Rethinking CF organization of care in the era of highly effective modulator: a nationwide research program HORIZON

Abstract

Highly effective modulator therapy (HEMT) is now available to a wider range of people with cystic fibrosis (pwCF). It has significantly improved short-term clinical outcomes and has the potential to alter the natural history of this fatal genetic disease. If long-term follow-up observational data is required to ensure clinical benefits, it is obvious that it will also change the needs of pwCF and the roles and missions of healthcare professionals (HCPs) in CF centers and beyond. We will conduct a nationwide research program called 'HORIZON' to support the necessary changes in the organization of CF care in the coming years. Our primary objective is to design a new model of CF care organization that can respond to the new needs and missions of pwCF and HCPs in this era of rapid and profound changes due to HEMT. This research program is based on the intervention mapping method, in which we will conduct the first four steps to design and plan the implementation of a new organization of care. The program will involve all stakeholders of the CF care network, including HCPs from CF care centers and outside, pwCF and their families, patient organizations, and experts from other countries. It will combine quantitative and qualitative research approaches and rely on an 'action research' method. Anticipating and supporting the reorganization of CF care in France requires a robust research program to find the best model that meets the expectations of all key stakeholders.