Canada’s regulatory approach to drugs for rare diseases: our role in bringing drugs to patients

From Health Canada

Our role in bringing drugs to patients

Canada’s health care system is complex and involves the participation of multiple stakeholders, who all have distinct roles in the planning and delivery of healthcare services. Health Canada’s decision-making role in approving a drug for marketing is distinct from the roles of those who make decisions about price-setting and public drug plan reimbursements.

Health Canada’s Health Products and Food Branch (HPFB) provides regulatory oversight for the manufacturing and sale of drugs in Canada, including drugs for rare diseases. Their goal is to protect Canadians from unsafe drugs, maximize treatment benefits and minimize associated risks.  In regulating pharmaceuticals, Health Canada is responsible for assessing the safety, efficacy and quality of drugs before approving them for sale in Canada.

Once Health Canada authorizes an orphan drug for sale, funding is not guaranteed through drug benefit programs.  The provincial and territorial body responsible for drug plans determines which drugs will be reimbursed, under what conditions for their eligible populations. Federal, provincial, territorial public plans and private plans reimburse drugs in Canada.

Before listing a drug, each public drug plan and cancer agency considers the recommendations of health technology assessment bodies, such as the Canadian Agency for Drugs and Technologies in Health and Institut national d’excellence en santé et services sociaux for Québec, and other factors, such as program mandate, jurisdictional priorities and budget impact before making its drug coverage decision. It is up to each jurisdiction to make its final formulary listing and reimbursement decisions. As a result, drug listing may vary between provinces.

The federal government also regulates the price of patented drugs in Canada through the Patented Medicine Prices Review Board to ensure that prices are not excessive, while the regulation of generic drug prices is a provincial responsibility. The pan-Canadian Pharmaceutical Alliance (pCPA) leverages the collective purchasing power of public payers to negotiate discounts for their respective drug plans.

Current work is ongoing to assess and develop a National Formulary through the Advisory Council on the Implementation of National Pharmacare.