Thank you very much, honourable chair and members of the health committee.
Thank you for the opportunity to speak to you today. My name is Durhane Wong-Rieger, and I am the president and CEO of the Canadian Organization for Rare Disorders. I'm here to discuss Bill C-64 and, in part, its implications for the rare disease community in Canada.
I'd like to start, though, with a few facts that paint a bleak picture for Canadians with rare diseases. You may know that rare diseases affect over three million Canadians, the majority of whom are children. While most rare diseases affect children, we also know there are a significant number of adult-onset rare conditions that are being diagnosed.
Among the 7,000 known rare diseases, only 5% have an effective drug therapy. Unfortunately, one in three rare disease patients in Canada cannot access their treatments. In fact, only 60% of the treatments for rare disorders are made available in Canada, and most get approved up to six years later than they do in the U.S. or in Europe.
Even after the treatments are approved in Canada, many patients continue to face immense hurdles and delays in accessing new treatments due to the challenges related to the evaluation and funding of these medicines. When there are effective, available therapies, access can often be very challenging for patients. As you may know, they vary from one province to the other. As a result of these challenges, many patients experience an avoidable decline in functionality, and certainly many experience early death.
In an effort to respond to these challenges, on March 22, 2023, the federal government announced measures in support of Canada's first-ever rare disease drug strategy, including, at this time, a $1.4-billion investment for provinces and territories to improve access and affordability of rare disease medicines. This money had already been promised half a decade ago as part of budget 2019.
However, it's now been over a year since the funding announcement, and not a single penny has been spent to help fund rare disease drugs. While CORD supports efforts to improve access to medicine for all Canadians who need them, we're also concerned that the federal government has taken on another major commitment to fund a national pharmacare program when it hasn't even delivered on this promise to fund rare disease treatments—a promise that, as we said, was made over five years ago.
Notably, clause 5 of today's Bill C-64 would commit the government to long-term funding, beginning with products for rare diseases. We have to say it's unconscionable and unethical, and certainly really challenging for patients, to introduce a program designed to transform and save lives, and then fail to execute on it.
Moreover, given the lack of promised progress on rare diseases, what does that say in terms of the prospects for success of this pharmacare legislation? We need to see the prioritization for rare diseases in action. It was a promise made. When it comes to improving medicine access and affordability, CORD strongly believes that rare diseases represent the area with the greatest unmet need in Canada.
The federal government should focus first on rolling out the promised funding for rare disease treatments before undertaking another major pharmacare plan.
However, with respect to the bill itself, CORD has a number of comments. The predetermined categories, lists of medicines and proposed single-payer approach all risk limiting treatment options and potentially bringing everyone's level of coverage down to the lowest-common denominator. This is a concern.
Additionally, Bill C-64 outlines specific timelines for its key components, yet the rare disease drug strategy lacks a detailed implementation plan and time frame. The rare disease drug strategy must also be afforded clear timelines, publicly accountable milestones and opportunities for patient and clinical input. This is not in the current rollout.
Lastly, the formation of expert committees, as stated under Bill C-64, must ensure genuine advisory roles. CORD's experience with the current rare disease drug strategy implementation advisory group has highlighted significant issues with transparency, communication and accountability. Effective implementation of national pharmacare requires these committees to provide meaningful input, rather than service mere formalities.
I'd like to close by noting that Canada has an opportunity to become a leader in providing access to cutting-edge therapies that significantly impact patients' lives. We must aim high, ensuring that our national pharmacare program and the rare disease drug strategy deliver the best possible outcomes for patients with rare and common diseases alike.
Thank you very much for your attention. I'm open to any questions you may have.