Disease-specific clinical trials networks: the example of cystic fibrosis

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. Conclusion: 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.