The Canadian access and reimbursement system for medicines includes a series of reviews, recommendations and decisions that aim to allocate scarce public resources to therapies that offer the most benefit.  Generally, with clear evidence of safety, efficacy, and quality, you can expect that the therapy will be reimbursed, and patients will have access.  This is especially the case when there is unmet medical need, and an absence of other approved therapies.

Few therapies are evaluated and approved for use with children, including to treat pediatric cancers, and most drugs used to treat children are prescribed off-label. Children with cancer did not benefit from the explosion of new drugs approved in the 1990s and 2000s, gaining access mostly through clinical trials. It has taken many years and much effort to conduct follow-on research and demonstrate how these drugs work in children, often led by academic clinicians.

We are finally seeing an increase in approvals of medicines for cancer in children, often for drugs that have been on the market for many years.  For example, the United States has made significant strides in expanding the number of pediatric cancer drug approvals through the FDA, demonstrating the potential of a more responsive framework. It is important to close gaps to ensure that children in Canada are not missing timely access to effective and potentially life-saving therapies that are already available elsewhere.

This project aims to systematically assess access to therapies for pediatric oncology patients across Canada that have achieved regulatory approval for use in children. The United States has the longest list of approved pediatric cancer indications – this study will review how many are approved in Canada, the European Union, and Australia. The study will also assess, for the first time, the public reimbursement status of each of the approved indications across Canada.

The project will also review and describe how each of the approved indications has been considered by the Canadian drug review and assessment “system”.

This work will be conducted in two phases:

1. Create a comprehensive dataset of pediatric cancer drugs approved since 2000 by the US FDA, Health Canada, European Medicines Agency, and Australia’s Therapeutic Goods Administration, and the public reimbursement status of each Canadian approved indication.
2. Describe how Canadian regulatory bodies like Health Canada, the Canadian Agency for Drugs and Technologies in Health (CADTH), and the pan-Canadian Pharmaceutical Alliance (pCPA) approach pediatric submissions.

Project deliverables will include a position paper, peer-reviewed manuscript, and actionable policy recommendations to improve pediatric oncology drug access in Canada. Ultimately, this project will be transformative for Canadian children with cancer and their families, helping to bridge access gaps and advocate for regulatory changes that better reflect the unique needs of pediatric oncology patients.

For more information, please contact Tricia Schneider.