Testing Medications: Who's Being Left Out?En Español (Spanish Version)
Children get sick, just like adults do. But many times the only medicines doctors can prescribe for children have never been tested in children. Why is that? And how could this prescription practice affect you and your family?
When drug companies wish to manufacture and market a new drug, they must first receive approval from the United States Food and Drug Administration (FDA). This is done by submitting scientific evidence proving that the drug is safe and effective.
That evidence is collected in experiments, usually first conducted in test tubes and laboratory animals, and then in human studies called clinical trials. Clinical trials take several years and cost millions of dollars to conduct.
The data collected during clinical trials is summarized in detail on an approved drug's labeling. While much of it may seem like medical jargon to the average layperson, it is often vital information for a treating physician. It can help them determine which medication may be best for an individual's condition. The research will also help you and your doctor weigh the benefits and potential harms of any medication.
Historically, certain segments of the population have been largely omitted from clinical trials of new drugs. These groups include children, women of childbearing potential, and elderly people.
There are several reasons for this:
Safety is a concern in all clinical trials, but it can be of particular concern with regard to populations who may be more vulnerable to adverse effects from drugs—such as children, developing fetuses in pregnant women, and elderly people.
Duplicating expensive trials in different populations may not be cost-effective for the company footing the bill.
Some experts argue that it is unethical to expose certain groups of people to the risk associated with taking an experimental drug. And in the case of children, it has been argued that it is unethical to enroll them in clinical trials when they are not old enough to give consent themselves. They would be too young to fully understand the effects of a medication trial.
On the flip side, others question if it is any less ethical than treating children with drugs that have not been specifically tested in children. In the end children may end up being exposed to medication with unclear benefits or harm in the pediatric population.
It can also be harder to have children in clinical trials. Many times they may initially agree to participate, but are lost during follow through.
Trials that are well-controlled may also require several different types of testing. Many times, it is difficult to get blood or urine tests from children. Children can also be more scared. Many facilities are not built for children. Everything from medical equipment, or how tests are done, are set up for an adult not a child. It makes a difficult situation more difficult.
Doctors are faced with a bit of a conundrum when prescribing medicines to certain patients. To many doctors, that is cause for concern. The lack of testing means a lack of knowledge regarding clinical effect, proper dosing, and potential side effects.
Children, many experts point out, are not simply small adults. You cannot just cut a drug dose in half and expect it to work the way it works in an adult. Similarly, geriatricians reject the argument that elderly people respond predictably to the same drugs shown to be safe and effective in younger adults.
Trials for children are needed for age-specific medications that will determine the best treatment available.
Fortunately, changes are happening. The FDA has helped to protect a company's patent for a longer period of time. This gives the company a financial advantage which they can use to pay for trials in special populations like children or elderly. Drug companies are also using pediatric experts to help them guide the complex process of the trials.
Although it appears to be slow going, drug companies, health organizations, and the FDA are making progress in getting children involved in clinical trials.
In the meantime, your best bet, as always, is to be an informed consumer. Whenever you or your child is prescribed a medicine, ask your doctor and pharmacist if the drug was specifically tested in your age group and what the potential risks associated with the drug are.
It is important to remember that a drug that has not been tested in a certain population is not necessarily dangerous to that population and
taking the drug to treat a condition may be riskier than taking it. In many cases, doctors may have substantial experience using that drug to treat people in that population and, if so, they should be able to tell you how the drug has worked in their experience.
American Academy of Pediatrics
Center for Drug Evaluation and Research
US Food and Drug Administration
College of Family Physicians of Canada
Children Neglected in Clinical Drug Trials. American Academy of Pediatrics website. Available at: http://www.aap.org/en-us/about-the-aap/aap-press-room/Pages/Children-Neglected-in-Clinical-Drug-Trials.aspx. Updated April 28, 2012. Accessed November 20, 2012.
Clinical Trials in Children. World Health Organization website. Available at: http://www.who.int/ictrp/child/en/. Accessed November 20, 2012.
Drug Research and Children. U.S. Food and Drug Administration website. Available at: http://www.fda.gov/Drugs/ResourcesForYou/Consumers/ucm143565.htm. Updated August 2011. Accessed November 20, 2012.
How FDA Evaluates Regulated Products: Drugs. U.S. Food and Drug Administration website. Available at: http://www.fda.gov/AboutFDA/Transparency/Basics/ucm269834.htm. Updated April 4, 2012. Accessed November 20, 2012.
Last Reviewed November 2012