Upward trend in market entry of high-cost drugs for rare diseases shows no sign of abating

From: Patented Medicine Prices Review Board Canada

Findings from the latest edition of the PMPRB’s Meds Entry Watch report show a continued rise in the number of new specialized treatments entering international markets. Coupled with high treatment costs, specialty orphan and cancer medicines now account for a sizable share of the new drug landscape.

This fourth installment in the Meds Entry Watch report series, published by the PMPRB under the National Prescription Drug Utilization Information System (NPDUIS) initiative, looks at the medicines that received first-time market approval through the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), and/or Health Canada in 2017 and 2018.

The number of new medicines that were approved annually in recent years is higher than the longer term average. Although fewer new medicines were authorized for market in Canada than in the US and Europe in 2017, those that were approved for sale here account for close to 90% of all new medicine sales across the OECD by the end of 2018, which suggests that the higher-impact new medicines are available to Canadian patients.

The increasingly high cost of new medicines poses access and affordability challenges and is the driving force behind a host of recent and pending reforms in pricing and reimbursement policy, both domestically and internationally. In Canada, the PMPRB protects consumers by regulating the ceiling prices of patented medicines sold here and keeps them informed on trends in the pharmaceutical market through its reporting function.

Quick facts

  • In 2018, 51 new medicines received first-time approval through the FDA, the EMA and/or Health Canada, almost equal to the 52 approved in 2017. In Canada, 18 and 27 new medicines got their approval for 2018 and 2017 respectively, trailing behind the United States and Europe.

  • Nearly two thirds of the new medicines approved internationally in 2017 came with treatment costs over $10,000 per year, or $5,000 per 28-day cycle for oncology. Fifteen of the new approvals were orphan-designated medicines with annual treatment costs exceeding $100,000 or 28-day cycle costs over $7,500.

  • From 2009 to 2017, slightly less than half of all new medicines were authorized for market in Canada, placing it in line with the OECD median but behind most of the PMPRB7 comparable countries. Despite this, Canada’s share of new medicine sales was fourth highest at 94%, indicating that the higher-selling medicines were approved.

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