Patient Reported Outcomes in Cystic Fibrosis

  • Kathryn Hill (Participant)

Impact: Health Impact, Quality of Life Impact

Description of impact

In 2017, ECFS-CTN started a project to develop patient reported outcome measures (PROMs), in collaboration with patient organisations. PROMs cover issues of concern to patients and the data can often be collected by self-administered questionnaires. These are of particular importance as they supply important patient-reported information about daily life which cannot be captured by usual measures. This can include quality of life measures such as patients’ resilience, coping strategies, and perception of their future and disease. In clinical trials they can provide a reliable way to gain the patients’ perspective regarding the benefits and limitations of a specific treatment. A survey of all CTN sites was conducted in 2019 to assess what quality of life/PROM/symptom report tools are used in current clinical practice. 120 interviews were also conducted with people with CF and their caregivers. The results from both of these projects have been summarised and are currently getting ready for publication: entitled: We have to keep asking questions: Patient Reported Outcome Measures in Cystic Fibrosis: A Qualitative Study of People with Cystic Fibrosis and their Caregivers. In 2019, ECFS-CTN continued the work (started in 2018) with our patient organisation partners through CF Europe to explore patient priorities and to look more deeply into the issues identified by patients. To do this, we formed a Patient Advisory Group, comprising people with CF, family members and representatives of both ECFS-CTN and CF Europe. During monthly conference calls, existing quality of life questionnaires were assessed and proposals for improvement were discussed. This hands-on approach has led to recommendations on how to assess patient reported outcomes, first in clinical trials and eventually also during routine clinic visits. A draft CF-specific questionnaire has now been created and the next steps involve wider scale testing.

Who is affected

People with cystic fibrosis, their carers, patient organisations and clinical trial networks


A CF specific questionnaire has been developed and is currently (2021) in the process of validation within 2 clinical trials (France and Belgium).
Impact statusOngoing
Impact date20202021
Category of impactHealth Impact, Quality of Life Impact
Impact levelEngagement