EUROPEAN CYSTIC FIBROSIS SOCIETY

Their core work

The European Cystic Fibrosis Society (ECFS) is a professional medical association that brings together clinicians, researchers, and allied health professionals working on cystic fibrosis across Europe. In H2020 projects, they contribute clinical expertise, patient registry infrastructure, and best-practice guidelines to drug development and clinical trial consortia. Their role spans supporting first-in-human trials for new CF therapies, facilitating personalised treatment approaches using intestinal organoids, and helping build paediatric clinical trial networks. They serve as the connective tissue between CF research teams, ensuring clinical standards and patient-centred outcomes across multi-country trials.

What they specialise in

How they've shifted over time

Early participation (2015–2017) centred on experimental therapeutics — specifically RNA-targeting oligonucleotides and the F508del mutation, reflecting a focus on early-stage drug discovery for CF. By 2018, the focus shifted toward clinical development infrastructure: pivotal clinical trials, mutation-independent therapies, and broader paediatric trial networks (c4c). This evolution shows a move from supporting individual experimental compounds to building the clinical and regulatory infrastructure needed to bring CF treatments to patients at scale.

ECFS is moving from project-level clinical support toward becoming a platform organisation for paediatric and rare-disease clinical trials across Europe.

How they like to work

ECFS participates exclusively as a partner, never as coordinator — consistent with their role as a professional society that contributes clinical expertise and network access rather than leading project management. With 80 unique consortium partners across 20 countries, they operate in large, multi-stakeholder consortia typical of clinical trial projects. This broad network makes them a valuable "bridge" partner who can connect consortium members to the wider CF clinical community across Europe.

ECFS has collaborated with 80 unique partners across 20 countries, reflecting the pan-European nature of clinical trial consortia. Their network spans academic hospitals, pharma companies, and patient organisations involved in CF and paediatric medicine.

What sets them apart

ECFS is the central professional society for cystic fibrosis in Europe, giving it unmatched access to the CF clinical community — registries, treatment centres, and specialist physicians across the continent. Unlike individual hospitals or research groups, ECFS can mobilise multi-country clinical networks for trials and provide disease-specific best-practice guidance. For any consortium working on CF therapies or rare respiratory diseases, they are the natural gateway to the European CF clinical ecosystem.

Highlights from their portfolio

• PRO-CF-MED
  Largest single EC contribution (EUR 230,720) and the earliest project — a first-in-human clinical trial for an RNA-targeting oligonucleotide therapy for CF.
• c4c
  Broadest scope project building pan-European paediatric clinical trial infrastructure, running until 2025 with EUR 185,196 — signals ECFS expanding beyond CF into general paediatric trials.
• HIT-CF
  Pioneering personalised treatment approach using intestinal organoids for patients with ultra-rare CFTR mutations, pushing toward precision medicine in CF.