By: Rachel Greenberg, MD, MHS and Danny Benjamin, MD, PhD, MPH | For too long, parents and pediatricians have faced a troubling dilemma: medications commonly prescribed to children are often not studied in pediatric populations. This leaves many questions unanswered about the safety, effectiveness, and dosing for the youngest of patients. In 2010, the Pediatric Trials Network (PTN) stepped in to change that. By conducting extensive research, for over a decade, on how drugs work in children’s bodies, PTN is filling a critical void and transforming pediatric care. The PTN is accomplishing this by conducting trials primarily with off-patent (or generic) drugs that are lacking data in pediatric populations. Though these drugs may be commonly used, because the exclusive right to market them has expired, companies almost never test them in children.
Prior to 2022, we did not know the correct dosing for >90% of medicines used in children; now that is dramatically different because of the work of PTN. Data collected from PTN trials help regulators to revise drug labels for safer and more effective use in infants and children. These efforts have resulted in 23 label changes by the Food and Drug Administration, improving health outcomes for children.
However, PTN’s efforts go beyond changing labels; today, we are studying the safe dosing of nearly 100 medicines used in children; and these studies have immediate impact. The CUDDLE study has significantly improved drug safety data for breastfeeding mothers. Historically, there was limited information on the effects of drugs on breast milk, causing uncertainty for new mothers. PTN is now researching 30 common medications used by breastfeeding women to provide crucial safety information.
For critically ill children who need specialized care, PTN’s research impacts treatments like Extracorporeal Membrane Oxygenation (ECMO) for severe heart and lung conditions in neonates and children. PTN optimizes drug dosing for these vulnerable patients, ensuring safe and effective treatments.
We have received feedback from physicians nationwide, highlighting how PTN’s work has significantly informed their practices and saved patients’ lives. In our own practice as physicians who provide clinical care to infants and children, we prescribe medications that have been studied by PTN every day. We have seen the antibiotic dosing that PTN has shown to be effective save lives of premature infants in the Neonatal Intensive Care Unit. It is because of PTN’s work that we are
now able to reassure new mothers who take medication for pain that only a minimal amount of the medication is passed to their babies in breast milk. These are just a few of the ways that clinicians like us are putting PTN’s work into real-life use.
Despite all that has been accomplished, the work needs to continue. Looking ahead, PTN plans to expand its efforts in enhancing pediatric health through ongoing research. Pediatric-specific drug research is important, particularly with new health challenges emerging. This research not only benefits families today but also contributes to public health by potentially reducing healthcare costs and improving outcomes for future generations. Increased funding would enable larger trials
to support clinicians’ critical decisions.
Parents, healthcare providers, and members of the public all play an important role in this initiative. Research shows that families often participate in advancing research to improve the care of other children with similar conditions or needs. Together, we can help ensure that children receive safe and effective treatments. To stay updated on developments within the Pediatric Trials Network, please subscribe to the newsletter at pediatrictrials.org.
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Rachel G. Greenberg, MD, MB, MHS is Chair of the Pediatric Trials Network Steering Committee and a neonatologist and Associate Professor of Pediatrics at Duke University Medical Center and Duke Clinical Research Institute.
Dr. Danny Benjamin is the Principal Investigator and Chair of the Pediatric Trials Network. Dr. Benjamin is Deputy Executive Director of the Duke Clinical Research Institute and has served as Principal Investigator for dozens of drug trials in children.
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