Dianne Murphy, MD, is the Director of FDA's Office of Pediatric Therapeutics (OPT). The mission of OPT is to enforce a Congressional mandate that assures access for children to innovative, safe and effective medical products. Historically, many medical products have not been tested for use in children, leading to an increase in adverse events and the use of ineffective products. "The Office of Pediatric Therapeutics is supposed to help the FDA work to develop the knowledge that is needed to use medicines and devices properly in children," says Murphy. While the Office of Pediatric Therapeutics is taking significant steps by implementing regulatory science initiatives by working with small business and industry entities, Murphy explains that "...one of the biggest areas that remains almost completely unstudied is neonates to prematures." She further notes that young children and neonates require the development of a new directional endpoint that can better help us to not treat children with our best guess, but with knowledge.
