Early Phase Clinical Trials in Pediatric Hematology and Oncology

 

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Abstract

Pediatric oncology is an unrivaled success story in the recent history of medicine. This success is mostly based on a persistent refinement of evidence based therapeutic concepts. With that regard physicians and their staff are highly experience in the conduct of prospective evidence based trials and are therefore competent partners for the pharmaceutical industry. In times of personalized medicine the individual target population is diminishing and the borders of indications are not more disease based. A situation that requires new concepts from the industry. Therefore children with cancer could benefit early from the current developments as well as the pharmaceutical industry could benefit from the legislative incentives through highly recruiting and well conducted prospective trials. Pivotal is a functional platform of communication in order to maintain a close dialogue between academia and pharmaceutical companies.

Zusammenfassung

Wie die Heilungsraten in der pädiatrischen Onkologie erzielt wurden, ist eine der großen Erfolgsgeschichten der Medizin der letzten Jahrzehnte. Erzielt wurden sie vor allem durch eine kontinuierliche Verbesserung evidenzbasierter Therapiekonzepte. Es stehen der pharmazeutische Industrie somit kompetente Partner mit langjähriger Erfahrung in der Durchführung prospektiver randomisierter klinischer Studien zur Verfügung. In Zeiten der personalisierten Medizin reduzieren sich die therapeutischen Zielgruppen, und die Indikationen dieser neuen Pharmaka überschreiten die Krankheitsgrenzen. Unter den gegebenen legislativen Voraussetzungen könnten sowohl Kinder mit onkologischen Erkrankungen als auch die pharmazeutische Industrie von professionell durchgeführten und gut rekrutierenden klinischen Studien profitieren. Wichtig ist dabei die Etablierung einer Kommunikationsplattform, um den Dialog zwischen der pharmazeutischen Industrie und akademischen Partnern aufrecht zu erhalten.

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