Ethical issues in including pediatric cancer patients in drug development trials

Since the biology, pharmacology and toxicology of pediatric cancer is so different from cancer in adults, it is impossible to extrapolate from adult cancer experience; however, conducting studies in children with cancer is ethically and clinically very complex. This is especially so in phase 1 trials, aimed at dose finding for new modalities, where the chances to cure the child who failed standard therapy are marginal to non-existent. Hence, the child may be exposed to risks and discomfort which may be unbalanced by potential benefits. This situation, which is difficult for consenting adults, is much more complex when a parent gives proxy consent. This review addresses the ethical and clinical framework of such studies in the most vulnerable of children.