First there were reports antidepressants increase suicidal behavior in children. Then came news of a possible cancer risk from creams used to treat baby eczema. Even a drug for attention deficit hyperactivity disorder became suspect and sales were suspended in Canada after several deaths in children.
Part of the worry is that most drugs taken by children have not been adequately tested on them. Dr. Donald Mattison, chief of the obstetric and pediatric pharmacology branch of the National Institute of Child Health & Human Development near Washington, D.C., is working to change that. Late last year his office, acting under a 2002 law passed by Congress, ordered its first set of tests of drugs used in children. It's developing a priority list of drugs to examine next. BusinessWeek's Carol Marie Cropper talked with him by telephone about the effort.
What are the facts when it comes to testing drugs used by children?
More than 90% of the drugs used in newborns and in children up to the age of 12 have never been adequately tested in those age groups -- and most of them haven't been tested at all. Of the drugs used in adolescents, 75% have not been adequately tested to establish the appropriate dose and answer questions about efficacy and safety.
Why does this matter?
We're guessing that drugs are beneficial in children because they treat diseases that have some similarity to a disease in adults -- with no tests on effectiveness, adverse consequences, or proper dosage. Each treatment essentially becomes an experiment on a single child.
Children don't have the same water or body-fat composition as adults. The rate at which their livers metabolize a drug and at which their kidneys eliminate it are different. And some side effects are more of a problem in children. One of the issues with the eczema cream, Elidel, for example, is that it suppresses the immune system. We don't know the long-term effect that has in a child under 2, whose immune system is still developing.
How are you working to solve the problem?
There are about 200 off-patent drugs currently being used in children. That does not include drugs still under patent that went on the market before being tested under a 2003 law that requires pediatric studies of new drugs. We anticipate that we'll study six drugs each year, typically by awarding contracts to pediatricians or pediatric experts at academic health centers to perform clinical trials. Our initial focus was on drugs used in critically ill children. We're now in the process of looking at drugs that are commonly used in children. My hope is that in 15 years we'll be able to say we've looked at those.
What should parents do?
When your child gets a prescription, ask if the drug has been used a lot in children. If not, ask if another drug is available that has been used more or for a longer period of time, and [find out] why the physician thinks this is the most appropriate drug. It's important for parents to understand the efficacy and safety issues the pediatrician is balancing. Practitioners should tell parents what side effects have been observed and whether there are any they should be especially concerned about.
Because we don't have a large amount of data, it's important for parents to watch for and report any unusual effects. That will help both the pediatrician and the rest of us understand better which drugs to use and which ones we should stay away from.
Parents can also check the Food & Drug Administration's Web site (www.fda.gov) for drug alerts. There's a Pediatrics link for information about children's drug issues and a table showing the labeling changes that have come about because of voluntary testing under a 1997 law, which gives drugmakers an extra six months of marketing exclusivity in return for doing tests in children.