Despite federal legislation encouraging the study of products used in the pediatric population, very little of these studies has translated into meaningful information regarding neonates (JAMA Pediatr 2014; 168: 130-36, thanks to Ben Gold for this reference).
This publication reviewed studies submitted to the FDA between 1997-2010. The authors identified all drugs with pediatric studies that included neonates. Subsequently, the use of these drugs was examined in a oohort of neonates admitted to 290 neonatal intensive care units (NICU) (Pediatrix Data Warehouse) in the U.S. form 2005-2010.
- 28 drugs (in 41 studies) were examined in neonates. This led to 24 labeling changes.
- 11 of 24 neonatal labeling changes included an approval for use in neonates, including 4 for HIV and 3 for anesthesia.
- 13 of 24 labeling changes were the following: “safety and effectiveness have not been established.” These drugs included several reflux medications: esomeprazole, lansoprazole, pantoprazole, and ranitidine.
- In the Pediatrix database involving 446,335 hospitalized neonates, there were 399 different drugs identified that had been administered. Of the 28 studied drugs, the gastroesophageal reflux medicines were used most frequently. 13 of the 28 studied drugs were not used at all in the NICUs.
- Of the 11 drugs with a neonatal indication, 7 were never used in the Pediatrix neonatal population and the other 4 drugs were used infrequently.
- Neonates are a vulnerable and an understudied population
- Most of the exposure to drugs was off-label for neonates.
- Most often, off-label drugs were prescribed “despite studies indicating they were not effective…For example, ranitidine, lansoprazole, and inhaled nitric oxide (for the prevention of bronchpulmonary dysplasia) were the top 3 drugs used in neonates…none have FDA labelling for the indication studied because of lack of efficacy.”
- Furthermore, drugs like ranitidine and lansoprazole” are associated with serious adverse effects in neonates.” (Clin Perinatol 2012; 39: 99-109)
Related blog entries: