Table 1.

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

Domain	Sample Problem	Potential Solutions
HTA Paradigms
Assembly	Priority-setting: • 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
Assessment	Evidence 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