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Federal Health Minister Jean-Yves Duclos speaks at a news conference in Montreal, on March 22, during which he announced details for a national strategy for drugs for rare diseases.Graham Hughes/The Canadian Press

Federal Health Minister Jean-Yves Duclos announced details of Ottawa’s long-awaited strategy for rare diseases on Wednesday, which includes $1.4-billion over three years to help provinces and territories pay for drugs as well as improve early diagnosis and screening.

“We estimate that the funding could benefit up to 9,600 additional patients, including over 4,500 children,” Mr. Duclos said.

Mr. Duclos made the announcement as he faces questions about why he asked the country’s drug regulator to suspend work on drug reforms late last year. The reforms would have, in part, set caps on the prices drug companies can charge for certain brand-name drugs. Many of the most expensive brand names on the market today are used to treat rare diseases, which is why advocates of price reform say those caps were needed.

But last November, Mr. Duclos asked the Patented Medicine Prices Review Board to suspend its consultation process, which has indefinitely delayed the adoption of the new price reforms. Mr. Duclos’s request led to several resignations, according to Matthew Herder, who left his position at the review board over what he described as inappropriate interference from the minister’s office.

Mr. Duclos’s office has stated that the request to suspend was above board because the board failed in its duty to conduct a rigorous consultation.

Patient advocates say the new federal funding and establishment of a national strategy will help address long-standing gaps, such as delays in accessing treatment and a lack of investment into studying conditions that only affect a small number of individuals. While there’s no set definition for what constitutes a rare disease, many describe it as a condition that affects fewer than one in 2,000 people. One in 12 Canadians live with a rare disease, according to the Canadian Organization for Rare Disorders, an advocacy group.

Rare diseases have been overlooked and neglected, but there has been a major rise in the development and availability of drugs to treat them in recent years, thanks in part to scientific advancement and more government incentives to focus on them. But the treatments are typically quite expensive, keeping them out of reach of many patients and raising complicated questions about how much governments should pay for these new therapies.

A January, 2022, report from the Patented Medicine Prices Review Board, an independent agency established to protect Canadians from excessive drug prices, found that rare-disease drugs priced in excess of $100,000 a year are “an increasingly important segment of the market.” Sales of those expensive drugs have grown at an average annual rate of nearly 32 per cent since 2011, accounting for more than 10 per cent of the Canadian pharmaceutical market in 2020, the report found.

Now that the review board’s promised drug-price reforms have been suspended indefinitely, it’s unclear how the government will continue to ensure patients have access to rare-disease drugs, given that their costs are often so high.

“The fastest growing expenditures on pharmaceuticals are EDRDs, the term used for expensive drugs for rare diseases,” Mr. Herder said. “If supporting meaningful price regulation is not part of the plan, I have no idea how that goal of affordability is going to be achieved.”

During his announcement on Wednesday, Mr. Duclos said Ottawa will create bilateral agreements with provinces and territories to determine how the funds will be spent. Once agreements are reached, he said, it will enable provinces and territories to add a range of rare-disease drugs to the list of medications they cover.

Durhane Wong-Rieger, president of the Canadian Organization for Rare Disorders, said she hopes the funding will help address long-standing problems, such as lack of access to timely screening and diagnosis for many rare conditions.

“You can’t treat someone if you don’t diagnose,” she said. “We are not up to date in terms of newborn screening and early screening and diagnosing and testing for many of these diseases.”

Ottawa will also provide $33-million to the Indigenous Services Canada’s Non-Insured Health Benefits Program to help First Nations and Inuit people living with rare diseases.

While most of the new funding is directed at funding for medications, the government is also earmarking $68-million toward collecting and using evidence to improve decisions around rare-disease treatments, developing better diagnostic tools and creating a rare-disease clinical trials network; and the creation of working groups to support the implementation of the strategy.

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