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If your pediatrician prescribes your child a drug to treat a disease or condition, it's likely to be an "off-label" use, with no specific instructions given for kids by the drug company who produced it.

But that's not to say the doctor is doing anything wrong – since most drug testing happens on adults, not kids, and most drugs prescribed to children are off-label. A new position statement from the American Academy of Pediatrics on the topic highlights the issues and suggests while there is much to do, great strides have been made in ensuring the safety and efficacy of drugs prescribed to children in the United States.

Canadian experts who work in the field say Canada lags behind the United States on supporting drug research with children – the Food and Drug Administration, for instance, has the authority to demand pharmaceutical companies whose products are widely used on children to conduct research related to kids. Health Canada does not have that mandate or power.

What off-label means

Most drugs used on kids are off-label, which simply means there is no mention of children in their labelling. Everything from painkillers such as morphine – which most experts agree is the most pressing category of drugs to research – to antibiotics and cancer drugs.

Some medications such as anti-seizure drugs are approved for use in kids older than 12, but are used with younger children, says Michael Rieder, a professor of pediatrics at the University of Western Ontario.

"Many psycho-active drugs that are not approved are commonly used in kids," he says, adding that this is the biggest growth area in drug therapy for children.

This is a long-standing practice. A popular asthma drug, the Ventolin inhaler, was approved five years ago for use in children, despite being used off-label for decades, says Dr. Rieder, also the chair of the Drug Therapy and Hazardous Substances Committee of the Canadian Paediatric Society.

Why are so few drugs approved for use in kids?

In short, we've been squeamish about conducting research on children, it's expensive and can be hard to do on small populations of children with particular diseases and disorders. Thus, kids end up being "therapeutic orphans," as a common medical phrase goes. About 70 per cent of drugs prescribed in Canada have no instructions related to children.

Dr. Rieder wrote a 2011 position paper for the Canadian Paediatric Society calling for greater research into the use of drug therapy on children. He's optimistic the AAP paper along with a forthcoming paper on the topic by the Council of Canadian Academies commissioned by Health Canada may spur change. He and the drug therapy committee of the CPS will be updating their statement after they've read the CCA paper.

Why off-label prescriptions for kids is not necessarily a bad thing

According to the new statement, physicians should be clear that "off-label" "does not imply an improper, illegal, contraindicated, or investigational use."

While adverse reactions and even tragedies have happened – in his 2011 paper, Dr. Rieder cites a 1937 mass poisoning which occurred after children were given a new anti-microbial drug – many of the off-label drugs are successful in treatment and proving it would bolster the practice of medicine.

"Evidence-based medicine has its own dogma; just because you don't have evidence for it doesn't mean it doesn't work. But for whatever reason no one's been able to get a trial to show that it works."

But evidence can, among other things, ensure drug access, he adds. Some expensive drugs are covered by provincial and private health plans; some families find that because a drug is being prescribed off-label, their insurer uses that loophole to deny coverage.

Dr. Shinya Ito, the head of clinical pharmacology and toxicology at The Hospital for Sick Children (SickKids) says doctors and pharmacists undergo an intense study when a new drug comes available to them to decide whether it can be used at the Toronto hospital. He says dosage calculations are based on how quickly a drug will move through a child's system, among other metrics.

What else the new AAP recommendations suggest

Since new legislation passed about a decade ago in the U.S., more than 500 labelling updates have been made to pharmaceuticals. Still, the AAP urges more focus in the area of pre-term and full-term newborns, children under the age of two and children with chronic and/or rare diseases. The group reminds pediatricians that the use of a drug either on- or off-label, should be based on "sound scientific evidence, expert medical judgment, or published literature." It also calls on its members to advocate for more study of drugs in children.

What else can Canada do?

Dr. Ito says more resources and infrastructure could allow childrens' hospitals across the country to collaborate on research studies with their patients. And he points out that no drug will ever be tested on children until after it's been tested in an adult population.

Dr. Rieder says we need to face the paradoxes involved in treating children with drugs only approved for adults.

"There are some people in our community who think it's unethical to do drug research in children but it's ethical to treat them with unproven drugs. Which I think is a curious double-standard. The ethical thing is to try to do the thing that is right for the child."

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