Untested medicine

A drug made for adults might save a child, but use is often based on an educated guess

October 16, 2007|By Stephanie Desmon | Stephanie Desmon,Sun reporter

The debate over infant cough and cold medicines has brought to light an open secret in medical circles: Most drugs given to children have never been tested for them, forcing physicians to sometimes use a best guess in determining dosing, efficacy and even safety.

Federal regulators and physicians have recognized the need for more pediatric drug research, realizing that children are not just small adults. Their bodies process medication differently - sometimes faster, sometimes slower, sometimes turning it into poison. Yet, adult drugs are routinely prescribed for children despite the lack of scientific evidence.

"If you extrapolate from an adult dose to a pediatric dose, you may be right ... you may be wrong," said Dr. Joseph M. Wiley, chief of pediatrics at Sinai Hospital in Baltimore.

Federal reform legislation passed a decade ago - and reauthorized this summer - made strides toward ensuring that new drugs entering the marketplace for children would be fully investigated before being prescribed. But it has failed to completely fill the gap in research, particularly in older medications. The Government Accountability Office, the investigative arm of Congress, said this year 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 startling results: Of the older drugs newly studied in children, 87 percent were being improperly prescribed. Children were getting medicine that didn't work, being overdosed, underdosed or exposed to previously unknown side effects.

"We found out that you can't predict how kids are going to handle things," said Dr. Dianne Murphy, director of the Food and Drug Administration's office of pediatric therapeutics.

Once a medication is FDA-approved for use in adults, it can be legally prescribed to anyone. Yet the data drawn from rigorous work determining safe doses, effectiveness and possible side effects applies only to full-grown adults, not children whose brains are still developing, who are still growing and whose body chemistry is immature.

The practice is called prescribing off-label, and it is done safely by physicians every single day. Some argue that if doctors stuck to what is approved for children, many sick kids would die and others would suffer unnecessarily.

Doctors say they prescribe drugs that haven't been studied in children because they have no alternative. They choose doses based on the child's weight, what they know about the drug and how it is metabolized in the body, and on what they know about the disease. Often, they have to rely on the experiences of others who have used the drug in children to make determinations. It's not all guesswork, they say.

A study published in the March issue of the Archives of Pediatrics & Adolescent Medicine showed that off-label drugs were used in nearly 80 percent of patients at major children's hospitals in 2004.

Dr. Anthony D. Slonim, an intensive-care specialist at Carilion Clinic Children's Hospital in Roanoke, Va., and one of the study's authors, said off-label drugs are a vital part of what he does. Morphine, for example, is the best thing he has to soothe severe pain while also calming patients.

In emergency situations, he administers medicines to children, including some that aren't approved for them, without asking parents.

"I don't have time when a child is critically ill in the ICU to talk about the numerous drugs I'm going to give to the child to save his life. We're left with, `Do the right thing for the child. The heck with the off-label use,'" Slonim said.

In nonemergencies, parents can ask doctors and pharmacists about a drug's approval history.

Slonim thinks that off-label has a bad connotation. He says it just means that a drug hasn't gone through the FDA approval process for that use. He says that for certain medications that have been used for such a long time - such as morphine - perhaps they should be able to get some sort of secondary approval based on real-life experience: "Shouldn't we just give them a bye?"

Failure to test medications before prescribing them is what led to the law that created the FDA in 1938. Sulfanilamide, a drug used to treat strep infections, had shown great success as a cure in the 1930s and was used safely in tablet and powder form. Doctors wanted a liquid form, primarily for children. Because the sulfa drug did not dissolve easily in water, a drug company chemist created an elixir with diethylene glycol, a chemical normally used as an antifreeze. More than 100 people, mostly children, died.

Safety studies had not previously been required.

New laws meant improved testing for drugs in adults, not children.

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