Federal Policies and Public Reimbursement of Pediatric Medicines in Canada

Key Messages

• Most medicines are initially researched and developed primarily for adult indications.
• There are several challenges with researching and developing drugs for pediatric populations including but not limited to low disease prevalence, small patient populations, challenges with conducting long-term studies, short supply of pediatric investigators, and pediatric-specific physiological and behavioural considerations (appropriate facilities, protocols, and so forth).
• Therefore, most pediatric prescriptions have not received regulatory approval by Health Canada (i.e., off label) for pediatric populations nor are they available in pediatric-appropriate routes of administration or doses.
• In Canada, reimbursement of drugs for pediatric indications occurs through private coverage or a public drug plan through universal coverage programs or programs that exist based on need such as income, high-cost drugs, or specific diseases. Drugs not listed on public drug plan formularies may be funded through a restricted benefit (reimbursement limited by specific clinical criteria or to a defined patient subgroup).
• Drug reimbursement decisions, informed by health technology assessments, are also impacted by similar gaps in pediatric-specific clinical data.
• This Environmental Scan summarizes various initiatives by Health Canada that aim to improve access to pediatric medicines. In addition, policies of public drug programs regarding access to and reimbursement of pediatric medicines and formulations, including compounded and off-label medicine, are also summarized.