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New National Initiative to Rigorously Study Pediatric Dosing and Safety of Prescription Medicines

New National Initiative to Rigorously Study Pediatric Dosing and Safety of Prescription Medicines
New National Initiative to Rigorously Study Pediatric Dosing and Safety of Prescription Medicines


Duke Health News Duke Health News

A groundbreaking effort to reduce the risks and dangers faced everyday by millions of children who are prescribed medications that lack definitive data about pediatric dosing, efficacy and safety was created today through an initiative supported by the National Institutes of Health (NIH), totaling up to $95 million over the next seven years.

The initiative, a Pediatric Trials Network (PTN), will be led through a collaboration between the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), the Best Pharmaceuticals for Children Act (BPCA) Program, and the Duke Clinical Research Institute (DCRI), and will involve the participation of several of the country's preeminent pediatric medicine experts, hospitals and medical centers.

"It's staggering to consider that less than 10 percent of licensed therapeutics have been adequately studied in children, so much of pediatric drug use is based on an educated guess by a pediatrician using studies conducted in adults, who often absorb drugs differently or experience different side effects than children," said Daniel K. Benjamin, Jr., MD, PhD, professor of pediatrics at Duke and associate director of the DCRI, who will lead the PTN.

The PTN was conceived to create a scientific, technical, and administrative infrastructure for conducting pediatric clinical trials in an environment that provides optimal safety for the research participants and sophisticated expertise to determine the balance of benefits and risks of drug and device treatment of medical illnesses in children.

Under an agreement among the organizations, the network will conduct 16 trials spanning a variety of therapeutic areas, including cardiovascular diseases, cancer, infectious diseases, gastroenterology, respiratory diseases, neonatology, and medical devices. The drugs and devices have yet to be determined. The compounds studied will be those which have already been on the market long enough that their patents have expired, otherwise known as generic drugs.

A network steering committee will be established to work alongside the NICHD to oversee the network operations. Program management will be conducted by the DCRI and the leadership team will include investigators from Duke Children's Hospital, Children's Mercy Hospital in Kansas City, the University of California, San Diego, Children's National Medical Center in Washington D.C., The Children's Hospital of Philadelphia, Wake Forest University Baptist Medical Center in Winston-Salem, NC, and Carilion Clinic Children's Hospital in conjunction with the Virginia Tech Carilion School of Medicine.

"Pediatric dose formulation and therapeutic and diagnostic pediatric device development are urgent pubic health issues of critical importance," said Robert Harrington, MD, director of the Duke Clinical Research Institute. "This network brings together an outstanding multi-disciplinary team across the spectrum of translational research to greatly expand upon a currently functioning proof-of-concept network underway at the DCRI and its sister organization, the Duke Clinical Research Unit (DCRU)."

According to Benjamin, the first trial in this project will likely be conducted to determine optimal dosing of medications used to treat high blood pressure in children. Most of the trials will enroll between 100 to 200 children. Aspects of this network will include product formulation, drug distribution and device development. "In effect, each trial will become a 'mini' drug development program," Benjamin explained.

Another objective for this project is to use the data collected from the PTN to help inform pediatric drug labeling, and to provide regulators, pediatricians, and researchers with new information on medication response in children.

"The Pediatric Trials Network has the potential to correct a problem that continues to surprise people—we have inadequate information about the proper treatment of children, even for commonly used treatments that have been on the market for years," said Robert M. Califf, MD, director of the Duke Translational Medicine Institute (DTMI) and vice chancellor for clinical research at Duke University. "Considering Duke's established leadership in translational medicine and the involvement and support of many of the pediatricians and clinical pharmacologists active in clinical trials in the United States, this team composed of the nation's experts in therapeutics in children should provide information that will be critical to pediatricians and the children they treat."

The PTN will comprise six groups dedicated to:

  • clinical operations and program management,
  • managing clinical pharmacology design components of trials,
  • pharmacokinetic and pharmacodynamic simulations, analysis and modeling,
  • common safety reporting across trials and an ethics review of each study protocol,
  • overseeing collaboration in the multi-disciplinary development of devices for use in children, and
  • training the next generation of clinical pharmacologists and clinical trialists by partnering junior investigators with program leaders and senior investigators.

When appropriate, the research will apply the specialized capabilities and technologies available in the Duke Clinical Research Unit (DCRU), which is one of just a handful of state-of-the-art, hospital-based, early-phase research units in the country. Benjamin serves as the program director for pediatrics in the DCRU.

The Duke Clinical Research Institute is the world's largest academic clinical research organization, which is known for conducting groundbreaking trials ranging from pivotal studies for investigational medicines to landmark outcomes research.

The DCRI and DCRU are part of the Duke Translational Medicine Institute, which was established in October 2006 with a grant from the National Institutes of Health National Center for Research Resources (NCRR) Clinical Translational Science Award (CTSA). The mission of the DTMI is to expedite the translation of new scientific discoveries into clinical practice while promoting measurable improvements in community health, and making personalized medicine a reality.

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