(302) Collaborative research to define patient‑reported experience measures of the cystic fibrosis care pathway in France: the ExPaParM study

Background: In France, the CF care pathway is coordinated by multidisciplinary teams from specialized CF centers or transplant centers. It includes care provided at home or out of hospital, risk prevention in daily life and adjustments to social life, which together contribute to the person’s quality of life. Patient experience is used to describe and evaluate care and life with the disease. Our research aims to identify the areas and criteria that characterize care and life from the patients’ perspective and to develop a questionnaire to collect patients’ and parents’ experience

Methods: The research group includes 3 academic researchers, 4 health care professionals and 5 patients as well as 2 parents of children with CF. This qualitative study is carried out in 4 phases: (1) recruiting patients and parents co-researchers, training them in qualitative research methods, defining the situations and profiles of patients in the study population, elaborating the detailed protocol [1]; (2) selecting the study sites, recruiting participants, carrying out semi-structured interviews, analyzing verbatims using the grounded theory approach and coding them in NVivo®; (3) co-elaborating Patient-Reported Experience questionnaires; (4) validating the construct with respondents and professionals from the study centers to assess the validity of the instrument.

Results: The protocol obtained a favourable opinion from the INSERM Ethics Committee (IRB00003888—no. 20-700). A 35-hour training was provided to the patients and parents co-researchers to enable them to participate effectively to the study. Eleven CF centers participated in the recruitment of 66 participants in mainland France and Reunion Island. Eighty hours of interviews were conducted. Three electronic questionnaires were developed (Lime Survey®) respectively for parents, non-transplanted adults and transplanted adults. Their testing with the participants (complete response rate = 67%) assessed their intelligibility, relevance and utility to describe the CF care and life pathway, a balanced distribution between closed and open questions. Time for answering was managed through thematic organization of the questions into several parts sent at different times and thanks to the ability to save answers and complete them later

Conclusions: The involvement of patients and parents in the research allowed to deepen the understanding of the various situations of care and life with the disease and to translate them into inclusion criteria. The training allowed them to conduct interviews, analyze verbatims in tandem with a researcher, test the questionnaires and contribute to the results and various communications. The questionnaires may be used by the CF centers to improve quality of care, by the patient association to identify avenues for advocacy or support to patients and families, and for further research. As a premice, the questionnaires appear to report the changes in the care pathways for the patients who received elexacaftor/tezacaftor/ivacaftor, suggesting their use to identify new care pathways.

Acknowledgements: This work was supported by the French Institute of Research in Public Health (IReSP) in the frame of the call for project “AAP General 2018”

References: [1] Pougheon Bertrand D et al. Collaborative research protocol to define patient‑reported experience measures of the cystic fibrosis care pathway in France: the ExPaParM study. Orphanet Journal of Rare Diseases. 2022;17:73-87. https://doi.org/10.1186/s13023-022-02204-0