Pharmacotherapy for pediatric cardiovascular diseases: the role of off-label drug use in the 21st century.

Rigorous scientific investigation is the foundation for many of the remarkable advances in medical care that have occurred over the course of the last century. Patients with cardiovascular diseases, in particular, have received considerable benefit from such research, especially that which pertains to drugs and medical devices. Infants and children, however, have not benefited proportionately from this great increase in knowledge, due to several factors: the economic return on investment is often insufficient for industry to justify the commitment of substantial resources to meet the opportunity cost of a relatively small patient population; the research capacity for pediatrics has been limited; and child patients have been considered ethically unsuitable subjects for study. As a result, drug therapy of many sorts in pediatric patients has been guided too often by theory or extrapolation from practice with adults and by expert experience and good intentions rather than by evidence obtained through clinical trials. To this day, most pediatric pharmacotherapy is based on the off-label use of drugs.1 This must change.