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.
Döring, G., Elborn, J. S., Johannesson, M., de Jonge, H., Griese, M., Smyth, A., & Heijerman, H. (2007). Clinical trials in cystic fibrosis. In Journal of Cystic Fibrosis (Vol. 6, pp. 85–99). https://doi.org/10.1016/j.jcf.2007.02.001