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

Gauvreau, Cindy L.; Wight, Lisa; Subasri, Mathushan; Palmer, Antonia; Hayeems, Robin Z.; Croker, Alysha; Abelson, Julia; Fraser, Brent; Bombard, Yvonne; Hepburn, Charlotte Moore; Wilson, Michael G.; Denburg, Avram

doi:10.1111/hex.13697

Cited by 4 publications

(1 citation statement)

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“…Thus, procedures and explicit criteria need to be developed to systematically incorporate the child's voice into value assessments [95]. Within health systems research, it is encouraging to see that efforts are underway to recognize the need to build an HTA framework that captures the distinct needs of children more comprehensively and incorporates the child's voice in these discussions [96].…”

Section: Discussionmentioning

confidence: 99%

Translating Precision Health for Pediatrics: A Scoping Review

et al. 2023

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Precision health aims to personalize treatment and prevention strategies based on individual genetic differences. While it has significantly improved healthcare for specific patient groups, broader translation faces challenges with evidence development, evidence appraisal, and implementation. These challenges are compounded in child health as existing methods fail to incorporate the physiology and socio-biology unique to childhood. This scoping review synthesizes the existing literature on evidence development, appraisal, prioritization, and implementation of precision child health. PubMed, Scopus, Web of Science, and Embase were searched. The included articles were related to pediatrics, precision health, and the translational pathway. Articles were excluded if they were too narrow in scope. In total, 74 articles identified challenges and solutions for putting pediatric precision health interventions into practice. The literature reinforced the unique attributes of children and their implications for study design and identified major themes for the value assessment of precision health interventions for children, including clinical benefit, cost-effectiveness, stakeholder values and preferences, and ethics and equity. Tackling these identified challenges will require developing international data networks and guidelines, re-thinking methods for value assessment, and broadening stakeholder support for the effective implementation of precision health within healthcare organizations. This research was funded by the SickKids Precision Child Health Catalyst Grant.

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Section: Discussionmentioning

confidence: 99%