Canada’s rare disease community saw cause for celebration in March of 2023, when its many years of advocacy appeared to pay off. At last, it was witnessing a firm commitment to significant action, as the federal government announced the National Strategy for Drugs for Rare Diseases.
The strategy came with a pledge to provide up to $1.5 billion over three years. The majority of this funding ($1.4 billion) was to be allocated through bilateral agreements with provinces and territories to help improve access to new and emerging drugs for Canadians with rare diseases.
However, as Rare Disease Day arrives close to one year later, on February 29, 2024, the overriding sentiment in the community is one of disappointment.
“There is no sense of urgency, no goals to treat patients as soon as possible, no timelines for expending the $1.4-billion, and, most grievously, no inclusion of patients and health-care providers in the negotiations,” states the Canadian Organization for Rare Disorders (CORD) and its Quebec counterpart, the Regroupement québécois des maladies orphelines (RQMO), in a recently released open letter to the Prime Minister, premiers and health ministers.
It is truly frustrating for those of us who have been pushing for progress all this time, especially the patients and their families.
The federal government first promised funding of $1-billion to set up a rare disease drug strategy in its 2019 budget. Then, we waited four years for the big announcement of 2023 – and we thought, finally, we will see people get access to some of the therapies. But we are still waiting for monies to flow to patients in funded treatments.
The community does appreciate that the federal government has invested $68-million in a number of positive initiatives, including more money for rare disease research and a clinical trials network, and better monitoring and data collection.
The landscape of rare diseases in Canada
A disease is “rare” when it affects fewer than one in 2,000 people. There are at least 7,000 different rare diseases, and the cumulative numbers are significant; in Canada, 3.2-million individuals have a rare disease. As most of the disorders are hereditary, more than two-thirds of those affected are children – and one-third of these children will not live to see their fifth birthday.
Safe and effective therapies have been approved in Canada for only a small fraction of the total array of rare disorders. (Treatments are available for only five per cent of these disorders.) But too many of those treatments are literally languishing in bureaucracy.
Patients have been waiting to get access to many therapies – sometimes for more than three or four years.
Many patients are urgent cases, suffering with diseases that are getting progressively worse. The government has this large pool of money to dispense, but waiting for provincial-territorial agreements to be reached could take years.
We are saying, take some of that money and start funding treatment for patients who need these drugs today. There are ways to fund patients with urgent needs even while they’re putting in place the bigger plans and negotiating pricing agreements for the long term. This type of approach – fund patients now and negotiate final prices later – has been used successfully by some countries in the European Union, for example.
The rare disease community is also frustrated that they have been given no concrete information about how the negotiations for bilateral agreements are going and that CORD’s offer to help with the discussions seem to have fallen on deaf ears.
What we are saying is let us help the governments achieve progress. We have the expertise and the knowledge of patients’ needs. After all, we first developed a detailed rare disease strategy in 2015.
Delivering a direct message to Parliamentarians
Rare Disease Day is held on the last day of February in more than 100 countries around the world. In Canada, patient advocates have for several years coordinated a campaign to raise awareness on that day by encouraging cities and provinces to illuminate legislative and city hall buildings and other landmarks, such as the CN Tower.
This year, CORD and its partners are marking the occasion with a two-day conference in Ottawa and a visit to Parliament Hill. The delegation’s visit today will include a meeting with Members of Parliament from all parties.
The goal is to reinforce the community’s urgent message: the promise was made and now is the time to make good on that promise, so start the flow of these committed funds to enable Canadians with rare disorders to gain timely access to life-changing treatments.
Durhane Wong-Rieger is the President and CEO of the Canadian Organization for Rare Disorders (CORD).