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Feb. 29 — that rarest of days — is Rare Disease Day globally. Here at home it will be recognized by the three million Canadians living with these diseases and their families and friends. Many landmarks across the country will be illuminated to honour the day.

Canadians with rare diseases face daunting challenges. For most, no therapies exist beyond symptom treatment and palliative care. For others more fortunate, a drug may be approved in Canada but access ranges from difficult to impossible, mostly due to restrictions imposed by private or government drug plans. For a growing number, treatments exist but are not available here because government incentives to encourage developers to bring them in are lacking.

Unlike the United States, Europe and Australia, Canada has no “orphan drug” act providing incentives to motivate developers to launch medicines for small numbers of patients. A study of 120 drugs approved as orphan medicines in the U.S. and/or the EU between 2015 and 2020 found that as of June 30, 2023, only 82 had been authorized for marketing in Canada. Moreover, the average wait time between first submission in the U.S. or Europe and submission to Health Canada was over 15 months; for a quarter of the drugs, the wait exceeded 20 months.

The Canadian Agency for Drugs and Technologies in Health (CADTH) conducted “health technology assessments” (HTAs) for 78 of the 82 drugs, recommending nearly 90 per cent for reimbursement. But HTAs averaged almost nine and a half months — much longer for a quarter of the drugs. Fewer than one in 10 HTAs were completed within CADTH’s “typical review” timeline of 180 days. CADTH underperforms even by its own metric.

For 60 of the 82 drugs, the pan-Canadian Pharmaceutical Alliance (pCPA) negotiated prices with manufacturers on behalf of government drug plans. The pCPA’s target just for deciding whether to negotiate is eight weeks. But it hit that for only 17 per cent of drugs. Once it did decide to negotiate, the average negotiation ran seven months. Only 20 per cent were completed within the self-imposed deadline of 18 weeks. The pCPA also misses its own targets.

Then finally the government plans must approve the medicines for funding. It took three and a half years or more before half the 82 drugs got through the HTAs, the price negotiations and the provincial listing processes so that some Canadians in some provinces were finally eligible to access them.

Back in 2019, the federal government committed $1.5 billion over three years to “increase access to, and affordability of, effective drugs for rare diseases” — but with no empowering law. We’re coming up to five years since that commitment and the first dollar has yet to flow in any province and no patient has received a drug through this “strategy.”

Compared to the billions of dollars Ottawa is providing for dental care and pharmacare or the tens of billions it’s spending on electric car batteries, $1.5 billion is petty cash. Canadians with rare diseases feel left out in the cold by their governments.

So, what should we do? We need more persuasive incentives to encourage developers to bring innovative drugs to Canada. We should have lower fees for regulatory evaluations and HTAs (for a novel therapy, manufacturers currently pay $565,465 for a Health Canada evaluation and between $98,670 and $172,450 for a CADTH HTA). Any orphan medicine that has gone through a successful pCPA price negotiation should have to be listed by all governments participating in the negotiation. And we should loosen restrictive and even harmful access criteria that patients and clinicians must satisfy before being able to obtain coverage.

Canadians with rare disorders want more than pretty lights and platitudes. They need real action that will help them access new medicines that can relieve their suffering and extend their lives. Feb. 29 should be more than just a day when Canadians with rare diseases publicize — yet again — the roadblocks and delays governments put between them and their access to innovative medicines that can help them. Instead, let it be the day when Canadians lucky enough not to be struck by these diseases and conditions resolve to improve the lives of their fellow Canadians who have been.

John Adams, co-founder and CEO of Canadian PKU and Allied Disorders Inc., has a son with phenylketonuria (PKU).

Beth Vanstone, whose daughter has cystic fibrosis (CF), advocates for Canadians with CF and other rare diseases.

Nigel Rawson is a senior fellow with the Macdonald-Laurier Institute

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