Posted On: May 17, 2012 by Patrick A. Malone

Drug Labels Still Leave Pediatricians Playing a Guessing Game

Nearly 50 years ago, Dr. Harry Shirkey coined the phrase "therapeutic orphan" to describe children who took prescription medicine. Because drug studies among a pediatric population were complicated and offered little financial return for pharmaceutical companies, no one really knew how drugs developed for adults would affect children who took them. Providers prescribed them based on their best guess about how to tailor an adult dose to a child.

In 1997, Congress passed the Food and Drug Administration Modernization Act (FDAMA). It offered incentives for pediatric drug development in the form of additional marketing exclusivity. Progress has been made in drug research among children, and drug labeling for these patients has improved.

But not enough.

According to a recent study published in the Journal of the American Medical Association, (JAMA) not even half of drug labels have information on safety and proper dosing for kids. More than 500 drug labels were evaluated.

According a Reuters story about the new study, drugs still pass the FDA approval process and make it to market without much research on how they should be given to children for the best treatment and least harm.

Pediatric research remains difficult—the number of children with a given disease usually is lower than that of adults, and the ethical concerns are thornier because minors can’t provide informed consent.

The JAMA study looked at hundreds of drugs that treated a range of problems from high blood pressure to allergies.

Labels were deemed “adequate” to use in children if they included how effective the drug is, if it’s safe in younger children and teens and enumerated doses of children of different ages.

Two hundred thirty-one drugs were adequately labeled and 29 others carried dosing for some younger age groups, but not all. Still, slightly more than half of all labels lacked useful pediatric information. Even after tossing the results of drugs that were irrelevant to kids (prostate cancer treatments, for example), 44 in 100 labels offered no information for young patients.

Some newer drugs, those approved by the FDA between 2002 and 2008, also lacked safety and dosing information for kids. But the good news is that drugs commonly prescribed for children—asthma meds and vaccines, for example—often undergo rigorous testing.

Still, kids get cancer, arthritis, and they suffer lung and heart disease; studies on how drugs to treat them react in children often fall by the wayside. And the “how much and at what cost” treatment guessing game continues.

Prescribing medications to children that have not been labeled for them is an off-label use (doctors are allowed to prescribe them, but drug companies may not market them for an unapproved use). That might result in benefit, no therapeutic effect or harm (adverse reactions). (To learn more about medication errors, link here.)

Doctors uncertain about a drug’s effect in a kid might be reluctant to prescribe it off-label, and that could deprive the patient of a real benefit.

If a child receiving an unlabeled drug therapy doesn’t respond well, is that because the drug just didn’t work? Was the dosing incorrect? If there are side effects, do they denote a marker of the drug itself, or of an incorrect dose?

The researchers concluded, “Labeling with pediatric information in only 46 percent of products is still insufficient. Legislation to increase pediatric clinical trials and require the resulting information be added to labeling is necessary. The current legislation expires in 2012 without reauthorization.”

That lapse is not acceptable. Contact your congressional representatives to appeal for completion of a process that has shown benefit but is not finished.

And if your child is prescribed a drug lacking information about appropriate doses for certain age groups, and possible side effects, ask your doctor and pharmacist for more information. If they’re uncertain, because there’s no science to inform them, ask if there are alternative therapies.

Children should not be guinea pigs.

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