By Allison Martell
TORONTO, Aug 13 (Reuters) - Canada's patented drug price regulator, set to gain new powers next year, may be "more forgiving" in setting price caps for drugs that treat rare diseases, the agency's executive director told Reuters, as some advocates warned the country's pricing reforms would hurt patients.
The Canadian government announced final regulations meant to cut drug spending on Friday. The reforms expand the Patented Medicine Prices Review Board's (PMPRB) powers, and among other things give it the ability to consider the cost-effectiveness of medications.
Doug Clark, executive director of the PMPRB, noted that improving access to rare disease drugs was part of the most recent federal budget.
"We've signaled for some time that we're going to be a bit more forgiving in the ceilings that we apply to these drugs, because we want to be consistent with the government's broader commitment to trying to facilitate access," he said in an interview on Friday, referring back to technical documents provided to some advisory committees.
While rare disease drugs face small markets by definition, high prices and policies that speed their approval have created a profitable niche for some drugmakers. That trend may accelerate with the introduction of gene therapies like Novartis's new Zolgensma, which at $2.125 million for a one-time treatment is the world's most expensive drug.
Clark's assurance was little comfort to Durhane Wong-Rieger, president of the Canadian Organization for Rare Disorders (CORD), who fears lower price caps would stop drugmakers from selling treatments for rare conditions in Canada.
"The overall budget impact of these drugs, even if we brought them all in, would not even reach 1 percent," she said, referring to provincial drug programs that often cover the most expensive treatments. "Why are we disproportionately disadvantaging patients for very little net gain?"
Canada's patented drug prices are among the highest in the world, and the federal government has said many countries with lower prices have similar or better access to drugs.
Much depends on how the PMPRB uses its new powers, which will be laid out in guidelines that are still being drafted. Clark did not go into much detail, citing that process.
The government's regulatory impact assessment assumed the PMPRB would judge drugs against cost per quality-adjusted life year benchmarks of between C$35,000 a year and C$150,000 a year, with the high end for rare diseases. Final thresholds will likely be laid out in the guidelines.
"That's not going to work," Wong-Rieger said of the C$150,000 benchmark. "It's not enough."
Under Clark, the PMPRB has formally challenged the price of two rare disease drugs: Alexion Pharmaceutical Inc's Soliris and Horizon Therapeutics' Procysbi.
Erin Little, whose daughter has a disease called cystinosis, runs a small cystinosis research foundation called Liv-A-Little. Procysbi is one treatment for cystinosis, and regulators have said it costs up to C$325,000 per year in Canada.
Little is critical of groups like CORD, which receive funding from drugmakers and often take similar policy positions. On PMPRB reforms especially, patient groups have been closely aligned with drugmakers, like the industry lobby group Innovative Medicines Canada, arguing lower prices could delay drug launches.
Wong-Rieger said pharmaceutical company funding has no influence over her positions.
For her part, Little said PMPRB reforms look like a move in the right direction.
"I am somebody who believes that our pharmaceutical companies need to be questioned as to why their prices are so high," she said. "The pharmaceutical companies are always 10 steps ahead of us, and they know what they can get away with, and that is the problem." ($1 = 1.3238 Canadian dollars) (Reporting by Allison Martell; editing by Denny Thomas and Jonathan Oatis)