Drug trials in pediatric oncology: a multi-facetted problem.

In the field of childhood malignancies, the last 30 years have witnessed the advance of highly effective treatment regimens and high cure rates through the clinical application of a growing number of cytotoxic agents. The therapeutic success was mainly brought about by the empirically based joint efforts of cooperative clinical trials that employed effective cytotoxic drug combinations at the threshold of maximum tolerable toxicity. Systematic clinical drug trials, however, aimed at the approval of specific substances for use in pediatric oncology have been lacking. The following presentation will try to describe briefly the various facets of the problem involved in the systematic development of drugs for childhood malignancies as is manifested in Germany today.