Klaus Rose, MD, MS

Many studies with a stated goal of advancing child health care in administratively-defined minors have no clinical value and may even harm young patients.

Institutional review boards and ethics committees have seen it as their main task in the past few decades to protect children from questionable studies; however, they are faced with a new challenge with pediatric drug development.

Imposing the FDA on-label/off-label framework on administratively defined "children" resulted in a regulatory demand for pediatric studies that had no basis in clinical medicine, with the exception of the small group of preterm newborns.

Pediatric drug development, which has a complex background and history, has fundamental catches based around how children are defined in the context of clinical studies.