Background: Recent progress in cancer research leads to the characterization of small subgroups of patients by genetic/biological features. Clinical studies in this setting are frequently promoted by international networks of independent researchers and are limited by practical and methodological constraints, not least the regulations recently issued by national and international institutions (EU Directive 2001/20/EC).
Procedure: We reviewed various methods in the design of international multicenter studies, with focus on randomized clinical trials.
Results: This paper reports our experience in planning and conducting international studies in childhood leukemia. We applied a decentralized study conduct based on a two-level structure, comprising a national and an international coordinating level. For the more recent trials this structure was implemented as a web-based system. This approach accommodates major legal requirements (e.g., safety reporting) and ensures Good Clinical Practice principles by implementing risk-oriented monitoring procedures.
Conclusions: Setting up international non-commercial trials is increasingly complicated. Still, they are strongly needed for answering relevant questions in limited populations.
(c) 2007 Wiley-Liss, Inc.