In Canada, one out of twelve people—many of whom are children—has a rare disease. There are thousands of different rare diseases that affect patients, their families and caregivers across the country. Unfortunately, treatments are only available for a small percentage of these conditions. Even when treatments that might dramatically improve the quality of a patient’s life are available, they are often unaffordable and out of reach.

Today, the Honourable Jean-Yves Duclos, Minister of Health, announced measures in support of the first-ever National Strategy for Drugs for Rare Diseases, with an investment of up to $1.5 billion over three years. Through this, we will help increase access to, and affordability of, effective drugs for rare diseases to improve the health of patients across Canada, including children.

As part of this overall investment, the Government of Canada will make available up to $1.4 billion to provinces and territories through bilateral agreements. This funding will help provinces and territories improve access to new and emerging drugs, as well as support enhanced access to existing drugs, early diagnosis, and screening for rare diseases. This will help patients with rare diseases, including children, have access to treatments as early as possible, for better quality of life.

As a critical next step to advance the development of these bilateral agreements, the Government of Canada will engage with provinces and territories to jointly determine a small set of new and emerging drugs that would be cost-shared and covered in a consistent way across the country, for the benefit of patients. Similarly, to further support eligible First Nations and Inuit patients living with rare diseases, $33 million from this funding will go to Indigenous Services Canada’s Non-Insured Health Benefits Program.

In addition, we will also invest $68 million for various initiatives to help improve consistent access to drugs for rare diseases across the country, including:

• $20 million over three years to the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Canadian Institute for Health Information (CIHI) to improve the collection and use of evidence to support decision-making;
• $32 million over five years to the Canadian Institutes of Health Research (CIHR) to advance rare disease research with a focus on developing better diagnostic tools and establishing a robust Canadian rare disease clinical trials network; and,
• $16 million over three years to support the establishment of national governance structures, such as a Health Canada secretariat and a stakeholder Implementation Advisory Group, to support the implementation of the Strategy.

Today’s announcement is the result of feedback from extensive consultation that garnered diverse perspectives from over 650 individuals and organizations, including patients with lived experience, family members, and caregivers. To create this Strategy, lived experiences were taken into careful consideration to ensure Canadians living with rare diseases have access to the drugs they need and receive better treatments and services, regardless of where they live. In addition, this Strategy will improve the collection and use of evidence and strengthen investments in critical research and innovation in rare diseases.

The Government of Canada will continue to work with provinces, territories, and key partners to deliver on these improvements so those living with rare diseases can have better health outcomes and access to cutting-edge treatment options.

Quotes

“Access and affordability should never be a barrier to care. Through this new Strategy, Canadians living with a rare disease will soon have improved screening and diagnostics, and better access to affordable and effective treatments they need, no matter where they live. We will keep working with provinces and territories and all relevant partners so those living with a rare disease, including children, have the best health outcome possible while their caregivers get the support they need and deserve.”

The Honourable Jean-Yves Duclos
Minister of Health

“As the Federal Government launches the National Strategy for Drugs for Rare Diseases, the Non-Insured Health Benefits Program (NIHB) at Indigenous Services Canada will receive new investments of $33 million. This investment will help ensure that First Nations and Inuit eligible for NIHB prescription coverage will have the support needed to better access drugs for rare diseases.”

The Honourable Patty Hajdu
Minister of Indigenous Services and Minister responsible for the Federal Economic Development Agency for Northern Ontario

“This investment will enable improved access to new and emerging drugs for Canadians with rare diseases, including children, as well as support to early diagnosis and screening. This is a first step in looking at drugs for rare diseases coverage from a national perspective – consistently ensuring that patients can access the drugs they need regardless of where they live in the country.”

Adam van Koeverden
Parliamentary Secretary to the Minister of Health and to the Minister of Sport

Quick facts

• Innovative treatments for rare disease can cost anywhere from $100,000 to more than $2 million per year. Budget 2019 provided funding up to $1 billion over two years, with up to $500 million per year ongoing, to help Canadians with rare diseases access the drugs they need.
• In July 2021, Health Canada released a What We Heard Report from national engagement on the National Strategy. The engagement garnered diverse perspectives from over 650 individuals and organizations, including patients with lived experience, family members, and caregivers.
• In December 2022, Health Canada pre-published proposed amendments to the Food and Drug Regulations that would introduce regulatory flexibilities such as the option of a rolling review, which may facilitate earlier market access for certain drugs, including drugs for rare diseases. The proposal would also allow for terms and conditions for all drugs, enabling Health Canada to include obligations on the manufacturer to optimize the benefits and manage any risks and uncertainties associated with the drug. This would be particularly important in the cases of drugs for rare diseases where patient populations are small.
• Additionally, Health Canada’s Pediatric Drug Action Plan aims to improve access to safe and effective medicine for children in Canada, including drugs for rare diseases, which tend to be more prevalent in the pediatric population.

After clinical and pharmacoeconomic reviews of published and unpublished clinical data, INESSS and the Canadian Agency for Drugs and Technologies in Health (CADTH) have now
published their reimbursement recommendations for nusinersen. Both INESSS and the Canadian Drug Expert Committee (CDEC) recognize the therapeutic value
and benefits of nusinersen however only for a subset of the population affected with SMA, notably within the Type 1 population.

Biogen recognizes that the INESSS and CDEC recommendations are important initial steps in providing formulary listing recommendations to provincial drug plans. We are disappointed at
the restrictive nature of these recommendations given the clinical value that nusinersen can provide to individuals living with SMA in Canada.
We believe that the medical and patient communities, as well as Biogen and the provincial
authorities share a common goal of bringing nusinersen to families impacted by SMA as quickly and as broadly as possible. We remain committed to finding the best way forward to achieve
broad and timely access. Nusinersen is the first and only Health Canada-approved therapy indicated for the treatment of SMA in pediatric and adult patients. The efficacy and safety of nusinersen
for the treatment of 5q SMA has been demonstrated in patients across the spectrum of the disease.

The nusinersen clinical program, the largest in SMA to date, is comprised of multiple studies across a range of SMA patients, including presymptomatic and symptomatic patients who had, or were likely to
develop Type 1, 2, or 3 SMA. The certainty of the benefits seen early on in these clinical trials resulted in accelerated and prioritized regulatory approvals across the globe.

Now that CDEC has issued its recommendation, the participating drug plans can act upon the recommendation and begin negotiations on a provincial level or via the pan-Canadian
Pharmaceutical Alliance (pCPA). Currently, it is not known how long pCPA negotiations will take nor which Provinces will engage in negotiations. Biogen is committed to working with the pCPA
and provincial governments to achieve public reimbursement as soon as possible. Biogen is dedicated to further improve the futures of patients and families living with SMA.
Biogen will continue to provide nusinersen for existing and new eligible infantile-onset (Type 1) patients through its expanded access program.

In addition to the already available extensive evidence proving clinical value of nusinersen across the spectrum of SMA patients, Biogen and the physician community will continue to
further expand the evidence available by collecting and analyzing data notably through SHINE, an open-label extension study across different SMA populations. These efforts will continue to
provide a deeper understanding of SMA and nusinersen. More information on studies in SMA can be obtained at www.clinicaltrials.gov.

We remain a dedicated partner to the SMA community and will continue to work tirelessly to ensure that patients who may benefit from nusinersen receive access as quickly as possible.

Sincerely,
The Biogen Team
For further questions, please contact Medical Information at:
1 866 477 3462 or canmed@biogen.com