Pediatric Drug Testing

Harry Shirkey, a medical practitioner, coined the term "therapeutic orphans" more than 40 years ago to describe how children are overlooked in medical research. His observations remain true today. Despite the best efforts of the American Academy of Pediatrics (AAP) and the Federal Drug Administration (FDA), nothing has really changed.

Guesstimating Medicine

The majority of drugs given to children have never been tested specifically for them. Physicians are prescribing drugs based on a "best guess" as to the dosage, efficacy and even safety. It's becoming clearer to federal regulators and physicians alike that children are not "small adults." With their bodies in different stages of development, they process medication differently; sometimes faster, sometimes slower and sometimes turning it into poison. Their body chemistry has not fully matured and yet adult drugs are being prescribed to children on a regular basis. Dr. Joseph M. Wiley, chief of pediatrics at Sinai Hospital in Baltimore, said, "If you extrapolate from an adult dose to a pediatric dose, you may be right ... you may be wrong."

The Path to Pediatric Testing

The road to pediatric drug testing has been a rocky one. Nobody wants to use children as guinea pigs, but the problem remains that drugs are being given to children in potentially unsafe doses.

The FDA understands that giving medications to children for which there is only adult data available could be harmful, considering that children have dosing concerns and side-effect risks that differ from adults. The latest legislation has made some headway in the battle against the lack of proper labeling; yet, it still fails to completely overcome the absence of research, particularly in the older medications.

Last year, the Government Accountability Office said that only one-third of the drugs prescribed for children have been studied and labeled for pediatric use. The pediatric research that has been done includes some frightening results: Of the older drugs just recently being studied in children, 87 percent were being improperly prescribed. Children were getting medicine that didn't work at all, was under- or overdosed, or had some possibly deadly side effects.

Dr. Dianne Murphy of the FDA's Office of Pediatric Therapeutics had this to say: "We found out that you can't predict how kids are going to handle things." The problem is that once a medication has been approved for use in adults, it legally can be prescribed to anyone for any reason, despite the lack of pediatric testing or proper labeling.

Off-Label Prescribing

This common practice of prescribing medicine to children that has only been tested and labeled for adult use is called "off-label prescribing" and it's done by physicians every day. A recent study published in the Archives of Pediatrics and Adolescent Medicine showed that off-label drugs were used in nearly 80 percent of major children's hospitals in 2004.

What needs to be understood is that "off-label" doesn't just mean it hasn't been properly tested on children, but additionally that it might be prescribed for a purpose for which it was never intended. Dr. Murphy says there are about 200 older drugs that have been studied in children, but new products come on the market all the time. Their studies, while underway, will take time to complete. That means a lot of untested medications are made available to doctors for off-label prescribing to children.

Consequences of Off-Label Prescribing

In hospitals and medical offices, pediatricians write off-label prescriptions every day with the best of intentions. They make an educated guess about which drug to prescribe and what would be an appropriate dosage for a child of a particular weight and age, hoping they're right. The problem with this practice is that the children can pay the price with unexpected side effects; worsening of the original complaint; allergies to the chemicals; and even coma and/or death.

Unfortunately, the actual facts in this matter have been difficult to determine. The British Pediatric Surveillance Unit believes this could be due to many factors including a reluctance to report (even in an anonymous way) and the number of deaths due to off-label prescribing of drugs to children. However, the European Medicines Agency believes that there is sufficient evidence that harm actually occurs and is under-reported.

What it comes down to is whether a pediatrician's "best guess" as to dosage and efficacy of a drug is going to be good enough for parents, and whether they should be accepting, in blind faith, every prescription written.

Questions for Parents to Ask Their Pediatrician

So, what should parents do if their child may need a prescription? Before accepting a prescription from their pediatrician, parents should ask the following important questions and then thoughtfully consider whether to give their child that chemical: Would a wait-and-watch approach be advisable in this instance? Is this the only treatment available or do I have options that don't include drug therapy? What are the side effects of this drug? Has the drug been tested for pediatric use? Is this the use for which it was intended or are you prescribing this off-label? Can we begin with a smaller dosage and then increase it only if necessary?