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.

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.

What is Known: • 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 .