Seventy-five percent of medications frequently prescribed to children — from anti-asthmatic agents to antibiotics — are not approved by the Food and Drug Administration (FDA) for use in children. A common reason: The drug manufacturer didn’t ask for FDA approval.
Why? Manufacturers often decide not to test and market their medicine for children because such studies are both difficult and expensive. This “hands off” approach may also allow them to avoid costly liability. Never making the claim that a medicine is “safe and effective in children” (a claim that the FDA carefully regulates through its own strict testing requirements) may protect the company from lawsuits should a child suffer an adverse reaction.
Lack of FDA approval, however, does not mean a medicine is unsafe or ineffective for children. In fact, most medicines have been tested in children at least to some degree Many of these studies, conducted using rigorous scientific methods, are funded by the federal government’s National Institutes of Health (NIH) in order to provide doctors with the data in which industry does not invest.
The results of NIH-funded studies usually can be found in the scientific literature, which brings us to the second part of my answer: What should doctors do for a child who might require medications that are not FDA approved for children?
They must start by doing their homework.
Doctors must monitor the scientific literature for case reports of the medicine’s use in children, and must determine whether NIH-supported scientists have conducted studies on the drug. They will often find excellent evidence supporting safety and effectiveness, even if the FDA has not officially approved the medicine for pediatric use.
Finally, there is the question of wisdom. Is it unwise to given anti-seizure or anti-cancer agents to a child with these severe disorders, even if such medications have only been tested mainly in adults? The answer, of course, is “it depends.”
In view of a severe disorder with potential life-long consequences (childhood depression and ADHD are two examples, just as surely as are asthma and diabetes), the parents and doctor must work together to review the evidence, weigh the risks of treating vs. not treating, and consider the risks and benefits of all treatment options.
To either simply reject medications out-of-hand, or to embrace them without a careful understan-ding of what the science says, is what’s unwise. Appropriately used after careful evaluation of the scientific evidence and the child’s circumstances, medications can be a very wise choice for many children. Inappropriately used, let the buyer beware.