When it comes to prescribing medication for children, there is often little data to support information about dosage, safety or efficiency. According to Scientific American, most clinical trials do not include children so the burden of deciding what treatments to use and how falls on physicians.
Scientific American reports that only 46 percent of drugs used in kids have been approved by the U.S. Food and Drug Administration (FDA) for pediatric use. In the past, that statistic was far worse.
The improvement to 46 percent in recent decades is attributed significantly to two pieces of legislation. One law, the Best Pharmaceuticals for Children Act, provides the incentive by offering exclusive sale rights for a short period of time. The other law is the Pediatric Research Equity Act, which says that if a drug can be used for the same purpose in children, it must undergo clinical testing in children for approval. The caveat of this is that children often get diseases that do not occur in adults.
“We need new legislation to fill in the holes. One of the concepts people have talked about within the field is maybe the requirements that go along with labeling should be based on mechanism of action of the drug and not necessarily the disease—then if you have cancer, in some ways, cancer is cancer.” said Kathleen Neville, chair of the Committee on Drugs at the American Academy of Pediatrics.
Decisions about medication in kids are often decided by small committees, who come up with guidelines on a hospital-by-hospital basis. Physicians also use LISTSERVS to obtain information about dosage and side effects with pharmacists, scour peer-reviewed literature and consider hospitals’ own patient experiences. However, these measures do not compare to the scrutiny needed to obtain FDA approval for a specific purpose, Scientific American reports.
Using a drug in any manner not approved by the FDA is known as “off-label”. According to Scientific American, off-label use constitutes the majority of pediatric pharmaceuticals. Justin Cole, a clinical pharmacist at Nationwide, says that over 90 percent of drugs are used off-label in the neonatal intensive care units because data is lacking even more in this area. In these cases, doctors are often prompted to ask parents for their permission before using the drugs.
Doctors are allowed to prescribe drugs off-label as they see fit, although it is illegal for pharmaceutical companies to promote such use. Because there is often not enough data on a drug’s effects in children, using a drug off-label is frequently the only choice available-this can be problematic.
Robert Nelson, deputy director and senior pediatric ethicist in the FDA’s Office of Pediatric Therapeutics, said, “I think it comes down to what is the quality of the data they are using, and that’s going to be variable,” according to Scientific American.
Children are not simply smaller versions of adults, Scientific American writes. Merely lowering the dosage so that it is proportional to their weight does not mean that the drug will work as desired. In fact, prescribing in this manner can have severe consequences. Bactrim, for instance, is often used to fight infections in adults but is ineffective in newborns because their livers cannot metabolize the drug. As a result, Bactrim can cause infants to suffer from jaundice and brain damage.
Furthermore, pharmaceutical companies have little motivation to get clinical trial data for kids since oftentimes they know the drug will be used off-label anyway. Sarah Erush, clinical manager in the department of pharmacy at The Children’s Hospital of Philadelphia (CHOP) said to Scientific American “They aren’t foolish. They know we’re going to use the drug anyway, but it’s really an extra burden on the provider”