Expensive Rare Disease Drugs Reflect Growing Portion of New Approvals in Canada

Spending on expensive drugs for rare diseases—drugs that cost more than $78,500 (CAN $100,000)—reached $3.1 billion in 2020, more than 11 times the total in 2011, according to a new report from Canada’s Patented Medicine Prices Review Board.

The Patented Medicine Review Board is an independent quasi-judicial body established by the Canadian Parliament in 1987. It is charged with ensuring drug prices are not excessive, with reporting on pharmaceutical trends of all medicines, and on research and development spending by drug developers.

The report said there has been a rapid rise in new approvals of orphan drugs to treat rare diseases over the past decade. The highest-cost orphan medicines—those with treatment costs in excess of $78,500 (CAN$100,000) per year, or $5,900 (CAN $7,500) per 28-day cycle for oncology—represent an increasingly important segment of the market classified by the report as “expensive drugs for rare diseases” (EDRDs).

As of the end of 2020, a total of 104 EDRDs had received approval in Canada. The report said most of those have come since 2015.

EDRD sales have grown at an average annual rate of nearly 32 percent since 2011, reaching $3.1 billion in 2020, or 10.4 percent of the Canadian pharmaceutical market.

Oncology medicines makes up 54 percent of the EDRDs approved in Canada, accounting for 76 percent of EDRD sales in 2020.

Of the new medicines approved from 2015 to 2019 by the U.S. Food and Drug Administration, European Medicines Agency, or Health Canada, 62 had both an orphan designation and a treatment cost that met the required threshold for an EDRD. These 62 medicines represent 28 percent of new approvals for the period, an indication that the rapid pace of EDRD launches in Canada has also been experienced in other countries.

Author: Rare Daily Staff