The Pediatric Trials Network (PTN) is studying the formulation, dosing, efficacy, and safety of drugs, as well as the development of medical devices, used in pediatric patients. Children are not simply “little adults” who respond to drugs according to scale. Rather, developing organs and changes in metabolism throughout infancy and childhood affect how drugs are processed by immature or maturing bodies. Thus, special care must be taken in tailoring the use of medicines in this unique and vulnerable population.
Unfortunately, only a small percentage of drugs and devices approved by the Food and Drug Administration are actually labeled for pediatric use. Pediatricians, consequently, are often forced to prescribe medical therapies “off-label,” or according to their best guess based on adult studies.
To fill this troubling information gap, the PTN is conducting trials primarily with off-patent 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 are not required and have no incentive to test them in younger age groups.
Data collected from PTN trials help regulators to revise drug labels for safer and more effective use in infants and children. See a complete list of NIH-funded pediatric labeling changes.
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