The Importance of Access to Medications for Pediatric Patients: Why Children Need Patient Advocacy the Most – A Canadian Perspective

by Cheryl Petruk, MBA

Access to timely, appropriate, and affordable medication is critical for all Canadians—but when it comes to children, the stakes are even higher. Pediatric patients are not simply “small adults”; they have unique physiological, developmental, and psychosocial needs that must be taken into account when delivering healthcare

Yet in Canada, significant barriers persist that impede access to medications for children, ranging from limited pediatric-specific drug approvals to gaps in coverage and off-label prescribing. In this context, patient advocacy becomes not only important but essential. Advocates play a key role in ensuring that children’s voices, often silent or underrepresented, are heard at all levels of the healthcare system.

This article explores the importance of medication access for pediatric patients, the unique challenges they face in Canada, and why patient advocacy must be at the forefront of change

Pediatric Pharmacotherapy: A Critical Need

Children with chronic conditions, rare diseases, cancer, or acute illnesses depend on a range of medications to manage symptoms, improve quality of life, or even survive.

Examples include insulin for Type 1 diabetes, chemotherapy for pediatric cancers, biologics for juvenile arthritis, and antiepileptics for seizure disorders. For many families, navigating access to these treatments is a complex, stressful journey.

While Canada prides itself on a universal healthcare system, prescription drug coverage is not universally insured. Instead, access to medications is determined by a patchwork of provincial drug plans, private insurance, and out-of-pocket spending. For pediatric patients, whose care often involves specialized treatments not always covered by public formularies, the result can be delayed treatment, financial burden, or in some cases, no treatment at all.

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UniqueChallengesforChildreninCanada

LimitedDrugApprovalsforPediatricUse

One of the most pressing concerns is that many medications used in children have not been formally approved for pediatric populations. According to the Canadian Pediatric Society, over 50% of medications prescribed to children are done so “off-label”—meaning they were not specifically tested or approved for that age group. This is due in part to a lack of pediatric clinical trials, regulatory hurdles, and insufficient incentives for pharmaceutical companies to pursue pediatricindicationsinCanada.

This presents risks in terms of efficacy and safety, as children metabolize medications differently than adults. Without robust pediatric data, healthcare providers must make best guessesondosage,frequency,andsideeffects,potentiallyjeopardizingoutcomes.

DisparitiesinCoverageandAccess

Access to medications varies widely across provinces and territories. While some provinces have established special programs for children (such as OHIP+ in Ontario, which covers most prescription drugs for those under 25 without private insurance), others rely heavily on meanstested public plans or require co-payments. For families with low income or those living in rural or Indigenous communities, the combination of costs, travel, and administrative hurdles can be overwhelming.

Rare Disease and Orphan Drug Access

Children represent a disproportionate share of patients with rare diseases, many of which have no Health Canada-approved treatments. Even when treatments exist, Canada’s review and reimbursement system is slow and often restrictive. For instance, while the Canadian Agency for Drugs and Technologies in Health (CADTH) conducts health technology assessments (HTAs) for public drug plans, their criteria often exclude the real-world needs and quality-of-life improvements vital to pediatric patients with rare or life-threatening diseases. This results in significant delays or outright denials in funding, despite international approvals.

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The Role of Pediatric Patient Advocacy in Canada

Advocacy organizations, caregivers, and health professionals are vital players in bridging the gap between pediatric patients and the medications they need. In a system where children often cannot advocate for themselves, adult allies must champion their voices.

1. Amplifying the Pediatric Patient Voice

Children are dependent on others to make decisions on their behalf—parents, caregivers, doctors, and policymakers. Advocacy ensures that these decisions reflect the best interests of the child, informed by lived experience, clinical evidence, and ethical considerations. Groups such as SickKids, Children’s Healthcare Canada, and various diseasespecific foundations have become powerful advocates in raising awareness of pediatric health inequities.

2. Driving Policy Reform and Coverage Expansion

Advocates push for improvements in federal and provincial policy, such as Canada’s emerging National Pharmacare strategy. A truly universal plan must account for pediatric populations, ensuring access to ageappropriate medications, eliminating co-pays for essential drugs, and prioritizing rapid review and funding of medications for children with rare diseases.

3. Fostering Pediatric Research and Trials

Advocacy organizations also play a crucial role in pushing for more pediatric-specific research. In Canada, this means advocating for funding to support pediatric clinical trials, ethical frameworks for child participation, and incentives for manufacturers to seek pediatric indications. For instance, initiatives like the Canadian Paediatric Clinical Trials Network are a step in the right direction, but they need sustained support and attention.

4. Real-World Impacts of Delayed Access

The consequences of delayed or denied access to medication can be devastating. Consider a child with a rare genetic disorder like spinal muscular atrophy (SMA), where early treatment with gene therapy or diseasemodifying drugs can significantly improve life expectancy and quality of life. Every day without treatment can mean irreversible damage. Families often turn to crowdfunding or advocacy campaigns to raise money or gain public attention—something no parent should be forced to do in a country with public healthcare.

Another example includes delays in approving medications like biologics for juvenile idiopathic arthritis Prolonged wait times can mean months of uncontrolled pain, missed school, and developmental delays Pediatric advocacy ensures that children are not treated as an afterthought in a system designed primarily for adults.