A scoping review of challenges in pediatric health technology assessments with a focus on pharmaceutical
Nora Hutchinson1,2, Lauren Otterman1, Paul A Bain3
1Multi-Regional Clinical Trials Center of Brigham and Women's Hospital and Harvard, Boston, MA, USA.
Insights
Conducting pediatric health technology assessments (HTAs) is challenging due to children's unique social positions and data limitations. Innovative methods are needed to address these complexities in pediatric HTA.
Area of Science:
- Health Technology Assessment (HTA)
- Pediatric Health
- Pharmaceutical Interventions
Background:
- Pediatric health technology assessments (HTAs) face unique challenges.
- Assessing new technologies for children, especially pharmaceuticals, requires specific considerations.
- Existing HTA frameworks may not adequately address pediatric populations.
Purpose of the Study:
- To synthesize literature on pediatric health technology assessments (HTAs).
- To identify and map the challenges in assessing new technologies for pediatric use.
- To focus specifically on pharmaceutical interventions within pediatric HTAs.
Main Methods:
- Scoping review conducted following Joanna Briggs Institute Methodology.
- Searches of multiple databases (PubMed, Embase, Web of Science, EconLit) and gray literature.
- Inclusion criteria focused on HTA challenges in pediatrics, excluding specific technology trials.
Main Results:
- 103 reports were included, comprising journal articles, abstracts, and guidelines.
- Key challenges include children's societal position and integrating them into population-wide HTA systems.
- Uncertainty in pediatric HTAs stems from data constraints and inconsistent guidance from HTA bodies.
Conclusions:
- Heterogeneity in pediatric populations, long-term outcomes, and social factors complicate pediatric HTAs.
- Current approaches are insufficient for addressing the unique needs of pediatric populations.
- Innovative strategies are essential for effective pediatric health technology assessment.
Objectives:
This 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.
Methods:
Conducted 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.
Results:
One 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.
Conclusions:
Many 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.
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