title: Clinical trials in cystic fibrosis.
abstract: In patients with cystic fibrosis (CF), clinical trials are of paramount importance. Here, the current status of drug development in CF is discussed and future directions highlighted. Methods for pre-clinical testing of drugs with potential activity in CF patients including relevant animal models are described. Study design options for phase II and phase III studies involving CF patients are provided, including required patient numbers, safety issues and surrogate end point parameters for drugs, tested for different disease manifestations. Finally, regulatory issues for licensing new therapies for CF patients are discussed, including new directives of the European Union and the structure of a European clinical trial network for clinical studies involving CF patients is proposed.
authors.affiliation: gerd.doering@med.uni-tuebingen.de
authors.forename: Gerd
authors.initials: G
authors.lastname: Döring
authors.forename: J Stuart
authors.initials: JS
authors.lastname: Elborn
authors.forename: Marie
authors.initials: M
authors.lastname: Johannesson
authors.forename: Hugo
authors.initials: H
authors.lastname: de Jonge
authors.forename: Matthias
authors.initials: M
authors.lastname: Griese
authors.forename: Alan
authors.initials: A
authors.lastname: Smyth
authors.forename: Harry
authors.initials: H
authors.lastname: Heijerman
mesh_terms: D002986:Clinical Trials as Topic; D003550:Cystic Fibrosis / Q000628:therapy*; D006801:Humans; D017063:Outcome Assessment, Health Care / Q000379:methods / Q000592:standards; D018579:Patient Selection; D012107:Research Design / Q000592:standards; D013673:Technology Assessment, Biomedical