P120: Assessing the Age-Appropriateness of Pediatric Medicines
Poster Presenter
Lauren Le
Student
Butler University United States
Objectives
To evaluate the age-appropriateness of oral pediatric medicines by assessing factors regarding patient acceptability, excipient safety, and dosing flexibility using publicly available regulatory data from the US Food and Drug Administration (FDA).
Method
Analyzed FDA drug approvals, focusing on pediatric labeling changes resulting from the Pediatric Research Equity Act (PREA) and the Best Pharmaceuticals for Children Act (BPCA). Pediatric formulations were classified as: (i) does not meet target, (ii) partially meets target, (iii) meets target.
Results
An adapted version of the World Health Organization’s (WHO) pediatric quality target product profile (pQTPP) tool was used to assess formulation appropriateness based on excipient safety, dosing flexibility, patient acceptability, and ease of administration.
As of December 2024, 218 oral formulations had pediatric labeling under BPCA and PREA. Before 2000, 24 oral formulations were FDA-approved for pediatric use. Approval rate remained similar up to 2009. A notable rise occurred between 2010 and 2014, with 34 approvals, and continued with 54 and 52 approvals from 2015 to 2019 and 2020 to 2024, respectively, thus reflecting ongoing efforts to enhance pediatric medicine development.
Traditional oral solid dosage forms, tablets and capsules, comprise most of the formulations (n = 126). Oral liquid forms, including solutions, suspensions, syrups, and reconstitutable tablets/capsules/powders, account for 60 formulations. Meanwhile, non-traditional oral solid dosage forms, such as orodispersible tablets, pellets, and granules, constitute 32 formulations.
Across all pediatric age groups, the majority of formulations (190/218) were partially compliant with the age-appropriateness target, 18 were compliant, and 10 were non-compliant. Additionally, 117 drugs were approved for pediatric use at the time of initial market entry while 101 gained pediatric approval through labeling changes, highlighting the impact of the BPCA and PREA.
Across all of the five-year periods, tablets have consistently been the most common dosage form for most pediatric age groups. However, the use of pellets and granules has steadily increased. From 2000 to 2004, there were only four pediatric-approved formulations in this category, but approvals have grown significantly, with 12 new formulations receiving approval in the past decade (2015–2024).
Conclusion
In recent years, significant progress has been made in the development of pediatric medicines. Many of these advancements have been particularly beneficial for school-aged children (6–12 years). However, despite improvements with orodispersible tablets and reconstitutable liquid dosage forms, challenges remain in developing equally flexible and acceptable formulations for neonates and infants. Only 23 formulations have been approved for this population, in contrast to 72 for school-aged children. Tablets and capsules have traditionally been compounded into liquids for pediatric use, but this practice carries risks such as dosing variability and stability concerns. The first FDA-approved tablet for suspension for pediatric use was introduced in 2012. In the past decade, 15 more have been approved, providing alternatives to extemporaneous compounding. Additionally, excipient safety assessments reveal that some liquid pediatric formulations on the market contain potentially harmful excipients. Among the 44 liquid pediatric formulations analyzed, 41% (n = 18) were found to contain sodium benzoate or benzoic acid, both of which are known to have potential allergic properties. Furthermore, 65% (n = 29) of these liquid formulations contained parabens, which are commonly used as preservatives. Of the 218 oral formulations analyzed, 101 were classified as anti-infectives (including antibiotics, antivirals, and antifungals) and 65 were related to the nervous system. However, there remains a significant gap in the availability of medications for pediatric patients, with fewer approvals in other therapeutic areas. This study underscores the ongoing need for innovation in pediatric drug development to ensure safe and effective treatment options for children. Greater emphasis on child-friendly formulations in regulatory pathways, along with increased collaboration between manufacturers, regulatory agencies, and healthcare providers, is essential to bridging these gaps.
