This article originally appeared in the Financial Post.
By Nigel Rawson and Barry Katsof, September 3, 2024
Barriers our governments have built make providing Canadians with access to innovative medicines slow and difficult. The Canadian Agency for Drugs and Technologies in Health (CADTH) and the pan-Canadian Pharmaceutical Alliance (pCPA) — both owned and managed by Canada’s governments — effectively discourage the launch of new drugs in this country.
CADTH performs “health technology assessments” (HTAs) to estimate drugs’ cost-effectiveness. It also makes reimbursement recommendations to all government drug plans except Quebec’s. For its part, the pCPA negotiates drug prices with manufacturers on behalf of all government drug plans. Despite CADTH and the pCPA being government agencies, when CADTH recommends a drug be reimbursed by government drug plans and the manufacturer successfully negotiates a price with the pCPA, government plans are not required to cover the drug. Manufacturers have to negotiate with each plan, which frequently results in a patchwork of coverage, with some Canadians having access and others not.
CADTH is transitioning into Canada’s Drug Agency (CDA). This presents an opportunity for it to pivot from a culture that prioritizes process and relies on data from randomized clinical trials to one that prioritizes patients by allowing access to innovative medicines while evidence on their real-world cost-effectiveness accumulates. But this opportunity is being squandered. CDA is failing to deal head-on with the complexities and deficiencies of its review process.
Instead, it’s introducing a new procedure, called Net Zero — yes, another Net Zero — to try to “achieve zero days between Health Canada’s approval of a drug and CDA’s reimbursement recommendation to government drug plans.” This sounds like an improvement, right? If it means just one submission can be made to both Health Canada and CDA, drug developers may well see Net Zero as a positive. But if it means two individually tailored applications need to be submitted simultaneously, maybe not.
CDA has also created a new type of recommendation, time-limited reimbursement (TLR), intended “to help provide earlier access to promising new treatments targeting unmet needs of Canadians living with severe, rare or debilitating illnesses” while stronger evidence of their cost-effectiveness is collected. This a long-overdue step forward. But the only TLR drug so far approved (Epkinly, a cancer drug) took nearly seven months to receive the recommendation — longer than CDA’s “typical” timeline of six months. In fact, six months should be too long for a full review, let alone for one part of a scheme purporting to provide earlier access.
The pCPA has introduced a complementary process to allow patients “temporary access to some drugs earlier”— which is another step in the right direction. Using it, the pCPA engaged with Epkinly’s developer before making its recommendation — a first for the agency — and reached a price agreement within its own performance target of 90 business days.
Despite these encouraging developments, CDA and the pCPA are only nibbling around the edges of the issue of delayed access to innovative medicines. Canadians requiring access to these new drugs need the barriers erected by these agencies and government drug plans to be radically downsized, if not completely removed.
Over the years, patients, their families and health care providers have produced countless articles, letters and other testimonials describing the obstacles to drug access in Canada and calling for their removal. But with little effect. In its 2019 budget, the federal government promised a strategy for drugs for rare diseases. But five years later, only two drugs have been included in the “strategy,” which has to be negotiated bilaterally with each province — which so far only British Columbia has done.
Politicians, government officials and their academic advisers close their eyes and ears to polite appeals from patients and their supporters — which is strange, considering that politicians usually like to be seen helping Canadians in trouble. Why are people in need of innovative medicines different? Is it fear of a negative political reaction to being seen to give in to Big Pharma?
Patients in desperate need of drugs for disorders that, untreated, lead to physical or mental disability and/or premature death need to become much more vocal. They need to conduct highly visible, forceful and relentless media campaigns to embarrass decision-makers into acting. One such very active media campaign in 2010-11 for Canadians with paroxysmal nocturnal hemoglobinuria (PNH), a rare, chronic, debilitating and life-threatening disease of the bone marrow that affects the blood and major organs, did lead to a new drug for PNH being funded across Canada. Making noise and confronting decision-makers in the media seems to be essential to getting change.
Ontario Premier Doug Ford has said: “We owe it to Canadians to do everything we can to give them the same timely access to life-changing treatments as patients in the rest of the world.” But instead of just talking the talk, Premier Ford needs to walk the walk — and persuade his fellow premiers to do the same. Our decision-makers need to be reminded that their job is to better the lives of Canadians, not just further their own ambitions.
Nigel Rawson is a senior fellow with the Macdonald-Laurier Institute.
Barry Katsof is founder and president of the Canadian Association of PNH Patients.
