Health technology assessment of paediatric medicines: European landscape, challenges and opportunities inside the conect4children project

Abstract

The medicine development process is complex and requires time and effort to ensure safety, efficacy and quality. In paediatrics, this process is even more challenging, as it involves a subgroup of the population that already faces a considerable gap in the clinical evaluation of medicines and devices compared to the adult population. Moreover, access to therapies is heavily influenced by national health technology assessment (HTA) recommendations, which often form the basis for pricing and reimbursement decisions that affect the effective availability of treatments within the national health systems. Yet performing an HTA to assess the relative effectiveness and cost-effectiveness of a new children's treatment has several nontrivial implications, creating a critical issue for the paediatric population. In addition, the advent of innovative health technologies for children emphasises the need to empower the role of HTAs in paediatrics.This article aims at describing the most relevant elements of the drug development process in the paediatric field by focusing on the HTA assessment. Particular attention will be paid to the factors that influence market access for new paediatric medicines and patients' access to treatment. The paper will also highlight some central methodological challenges in conducting HTA in the paediatric field. Finally, the article will provide insight into how initiatives, such as conect4children, may subsequently reinforce HTA awareness in the paediatric community and strengthen collaborations through network mechanisms.

Translating Precision Health for Pediatrics: A Scoping Review

“…Another prominent theme was the notion of increased phenotypic heterogeneity in children compared to adults; namely, children have natural genetic variations that often change throughout their development, impacting gene expression and possibly gene function [18][19][20][21][22][23][24][25][26][27][28][29][30][31][32][33][34]. Moreover, these differences may not only exist between children and adults, but also across the developmental trajectory between infants, children, and adolescents [19,21,22,35]. Specifically, during rapid child-hood development, there are age-related differences in drug-metabolizing enzymes and drug receptors.…”

Innovative Methodologies in paediatric drug development: a conect4children (c4c) special issue

“…The effectiveness and cost effectiveness of a drug is determined in order to support evidencebased decision making of both policy makers and healthcare professionals and an important use of HTA are decisions related to its reimbursement. Moretti et al, describe in their paper, paediatric specific aspects related to value of medicines in the context of the regulations supporting drug development 7 . Moreover, they describe challenges related to HTA evaluations for paediatric medicines, including lack of long-term data, small study populations, but also differences in economics of treating children, including impact on families, society and the different weight quality-adjusted life year may have in the evaluation of medicines for children.…”

Access to novel drugs and therapeutics for children and youth: Eliciting citizens' values to inform public funding decisions

“…[15][16][17][18][19][20][21][22] The third constraint remains largely unaddressed and stems from a more fundamental problem: the lack of HTA approaches that attend to the unique dimensions of child health and illness. 12,[23][24][25][26] HTA frameworks set out the principles and methods for systematic weighing of clinical, economic, organizational and patient-related impacts of novel interventions to ascertain value increments compared to existing alternatives, with the extra charge of assessing contextual equity, sustainability and health system implementation impacts. In the context of precision health interventions, evaluations must weigh uncertainties related to limited data on safety and efficacy.…”