This Rare Disease Day, Feb. 29, CORD celebrates the launch of the Canadian Rare Disease Network - but patients still have no access to the promised $1.4 billion in federal funds for rare disease drugs

• The federal government promised a Rare Disease Drug Strategy five years ago and announced $1.4 billion in drug funding last March. Today not a single patient has received a single penny from that fund, despite urgent unmet needs
  
  
• The uniting of healthcare professionals and patient organizations under the new Canadian Rare Disease Network will accelerate patient diagnosis and specialist care – but for many patients they will have no access to life-saving and life-altering drugs that are already approved and recommended but not yet available through public drug plans

OTTAWA, ON, Feb. 29, 2024 /CNW/ - The Canadian Organization for Rare Disorders (CORD) is marking Rare Disease Day today – the rarest day on the calendar – with both optimism and impatience.

CORD is wildly optimistic with the milestone launch of the Canadian Rare Disease Network (CRDN) – a collaboration that brings into reality the vision and strategy that CORD first proposed in 2015. CRDN brings together patient groups, clinicians and researchers under a shared vision to take coordinated action to provide support and care for the one out every 12 Canadians with a rare disorder and their families (since two-thirds of those with rare diseases are children).

Patients are at the heart of the CRDN and meeting the needs of patients across the diverse spectrum of rare diseases is the group's top priority. The CRDN will work to bridge gaps in rare disease care, innovation and research while working in a united way to ensure better access to diagnosis, care and treatments to empower patients and families, while also ensuring better collection of data to drive future investments and care plans.

While celebrating the birth of the CRDN, the rare disease community is marking Rare Disease Day with mounting impatience due to unconscionable delays in making available the committed federal government funding for rare disease medicines.

Funding was first promised in 2019 – five years ago. Last March, the federal government announced its rare disease strategy and spending plan of $1.4 billion for treatments through bilateral agreements with the provinces/territories and $100 million for federal drug plans and other research, data collection and evaluation initiatives.

However, to date, no new drug funding has become available and not a single Canadian rare disease patient has benefited, despite a huge need for new, approved, and recommended treatments for life-threatening diseases.

"We had hoped that this February 29th Rare Disease Day 2024 would have been a full-throttled celebration with a Canadian Rare Disease Network to support the roll-out of the funding from the Canadian Rare Disease Drug Strategy," said Durhane Wong-Rieger, President and CEO of CORD. "We are thrilled and congratulate everyone involved in working to create the new CRDN which will play a huge role in helping patients access better diagnosis, care and treatments for rare disorders while also promoting research and greater clinical collaboration across Canada."

"However," she added, "the vital missing link is access to the long-promised new funding for treatments. Patients have every right to be impatient. We need access to those new treatments now before more lives are put on hold or even lost. We support negotiations and agreements to assure sustainable access, but we can also take immediate action to address patients with urgent needs. You must serve those in the Emergency Room even while you are developing plans for long-term care."

CORD has proposed an implementation plan by which rare disease therapies that are approved by Health Canada and recommended by value assessments would be made available immediately to patients along with a plan for on-going monitoring, data collection and assessment of benefit and potential harm.

According to Wong-Rieger: "We know exactly which drugs are languishing in bureaucracy while patients wait, and they are often ones that have already gone through health technology assessments and the pan-Canadian Pharmaceutical Alliance to establish favourable funding terms. The federal government has shared funding responsibility in the past with provinces, such as the case for Fabry's disease nearly twenty years ago. This is not rocket science – this is simply doing what you promised to do."

Ironically, Canada was the first country in the world to approve two new therapies for rare diseases, but no Canadian patient has gained access due to the lack of funding for the treatments.

CORD has been holding a Rare Disease Day 2024 Summit in Ottawa February 28 and 29. As part of the Summit activities, participants will march to Parliament Hill on the morning of February 29 to further press their case for urgent action to release the new federal funding and make vital new treatments available to Canadians with rare diseases.

About Canadian Organization for Rare Disorders (CORD)
CORD is Canada's national network for organizations representing all those with rare disorders. CORD provides a strong common voice to advocate for health policy and a healthcare system that works for those with rare disorders. CORD works with governments, researchers, clinicians and industry to promote research, diagnosis, treatment and services for all rare disorders in Canada. For more information, visit www.raredisorders.ca

SOURCE CANADIAN ORGANIZATION FOR RARE DISORDERS (CORD)

For interviews and media enquiries: Don Sancton on behalf of CORD, [phone], [email protected]