Additional barriers could further delay or prevent specialty drugs from coming to market
.jpg)
The federal government’s drive to improve “affordability, accessibility and appropriate use of prescription drugs” will disproportionately affect those with rare disorders, according to a new study from the Fraser Institute.
The study, titled Regulatory, Reimbursement, and Pricing Barriers to Accessing Drugs for Rare Disorders in Canada, examined different hurdles that drug treatments of rare disorders must overcome before getting to Canadian patients. These include the national regulatory review and approval process, pricing controls, health technology assessment processes, price-negotiation processes between public plans and drug manufacturers, and eligibility criteria for patients themselves.
“Pharmaceutical manufacturers place Canada lower than the United States and Europe on their product launch sequence of countries,” wrote Dr. Nigel Rawson, president of Eastlake Research Group and author of the paper. Of the drugs against rare or ultra-rare disorders submitted to Health Canada between 2002 and 2016, 84% were first submitted to the US Food and Drug Administration and the European Medicines Agency; the median delay was 253 days.
In addition, “23 drugs for rare or ultra-rare drugs approved by the European Medicines Agency and/or the Food and Drug Administration between 2002 and 2016 were not approved in Canada by the end of 2016,” Rawson said.
He warned that proposed reforms to the Patented Medicine Prices Review Board’s (PMPRB) processes could present even bigger barriers. The removal of US and Switzerland from the PMPRB’s comparator countries for drug pricing, followed by their replacement with seven lower-priced markets such as Australia, Japan, and Norway, is expected to lower the bar for “excessive” prices. Other new measures include a cost-effectiveness test as well as an assessment of a drug’s potential impact on patients’ finances, which is based on its anticipated market size over the first three to five years of use and Canada’s GDP per capita.
“The proposed changes are anticipated to result in reductions in the price of drugs for rare and ultra-rare disorders by 70 to 90 percent, which would be a major disincentive to the marketing of these drugs in Canada,” Rawson said. Not only would the lower maximum allowable prices not be enough to cover distribution costs, he asserted, but they would also encourage other countries that watch Canadian prices to also demand discounts.
“Rather than providing hope to patients needing costly new drugs for previously untreatable conditions, Canadian governments appear to be moving towards a basic ‘pharmacare’ system built on a formulary of inexpensive genericized drugs and a small, restricted-access list of specialty drugs (including drugs for rare and ultra-rare disorders) limited to those available from manufacturers willing to negotiate substantial price reductions,” he said.
