Developing Safe and Effective Medicines for Children NIH MedlinePlus the Magazine
Children are not “small adults,” when it comes to medicines.“As a mother, if my child gets sick, I want him to get the right medicine, and I want it to be safe and effective,” says Dr. Anne Zajicek about her 12-year-old son Eli. “And the Best Pharmaceuticals for Children Act does that.”
Dr. Zajicek, MD, PharmD, is not only a mother. She is a pediatrician and the chief of the Obstetric and Pediatric Pharmacology Branch (OPPB) at the NIH’s Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
She is also helping to make drugs for children safe, effective, and prescribed in proper dosages as part of a long-range, multi-million-dollar clinical research effort being carried out under the Best Pharmaceuticals for Children Act (BPCA). The NICHD has the lead role in funding and overseeing much of the BPCA-related research at the National Institutes of Health (NIH), which covers drug and medical device development for newborn and premature babies, and children until the age of 18.
Since 2007 alone, more than 346 pediatric studies have been carried out under BPCA, involving 155,755 patients, according to the U.S. Food and Drug Administration (FDA). Since 1998, over 424 drugs have received specific pediatric labeling information changes. These drugs are used to treat many diseases, including asthma, sickle cell disease, ADHD, influenza, high blood pressure, leukemia, cerebral palsy, diabetes, skin diseases, and kidney failure.
Ballpark DosesDr. Danny Benjamin, MD, PhD, is a professor of pediatrics at Duke University and director of the Duke Clinical Research Unit pediatrics program. (See interview on following page.) He is also the father of a 10-year-old who takes seven different medicines every day. Many of them lack age-appropriate information about how much to take (dosing) and in what form (whether liquid or chewable). So, like many other parents in similar situations, Dr. Benjamin has to rely on simple techniques to estimate the doses.
“Every day, I use a kitchen knife to cut up Jack’s pills—to give him a ‘ballpark’ dose,” says Dr. Benjamin. “You can’t think of children as miniature adults.” He points out that how children metabolize drugs is not predictable because, among other things, their rates of growth and development vary.
For example, the proportions of fat, protein, and water that make up body weight change dramatically between infancy and childhood. A newborn’s weight is about 70 to 80 percent water but decreases to 60 percent by five months. Food moves through their digestive systems faster. And their kidneys and livers are smaller than adults’. All this affects how drugs impact children.
- Prior to 1998, about 70 percent of the drugs prescribed for children had too little scientifically verified safety, effectiveness, and dosing information.
- The Best Pharmaceuticals for Children Act (BPCA), passed by Congress, encourages clinical research trials on children’s medications across all therapeutic areas.
- As of 2008, an estimated 50% to 60% of prescription drugs used to treat children had been studied in some part of the pediatric population, according to the FDA.
- Research is being conducted on drugs developed for newborn and premature babies through children age 18 in the United States.
- Clinical trials are being conducted on drugs to treat children for a range of problems, including heart diseases, cancer, infectious diseases, respiratory diseases, and many more.
Questions to Ask Your Health ProfessionalGood communication between you and your child’s healthcare provider is very important. You should feel free to ask any and all questions that will help you understand instructions that your child’s healthcare provider may give you. This is especially important when it comes to medications that are prescribed.
- Have any of my child’s medicines been specifically tested for use in children?
- How can I tell if my child’s medicine has been studied in children?
- Is my child currently taking any medicines that have not been studied in children?
- Would my child benefit from or be a good candidate for a clinical trial?
- How long do most clinical trials last, and how many patients are needed for clinical trials?
What Is the “Best Pharmaceuticals For Children Act” (BPCA)?Federal law and U.S. Food and Drug Administration (FDA) regulations require that drugs be tested for safety and effectiveness in specific populations, at specific dosages, and for specific time periods before being approved for use.
In 1997, Congress created an incentive that encouraged drug developers to test medications in children if the drugs would be prescribed for children. The resulting trials confirmed that pediatric dosing recommendations had often been little more than “guesstimates.”
This incentive was extended as part of the Best Pharmaceuticals for Children Act (BPCA) in 2002 and again in 2007. Related legislation, the Pediatric Research Equity Act (PREA), was passed in 2003 and reauthorized in 2007. Together, BPCA and PREA have helped to spur increased research and development into safe drugs for children. BPCA is scheduled to end under “sunset” rules in late 2012, unless extended again or made permanent by Congress.
Today, the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) is the lead NIH agency helping to direct the BPCA pediatric clinical research trials on developing safe and effective children’s drugs and some medical devices at the NIH.
These clinical trials include 16 broad disease categories, such as heart diseases, cancer, infectious diseases, respiratory diseases, newborn and premature babies, and medical devices. The BPCA provides ways for studying patented and off-patent (generic) drugs.
BPCA: To Find Out More
- BPCA home website at NICHDhttp://bpca.nichd.nih.gov/
- FDA—Best Pharmaceuticals for Children Act (BPCA)http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/ucm148011.htm
- FDA Pediatric Drug Developmenthttp://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm049867.htm