(BSPOT30) Pediatric Utilization of New Molecular Entities (NMEs): A Summary of Early Post-marketing Uptake in the Sentinel Distributed Database

Background: A wide range of NMEs are approved by the US Food and Drug Administration (FDA) each year. Many of these products have no information regarding appropriate use in the pediatric population at the time of approval. Currently there is a gap in our understanding of early post-marketing utilization of newly approved NMEs in pediatric patients.

Objectives: To characterize the uptake of NMEs approved from 2017 to 2021 among users 0-17 years of age in the Sentinel Distributed Database (SDD).

Methods: We conducted a retrospective cohort study using claims data to determine uptake of NMEs approved between 2017 to 2021 across the Sentinel distributed network of data partners. The cohort was defined from January of the approval year. Patients were observed from index date, defined as the date of first qualified dispensing or administration of the NME, to the end of data availability. Uptake of NMEs were identified by National Drug Code (NDCs) and Healthcare Common Procedure Codes (HCPCS) available on prescription or medical claims. Each NME was reviewed for the proportion of users < 18 years of age. We conducted a descriptive analysis of pediatric utilization stratified among NMEs with 10,000 to 50,000 users and >50,000 users.

Results: There were 260 NMEs approved during the study period. Of these, evidence of uptake in the pediatric population was observed in 61.2% (159) of all NMEs. Annual utilization for each NME in patients < 18 years of age was 77.1% (37 of the 48 NMEs approved in 2017), 80% (48 of the 60 NMEs approved in 2018), 56.3% (27 of the 48 NMEs approved in 2019), 45.3% (24 of the 53 NMEs approved in 2020) and 45.1% (23 of the 51 NMEs approved in 2021). Among NMEs with >50,000 users (N=16), notable pediatric utilization was observed with baloxavir (17.3%; 19,064 /110,106 patients) and dupilumab (8.5%; 7,038/83,061 patients). In medications with 10,000 to 50,000 overall users (N=32), pediatric utilization was observed with cannabidiol (52.8%; 11,082/ 20,012 patients), sarecycline (44.1%; 6,312/14,322 patients) and trifarotene (39.5%; 8,457/21,437 patients). Four NMEs were exclusively used by pediatric patients: lonapegsomatropin (n=211), vosoritide (n=36), cerliponase (n=18) and naxitamab (n=14). In NMEs with ≥10,000 users and evidence of pediatric utilization, six (15.4%) of the 39 medications had information regarding safety and efficacy in the pediatric population prior to approval.

Conclusions: Early utilization in pediatric patients was observed in more than half of NMEs approved from 2017-2021. For these medications, post-marketing studies using real-world evidence specific to pediatric patients are needed to generate additional age-specific safety and efficacy information.