Disease-specific clinical trials networks: the example of cystic fibrosis
Open Access
- 15 March 2016
- journal article
- research article
- Published by Springer Science and Business Media LLC in European Journal of Pediatrics
- Vol. 175 (6), 817-824
- https://doi.org/10.1007/s00431-016-2712-z
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.Keywords
This publication has 16 references indexed in Scilit:
- Sharing Individual Patient Data from Clinical TrialsThe New England Journal of Medicine, 2015
- New pharmacological approaches for cystic fibrosis: Promises, progress, pitfallsPharmacology & Therapeutics, 2015
- Lung clearance index: Evidence for use in clinical trials in cystic fibrosisJournal of Cystic Fibrosis, 2013
- Clinimetric properties of bronchoalveolar lavage inflammatory markers in cystic fibrosisEuropean Respiratory Journal, 2013
- A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defectThe Lancet Respiratory Medicine, 2013
- A new Clinical Trials Directive to boost academic trials?The Lancet. Respiratory Medicine, 2013
- CFTR biomarkers: time for promotion to surrogate end-pointEuropean Respiratory Journal, 2012
- Controlled clinical trials in cystic fibrosis — are we doing better?Journal of Cystic Fibrosis, 2006
- Standards of care for patients with cystic fibrosis: a European consensusJournal of Cystic Fibrosis, 2005
- Organizing multicenter trials: Lessons from the cooperative oncology groupsPreventive Medicine, 1991