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Table 1 Problems and potential solutions in relation to drug policy for children in Canada

From: ‘The problem is small enough, the problem is big enough’: a qualitative study of health technology assessment and public policy on drug funding decisions for children

DomainSample ProblemPotential Solutions
HTA Paradigms
• Manufacturer-driven process, minimal incentive for paediatric submissions
Transition from ‘push’ to ‘pull’ system for technology assembly:
• National priority-setting framework
• Resources for public sector-initiated HTA submissions
• Patient/public engagement on social values to guide priority-setting
 AssessmentEvidence appraisal:
• Structural barriers to RCT-level evidence in paediatrics
Innovative trial design and evidence appraisal:
• Basket trials, n-of-1 trials
• Real-world evidence and performance-based funding mechanisms
• Child health expertise in HTA bodies
Economic evaluation:
• Weak incorporation of child-specific considerations (developmental trajectory, preference elicitation) in pharmacoeconomic models
Advancement of science on child health economic evaluation:
• Child-centred preference elicitation, family utility generation
• Research on public preferences for resource allocation to children, including inquiry into life-course QALY weights
 Integration• Poor integration across phases and sectors involved in drug regulation and reimbursement• Adaptive pathways approach to drug development, market access, and iterative evidence appraisal
• Ring-fenced funding for paediatric drugs and health technologies
• Engagement with child health experts throughout drug life-cycle
Sociopolitical Context
 Markets• Weak industry incentives for paediatric drug development and licensing• Federal regulatory mechanisms to incentivize/compel development and submission of paediatric clinical data
• Public funding to subsidize novel drug trials in children
 Governance• Lack of formal paediatric indications, widespread off-label prescribing
• Vertical and horizontal system fragmentation
• Dedicated paediatric expertise and resources within federal regulator and HTA bodies
• Improved system integration along pharmaceutical value chain
• National framework for drug regulation and funding for children to drive policy harmonization
 Politics• Weak political voice for children
• Politically charged funding environment
• Enhanced receptors for child and family perspectives in drug regulation and funding decisions
• Child-specific HTA framework to drive transparency and legitimacy in paediatric technology assessments, and mitigate potential for politically motivated drug funding decisions