020 Jan;19(1):26-33. doi: 10.1016/j.jcf.2019.06.005. Epub 2019 Jun 22.


There are many uncertainties regarding Cystic Fibrosis (CF) treatment. Recently, the first James Lind Alliance (JLA) Priority Setting Partnership (PSP) in CF was completed, bringing clinicians, patients and carers together to identify the Top 10 research priorities. Here we investigate how well the current clinical trials landscape reflects these priorities. Trials in CF were identified through searches of research databases (Pubmed, ANZCTR, EU clinical trials register, and ISRCTN). Trials meeting inclusion criteria of registered intervention studies in CF published between 01.012016 and 11.09.2017 were matched to the Top 10 priorities. We identified 259 trials, with 193 fulfilling the inclusion criteria. Only 63 (33%) of these matched one or more of the JLA priorities showing that current clinical trials poorly reflect the JLA Top 10. By increasing awareness of the Top 10 priorities, it is hoped that this will fuel future research in areas important to the CF community.

Keywords: Clinical trial; Cystic fibrosis; Research priority.

Publication types

  • Research Support, Non-U.S. Gov't
  • Systematic Review

MeSH terms

  • Biomedical Research / methods*
  • Clinical Trials as Topic / standards*
  • Cystic Fibrosis* / genetics
  • Cystic Fibrosis* / therapy
  • Humans
  • Needs Assessment
  • Research*