A scoping review of challenges in pediatric health technology assessments with a focus on pharmaceutical interventions.

objectivesThis scoping review aims to synthesize the literature on pediatric health technology assessments (HTAs) and map out the challenges of assessing new technologies for use in children, with a particular focus on pharmaceutical interventions.

methodsConducted in accordance with the Joanna Briggs Institute Methodology, this scoping review addressed HTAs in the pediatric domain through searches of PubMed, Embase, Web of Science Core Collection, and EconLit through 22 January 2024, as well as the gray literature. Sources were excluded if they (i) were a clinical trial investigating a specific technology or an HTA of that technology, (ii) did not address the challenges of HTAs, or (iii) had no relevance to pediatrics. Two authors performed screening and data extraction independently and in duplicate.

resultsOne hundred and three reports were included. Of these, sixty were full journal articles, twenty-three were conference abstracts, and twenty were guidelines, reports, and other documents. Two important themes emerged from this work. The first was the unique position of children within society and the resulting difficulty of incorporating them within a population-wide HTA system. The second was the uncertainty that characterized pediatric HTAs due to data constraints and either a lack of guidance by HTA bodies or variations in guidance between bodies.

conclusionsMany factors inherent to children, including the heterogeneity of pediatric disease populations, long-term outcomes, and children's distinct social positions, render conducting pediatric HTAs challenging. Innovative approaches are required to address these challenges and respond to the needs of pediatric populations.