Web-based international studies in limited populations of pediatric leukemia.
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).


METHODS
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.
Valsecchi
Maria Grazia
MG
Unit of Medical Statistics, Department of Clinical Medicine and Prevention, University of Milano-Bicocca, Via Cadore, Monza (MI), Italy. grazia.valsecchi@unimib.it
Silvestri
Daniela
D
Covezzoli
Anna
A
De Lorenzo
Paola
P
D002675:Child, Preschool; D006801:Humans; D007391:International Cooperation; D020407:Internet; D015201:Meta-Analysis as Topic; D054198:Precursor Cell Lymphoblastic Leukemia-Lymphoma / Q000628:therapy*; D011446:Prospective Studies; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D012107:Research Design; D012307:Risk Factors