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Children's Mercy Physician Leads Effort to Update American Academy of Pediatrics Off-Label Pharmaceuticals Policy

Policy statement suggests ways to improve drug safety and efficacy in children, aligning with mission of Children's Mercy to improve patient outcomes.

With less than half of medications including specific labeling for children, Kathleen Neville, MD, MS, a physician at Children's Mercy Hospital, recently led an American Academy of Pediatrics (AAP) committee in updating the policy with new recommendations guiding the off-label use of drugs in pediatric patients. The policy statement, "Off-Label Use of Drugs in Children," was published online today in the March 2014 issue of Pediatrics. 

Dr. Neville is chair of the AAP Committee on Drugs and Director of Experimental Therapeutics in Pediatric Cancer at Children's Mercy. The AAP statement offers guidance to physicians using drugs off-label in the treatment of children, particularly in special populations, including preterm infants and newborns, and those with chronic or rare diseases.

The policy statement updates guidance from nearly a decade ago that resulted in more than 500 labeling changes, including expanded labeling with pediatric information. 

"Our goal is to provide children with the best care possible, and scientific evidence remains the best way to make treatment decisions," said Dr. Neville. "There's still much work to be done. Ultimately, all drugs used to treat children should have sufficient age-appropriate evidence to support updated labeling."

Experimental Therapeutics at Children's Mercy
Children's Mercy has the largest and most productive pediatric clinical pharmacology program in North America. The program pairs clinicians who identify clinically significant medication-related problems with basic science faculty members who have the expertise necessary to solve them.

Under the direction of Dr. Neville, the Experimental Therapeutics in Pediatric Cancer Program is quickly gaining recognition as a regional referral center, giving children with recurrent or refractory cancer a local option to pursue experimental treatment with early-phase anti-cancer drugs. Neville has had extensive training in experimental therapeutics for children with cancer, and is leading Phase I research efforts to develop new agents for the treatment of pediatric cancer and supportive care for patients.

In collaboration with the Institute for Advancing Medical Innovation (AMI) and the National Institutes of Health (NIH), the team has begun screening compounds, identifying several that appear promising as potential drug development candidates. The program is currently involved in more than 15 Phase I and Phase II trials.

About Children's Mercy
Children's Mercy, located in Kansas City, Mo., is one of the nation's top pediatric medical centers. The 354-bed hospital provides care for children from birth through the age of 21, and has been ranked by U.S. News & World Report as one of "America's Best Children's Hospitals." For the third time in a row, Children's Mercy has achieved Magnet nursing designation, awarded to fewer than seven percent of all hospitals nationally, for excellence in quality care. Its faculty of 600 pediatricians and researchers across more than 40 subspecialties are actively involved in clinical care, pediatric research, and educating the next generation of pediatric subspecialists.