The process of drug development in pediatric oncology: a review of basic principals and a look into the future

Andre Tesainer Brunetto, Rafael Roesler, Ana Lucia Abujamra, Claudio Galvao de Castro, Gilberto chwartsmann, Algemir Lunardi Brunetto

This article will focus on basic concepts in the development of new agents for pediatric malignancies and future directions, while considering the limitations we have today and the lessons we have learned from adult cancers. In addition, basic ethical principles for conducting trials in children are summarized. The rising numbers of targeted agents that have become available over the last few years will significantly change the way clinical studies are planned and a shift towards the use of alternative end points, different selection criteria and innovative designs is necessary. Pediatric malignancies have specific particularities and disparities that distinguish them regarding the conduction and design of clinical trials, which may influence the use of novel compounds. Due to the rarity of these diseases, international groups have cooperated to increase accrual rates, and incentives to the pharmaceutical companies have been made available by regulatory authorities; however, despite this a lack of trial participation in developing countries still remains.