On January 11, 2022, the Patented Medicine Prices Review Board (PMPRB) released a report entitled Expensive Drugs for Rare Disorders: Canadian Trends and International Comparisons, 2011-2020 as part of the National Drug System initiative. Prescription Drug Utilization Information (NPDUIS).
Expensive drugs for rare diseases (EDRD) are defined in the report as drugs with at least one indication designated as orphan by the United States Food and Drug Administration (FDA) or the European Medicines Agency (EMA), and whose estimated treatment costs exceed $100,000 per year. for non-oncology drugs or $7,500 per 28 days for oncology drugs.
The report provides an overview of the EDRD market, including a comparison between Canada and PMPRB comparator countries. Notable findings include:
- By the end of 2020, a total of 104 EDRDs had been approved in Canada. Oncology drugs represent 54% of EDRDs approved in Canada.
- The total number of EDRDs more than doubled between 2011 and 2015, and doubled again between 2016 and 2020. The majority of EDRDs currently on the market were approved after 2015.
- Most EDRDs received a refund recommendation from CADTH with a discounted price as a condition.
- Between 2011 and 2020, the compound annual growth rate of EDRD sales in Canada was 31.6%.
- EDRD sales in 2020 accounted for more than one-tenth of the total Canadian pharmaceutical market. Almost three-quarters of EDRD sales in Canada in 2020 can be attributed to just 10 drugs, the majority of which are used for oncology indications.
- Biologics account for nearly half of all Canadian EDRD sales. The sales shares of organic and non-organic EDRD have remained relatively constant over the years.
- Current Canadian prices for patented DRDUs are comparable to those in international markets. However, the results do not reflect the confidential discounts negotiated in each country.
- EDRDs represent an important segment of recent international approvals. New EDRDs are launched in Canada at a similar pace to comparator countries.
- Orphan drugs represent a substantial part of the global development pipeline. This is particularly true in phase III and pre-registration, where a third of drugs have an orphan designation.