New PMPRB rules could impact patients with rare diseases: study

The federal government’s plan to increasingly regulate pharmaceutical costs could mean Canadians with rare diseases may lose access to new innovative drug treatments, according to a new study by the Fraser Institute.

In 2019, the Patented Medicine Prices Review Board is planning to use new rules for establishing drug prices with the goal of lowering costs.

“Drugs for rare diseases are often very expensive because they are costly to develop and target a small number of patients, so when governments push too hard to lower prices, pharmaceutical companies have little incentive to launch these drugs,” said Dr. Nigel Rawson, a senior fellow at the public policy think tank and author of the report. 

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The study, which defines rare diseases as those affecting less than 10 of every 100,000 people, found patients with these diseases are already denied new drugs that are available elsewhere. For example, between 2002 and 2016, 23 drugs for rare or ultra-rare diseases that were approved for use in Europe and the United States weren’t available in Canada, noted the study.

Moreover, it said Canada currently doesn’t have an orphan drug policy, which it noted can help incentivize pharmaceutical companies to launch medicines for rare diseases. In the U.S. and Europe, on the other hand, pharmaceutical companies receive tax credits for clinical testing, reduced or waived administrative fees and temporary market exclusivity for orphan drugs, according to the study. 

Read: Plan sponsors bracing for onset of new orphan drugs

The study argued that lowering costs by restricting the prices that manufacturers can charge for certain drugs in Canada will discourage pharmaceutical companies from marketing all drugs in this country, but especially those for rare diseases.

“Patients suffering from rare diseases in Canada are already denied many treatments available elsewhere, and the federal government’s changes to drug pricing will make the situation even worse,” said Dr. Rawson.