title: Disease-specific clinical trials networks: the example of cystic fibrosis.
abstract: This article describes the steps of the development and the structure of a disease-specific clinical trials network for cystic fibrosis in Europe. Activities such as reviewing study protocols, feasibility assessments, training and standardizing of procedures, and outcome measurements help to bring high-quality clinical trials to the patients. Cooperation with the pharmaceutical industry, other research networks, patient organizations, and regulatory agencies is very important throughout all activities.
  
  
  CONCLUSIONS
  The European Cystic Fibrosis Society-Clinical Trials Network facilitates the development of new treatments for a rare disease and could be a prototype for other diseases.
  
  
  BACKGROUND
  • Clinical research has led to the first approved treatments targeting the basic Cystic Fibrosis defect. • For a rare disease like Cystic Fibrosis, multicenter international collaboration is needed to obtain solid evidence when testing possible new treatments. What is New: • The Clinical Trials Network established by the European Cystic Fibrosis Society has grown to a fully operational network with well-defined structures, procedures and partnerships. • Standardization of outcome parameters, protocol review, feasibility assessment and other activities help to develop high quality, efficient, relevant and feasible clinical trials, with the aim to bring new treatments to the patients.
authors.affiliation: Department of Pediatric Pulmonology, University Hospital Leuven, Herestraat 49, 3000, Leuven, Belgium. christiane.deboeck@uzleuven.be.
authors.forename: Kris
authors.initials: K
authors.lastname: De Boeck
authors.affiliation: Department of Development and Regeneration, KU Leuven - University of Leuven, Herestraat 49, 3000, Leuven, Belgium.
authors.forename: Veerle
authors.initials: V
authors.lastname: Bulteel
authors.affiliation: Université Paris Descartes, Sorbonne Paris Cité, Paris, France.|Service de Physiologie et Explorations Fonctionnelles, Hôpital Cochin, AP-HP, 27, rue du fg St Jacques, 75014, Paris, France.
authors.forename: Isabelle
authors.initials: I
authors.lastname: Fajac
mesh_terms: D035843:Biomedical Research / Q000592:standards*; D002985:Clinical Protocols / Q000592:standards*; D002986:Clinical Trials as Topic; D003299:Cooperative Behavior; D003550:Cystic Fibrosis / Q000628:therapy*; D005060:Europe; D006801:Humans; D010043:Outcome and Process Assessment, Health Care
keywords: Clinical trials; Cystic fibrosis; Rare diseases; Research network