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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


Sample Problem

Potential 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


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


• 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


• 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


• 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


• 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