A medical doctor and professor of pediatrics at Duke University makes the point perfectly. We wouldn’t think, given his credentials, that Dr. Danny Benjamin would have this kind of problem, but he’s also a parent. Any of us with young children can relate. He has a 10-year-old son who takes seven medications every day. Many of these prescriptions lack age-appropriate dosing information – how much to take – as well as in what form (liquid, tablets, chewables). So, just like the rest of us, the doctor has to use very rudimentary methods to estimate the various doses:
Every day, I use a kitchen knife to cut up Jack’s pills – to give him a ‘ballpark’ dose…You can’t think of children as miniature adults.
Dr. Benjamin goes on to discuss that how children metabolize drugs and medications is not readily predictable. There are many reasons for this, including the fact that their rates of growth and development vary widely. The proportions of fat, protein and water that make up their body weight change very dramatically during the transition from infancy to childhood. For example, a newborn’s weight is about 70 to 80 percent water. This decreases to 60 percent by five months. And food moves more quickly through their digestive systems. Their kidneys are smaller. Their livers are smaller. All these differences affect how drugs work on children.
Here are some other interesting facts:
- Before 1998, nearly 70 percent of all the drugs prescribed for children had very, very little scientifically verified safety, dosing and effectiveness information.
- The Best Pharmaceuticals for Children Act (BPCA), passed by Congress, encourages clinical research trials on medications for children across all therapeutic areas.
- Research is being conducted on drugs designed and developed for newborn and premature babies through children age 18 in the US.
- As of 2008, according to the FDA, between 50% and 60% of prescription drugs and medications used to treat children had been researched and studied in some part of the pediatric population.
- At present, clinical trials are being conducted on drugs to treat children for a host of problems, including heart disease, infectious diseases, cancer, respiratory diseases and many others.
The BPCA is an important player in the quest for right drug, right dose. Federal law and FDA regulations require that all drugs approved for use in the US be tested for safety and effectiveness in specific populations, at specific dosages and for specific time periods. In 1997, Congress created an incentive to encourage drug makers and developers to test medications specifically for children when and if the drugs were to be prescribed for patients under 18. One significant discovery during the ensuing clinical trials was that, as suspected, pediatric dosing recommendations up until that time had been little more than ‘guesstimates’.
The incentive was extended in 2002, as part of the BPCA, and again in 2007. Related legislation, the Pediatric Research Equity Act (PREA), was passed in 2003 and reauthorized in 2007. These two acts together, the BPCA and the PREA, have inspired increased research and development into safer, more effective drugs for children. The BPCA, by the way, is scheduled to ‘sunset’ later this year unless is extended once more or made permanent by Congress.
The Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) is the lead agency at the NIH, helping to direct the BPCA pediatric clinical research trials as scientists work to develop safe, effective and appropriate children’s drugs, treatments and medical equipment. The trials include 16 broad disease categories, including cancer, heart disease, infectious diseases, newborn and premature babies, medical equipment and devices, and respiratory illness. The BPCA finds ways for studying patented and off-patent (generic) drugs.
If you are a parent, remember that good communication between you and all of your child’s healthcare providers is vital. Feel free to ask any and all questions that will help you understand the instructions you are given regarding the medications prescribed for your child. Here are some other suggestions:
- Have any of the medications recommended for my child been specifically tested for use in children?
- How can I tell if my child’s medications have been studied in children?
- Would my child benefit from, or be a good candidate for, a clinical trial?
- Is my child presently taking any medicines that have not been studied in children?
- How long do most clinical trials last? How many patients are needed for clinical trials?
For more information, check out the BPCA home website: BPCA.
Special thanks to NIH Medline Plus, Medicines for Children.