On January 11, 2022, the Patented Medicine Prices Review Board (PMPRB) released a report entitled Expensive Drugs for Rare Diseases: Canadian Trends and International Comparisons, 2011-2020 as part of the National Prescription Drug Utilization Information System (NPDUIS) initiative.
Expensive drugs for rare diseases (EDRDs) are defined in the report as medicines with at least one orphan designated indication through either the US Food and Drug Administration (FDA) or European Medicines Agency (EMA), and with estimated treatment costs exceeding $100,000 per year for non-oncology medicines or $7,500 per 28 days for oncology medicines.
The report provides an overview of the market for EDRDs, including a comparison between Canada and PMPRB comparator countries. Noteworthy findings include:
- As of the end of 2020, a total of 104 EDRDs had received approval in Canada. Oncology medicines make up 54% of the EDRDs approved in Canada.
- The total number of EDRDs more than doubled from 2011 to 2015, and doubled again from 2016 to 2020. The majority of EDRDs currently on the market were approved after 2015.
- Most EDRDs have received a reimbursement recommendation from CADTH with a reduced price as a condition.
- Between 2011 and 2020, the compound annual growth rate of EDRD sales in Canada was 31.6%.
- Sales of EDRDs in 2020 constituted more than one-tenth of the total Canadian pharmaceutical market. Nearly three-quarters of the 2020 EDRD sales in Canada can be attributed to just 10 medicines, the majority of which are used for oncology indications.
- Biologics make up nearly half of all Canadian EDRD sales. The shares of biologic and non-biologic EDRD sales have remained relatively consistent over the years.
- Canadian list prices for patented EDRDs are comparable to international markets. However, the results do not reflect confidential rebates negotiated in each country.
- EDRDs account for a sizable segment of recent international approvals. New EDRDs are launching in Canada at a rate similar to comparator countries.
- Orphan medicines account for a substantial share of the global development pipeline. This is especially true in Phase III and pre-registration, where one-third of medicines have an orphan designation.
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