The last decade has witnessed developments in the CF drug pipeline which are both exciting and unprecedented, bringing with them previously unconsidered challenges. The Task Force group was brought together to consider these challenges and possible strategies to address them. Over the last 18 months, we have discussed internally and gathered views from a broad range of individuals representing patient organisations, clinical and research teams, the pharmaceutical industry and regulatory agencies. In this and the accompanying article, we discuss two main areas of focus: i) optimising trial design and delivery for speed and efficiency; ii) drug development for patients with rare CFTR mutations. We propose some strategies to tackle the challenges ahead and highlight areas where further thought is needed. We see this as the start of a process rather than the end and hope herewith to engage the wider community in seeking solutions to improved treatments for all patients with CF.

Hadassah Hebrew University Medical Center Jerusalem Israel

Leeds Teaching Hospitals NHS Trust Leeds UK

Motol University Hospital Prague Czech Republic

National Heart and Lung Institute Imperial College London UK; Royal Brompton and Harefield NHS Foundation Trust London UK

Queens University Belfast UK

University of Leuven Belgium

University of Lisboa Portugal

References provided by Crossref.org

Organoids as a personalized medicine tool for ultra-rare mutations in cystic fibrosis: The case of S955P and 1717-2A>G

Building global development strategies for cf therapeutics during a transitional cftr modulator era