Time to Jump Start Rare Disease Drug Strategy

Open Letter to Prime Minister and Federal Health Minister

POSTED PUBLICLY: www.raredisorders.ca
VIA EMAIL: pm@pm.gc.ca and hcminister.ministresc@canada.ca

April 26, 2021

Dear Prime Minister Trudeau and Health Minister Hajdu:

Subject: Time to Jump Start Rare Disease Drug Strategy

On behalf of the Canadian Organization for Rare Disorders (CORD), which represents over 100 rare disease patient groups and hundreds of thousands of patients and their families, I am writing to say “thank you” and to urge taking action now.

JUMP START RARE DISEASE DRUG STRATEGY!

On April 19 with the tabling of the 2021 budget, Finance Minister Freeland announced lots of new money for lots of new initiatives. For the rare disease community, there was no new money. There was just one reference, just one sentence, on just one page (p. 238) in the 739-page document, but it was enough to signal that the future is bright for rare disease patients in Canada. “To maintain momentum, the government will proceed with its announced plan to provide ongoing funding of $500 million for the program for high-cost drugs for rare diseases.” 

The funding commitment was made in the 2019 federal budget. This confirmation underscores and reifies the multi-stakeholder drug strategy consultations conducted by Health Canada this past February and March. CORD had already been leading the way by hosting public dialogues over the past year, bringing together international and Canadian experts to discuss global best practices and ways forward. Drawing upon this input, CORD set forth a collective vision for a Canadian Rare Drug Strategy that is undergird by a comprehensive Rare Disease Strategic Framework, with a 12-step implementation plan.

As importantly, the bright future envisioned in a Canadian Rare Drug Strategy can begin right now. We can jump start at least half of CORD’s proposed 12 steps in advance of the $500 million realization. In reality, the $500 million “ongoing” funding should not be allocated as an annual expenditure but as an annual investment. We are not spending $500 million a year for rare disease drugs but investing into an optimal rare disease system that will assure the right drugs are being used effectively and cost-effectively. Moreover, CORD calls upon the pharmaceutical industry and other stakeholders to match the annual $500 million to produce a $1 billion annual investment fund.

How can we invest an annual $500 million federal plus $500 million private funding to leverage the best patient and societal outcomes? Rare disease therapies are increasingly specialized, complex, uncertain, and expensive. The goal of getting effective rare disease drugs to patients as early as possible so as to avoid or minimize symptom impact requires investment in screening, testing, and early diagnosis. To achieve optimal drug use, we must provide excellent comprehensive care and treatment through networked centres of rare disease expertise linked to local healthcare providers and community services. Patient support programs and rare disease patient organizations are essential to educating patients, promoting adherence, monitoring outcomes, and collecting and reporting real-world data. In short, we need to deliver comprehensive coordinated rare disease care through a network of integrated rare disease specialists, allied health providers, primary care providers, and community-based resources. 

In Canada, there are many world-class, state-of-the-art national research-supported comprehensive care programs that focus on a single rare disease or a cluster of conditions. Examples include but are not limited to: the Canadian Inherited Metabolic Diseases Research Network, the Canadian Fabry Disease Initiative, the Neuromuscular Disease Network for Canada, Pediatric Bone Health Clinical and Research program, Canadian Hemophilia Treatment Centres, and the Cystic Fibrosis Care Centers. 

The imperative for a Rare Disease Strategy to assure the success of a Rare Disease Drug Strategy cannot be overstated. The 2021 budget rightly recognized pediatric cancers as a leading cause of death among Canadian children. in reality an estimated 12% of pediatric deaths are due to (rare) cancers and 46.6% are due to rare disease NOT including cancers. That translates to 29,000 Canadian children under the age of 15 dying as a consequence of a rare disease every year. As concerning, more than half of pediatric hospital beds are occupied by a child with a rare disease. Collectively, more children and adults are affected by rare diseases than by all cancers combined or by all cardiovascular diseases, including stroke. More than 80% experience delayed, wrong, or no diagnosis. The result is an insidious cycle of numerous consultations with doctor after doctor, predictably frequent ER admissions, referrals for inappropriate, useless, and costly treatments and prescriptions for wrong, ineffective, or sometimes harmful medications.

Unlike cancers, the burden of rare disease on the healthcare system, the impact on healthcare spending, and the widespread familial and societal costs are mostly unrecognized. Many cases of rare conditions are undocumented or undiagnosed and there is no cumulative accounting of up to 7,000 rare diseases. Nevertheless, the impact is real and it is enormous; we are just beginning to realize how much it costs NOT to prevent, diagnose, and appropriately treat rare diseases. With a burgeoning number of rare, complex, and precision therapies in the pharmaceutical pipeline, inaction is not an option. Health Canada’s consultations touched upon a number of key issues that are essential to a Rare Drug Strategy, notably patient engagement, expert committee(s), managed access programs, patient registries, and use of real-world data for post-launch assessment. All of these are components of a Rare Disease Strategy and Framework, as articulated by CORD initially in 2015. Many of these already exist in the disease-specific programs previously introduced but many programs require additional resourcing to be sustainable and to support the new drugs and other technologies.

We can “jump start” the new Canadian Rare Drug Strategy by building on existing infrastructure and expanding to other disease areas, especially those which can be flagged with imminent innovative therapies.

CORD absolutely supports fair drug pricing in Canada, for all payers, including public and private drug plans and individual consumers. But if “fair pricing” compared to other countries is the goal, then the proposed reference basket of similar countries is reasonable and sufficient. What is deeply concerning is the proposed economic factors that will create significant confusion and uncertainty in the Canadian marketplace and have a profound impact on patient access to medicines, especially for those patients with severe, life-threatening conditions where alternative therapies are not available. This is truly a matter of life and death for many patients. The federal government needs to take the time to rethink its approach and properly consult with stakeholders.  

These are all actions we can undertake almost immediately with minimal resourcing if there is a collective will. As the patient community, we are ready to take part and indeed willing to lead where appropriate.

Finally, on behalf of all Canadian rare disease families, our heartfelt appreciation for advancing the vision of optimal access to rare disease care and treatment.

Sincerely,
Durhane Wong-Rieger
President and CEO
Canadian Organization for Rare Disorders