Surfactant Administration in Preterm Infants: Drug Development Opportunities

Abstract

Objective: To evaluate how frequently surfactant is used off-label in preterm infants. Study design:We conducted a retrospective cohort analysis of prospectively collected administrative data between 2005 and 2015 from 348 neonatal intensive care units in the United States. We quantified off-label administration of poractant alfa, calfactant, or beractant in inborn infants <37 weeks gestational age. Off-label surfactant administration was defined according to the Food and Drug Administration (FDA) label.Results: Of 110,822 preterm infants who received surfactant, 68,226 (62%) received surfactant off-label. The majority of infants who received surfactant off-label had a higher birth weight than label (40,716 [37%]), had a later gestational age than label (35,191 [32%]), or were intubated and administered surfactant followed by immediate extubation (INSURE) (32,310 [29%]). Poractant alfa was administered via INSURE more frequently than beractant or calfactant (16,688 [38%]; 7137 [20%]; 8485 [27%], respectively). An increasing number of infants received surfactant via INSURE from 2005 to 2015 (1697 [19%]; 3368 [36%]). Conclusions:The majority of surfactant given to preterm infants is administered off-label. The uptrend in administration via INSURE coincides with increased supporting evidence. The gap between FDA labelling and current clinic practice exemplifies an opportunity for label expansion, which may require additional prospective or retrospective safety and/or effectiveness data for infants with later gestational age and higher birth weight.Surfactant is one of the most frequently used medications in neonatal intensive care units (NICUs). 1 Beractant, calfactant, and poractant alfa, animal derived surfactants, are labeled

Outcomes of outborn extremely preterm neonates admitted to a NICU with respiratory distress

“…The Pediatrix Clinical Data Warehouse (CDW), a large, multicentre, deidentified data set20 that has been used by the National Institutes of Health,21 the US Food and Drug Administration,22 the Eunice Kennedy Shriver National Institute of Child Health and Human Development Pediatric Trials Network,22–24 and independent investigators,25–28 served as the data source for this retrospective cohort study.…”

Association of Adherence to Surfactant Best Practice Uses With Clinical Outcomes Among Neonates in Sweden

“…Off-label use and omission of use are also important areas that have been poorly defined in terms of size of the problem and associations with outcome. 18 …”

Examining Variations in Surfactant Administration (ENVISION): A Neonatology Insights Pilot Project

“…The INSURE strategy is being utilized worldwide on the basis of increased supportive evidence [ 2 , 21 ]. Our findings show that 83% of HCPs utilize the INSURE technique, however, we did not determine the regularity with which it was used or identify a specific sub-population of infants in which it is utilized.…”