Patient Co-design: development of a novel disease-specific PROM

Kate Hayes, Elise Lammertyn, Hilde de Keyser, Trudy Havermans, Audrey Chansard, Rosa Coucke, Jutta Bend, Isabelle Sermet-Gaudelus

Research output: Contribution to conferencePosterpeer-review


Aims: The Patient Advisory Group of the European Cystic Fibrosis Society-Clinical Trial Network (ECFS-CTN) and Cystic Fibrosis Europe (CFE), the federation of national CF patient associations in Europe, are conducting a patient-led project which will enhance patient-clinician informed care in CF. The objective is qualitative development and cognitive evaluation of a new patient-led patient reported outcome measure (PROM) to assess health-related quality of life and symptom report in CF. Utilising research co-design processes such as the ‘INVVOLVE’ guidelines (invest in co-design; needs assessment; vision roles, responsibilities and rewards; validate participants; organise interaction carefully; lead the engagement; value patient time and input and evaluate and report) allows a structured approach to the meaningful involvement of all research users. Methods: A previous qualitative study (n=125) of people with CF (pwCF) and their caregivers identified key themes and 10 structured domains for inclusion in the development of a new CF-specific PROM questionnaire, the Patient-Reported Outcomes in CF (PRO-CF). The results of this study were combined with the expertise of a pan-European expert-patient group who met monthly during 2019-2020 by teleconference to further identify and refine key themes for inclusion in a new CF-specific PROM. A 2nd round review of the tool for content and face validity was conducted (n=15) in 2021, with additional expert patients reviewing and refining the tool to create the current 10 domain, 68-item questionnaire. Results: Initial review of the tool has confirmed that introductory instructions require clarification, and response scales are relevant and can be clearly interpreted by pwCF. Additional items were also identified for inclusion. The tool is currently being refined and next steps will address validation and translation including additional psychometric evaluation in patients with CF including those awaiting transplant. Conclusion: There is a lack of sensitive, standardised patient reported outcome measures in the current context of new disease modifying therapies in CF. This collaborative project by the ECFS-CTN & CFE is unique, engaging people with CF to create a meaningful and sensitive disease-specific PROM, to validate a new questionnaire for use in CF clinical trials and enhance patient-clinician informed care in CF.
Original languageEnglish
Publication statusAccepted - Sep 2021
EventInternational Society for Quality of Life Research Annual Conference - Virtual (due to pandemic)
Duration: 13 Oct 202116 Oct 2021


ConferenceInternational Society for Quality of Life Research Annual Conference
Internet address


  • Patients
  • Co-Design
  • questionnaire
  • cystic fibrosis

ASJC Scopus subject areas

  • Health Professions(all)


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