Question Period Note: Drugs for Rare Diseases

• Rare diseases are often chronic, can be seriously debilitating and potentially life-threatening. With few or no treatment options, available treatments can be high-cost, which poses significant challenges to patients, caregivers, and the health care system, including the sustainability of public and private drug plans that pay for these drugs.

• On March 22, 2023, the Government of Canada 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.
• We are implementing this Strategy by working with provinces and territories towards the development of bilateral agreements. Funding for the bilateral agreements will be available April 1, 2024 and run until March 31, 2027.
• These bilateral agreements will help increase access and improve affordability of effective drugs for rare diseases to improve the health of patients across Canada.
• Further, funding to the Canadian Institutes for Health Information (CIHI) and Canadian Agency for Drugs and Technologies in Health (CADTH) is improving the collection and use of evidence to support decision-making.
• Funding to the Canadian Institutes for Health Research (CIHR) is advancing rare disease research.
IF PRESSED ON DETAILS ON PROGRESS OF BILATERAL AGREEMENTS
• Discussions with provinces and territories are underway to jointly determine a small set of new and emerging drugs, which will be cost-shared and covered in a consistent way across Canada, for the benefit of patients.
• Funding for the bilateral agreements with willing provinces and territories will start on April 1, 2024 and run for three years until March 31, 2027.
• Funding has also gone to support eligible First Nations and Inuit patients living with rare diseases, through Indigenous Services Canada’s Non-Insured Health Benefits Program.
IF PRESSED ON HOW 2023-24 BILATERAL AGREEMENT FUNDS HAVE BEEN SPENT

• Funding for the bilateral agreements with willing provinces and territories will start on April 1, 2024 and run for three years until March 31, 2027.
IF PRESSED ON QUEBEC’S PARTICIPATION IN THE NATIONAL STRATEGY
• Our Government is working with all provinces and territories to help increase access to, and affordability of, promising and effective drugs for rare diseases to improve the health of patients across Canada, while also providing flexibility for jurisdictions to address their own unique circumstances.
• All provinces and territories have been invited to engage all steps in the implementation of the Strategy, including jointly determining a small set of new and emerging drugs that would be cost-shared and covered in a consistent way.
• They have also been engaged to share in the other pillars of the Strategy: the benefits of collaborative approaches to the collection and use of evidence and innovation and research.
IF PRESSED ON DETAILS ON STAKEHOLDER CONSULTATION THROUGH THE IMPLEMENTATION ADVISORY GROUP

• An Implementation Advisory Group was launched in October 2023 to support the implementation of the National Strategy from a patient-centered perspective and to help improve consistent access and affordability of effective drugs for rare diseases across the country.
• The Implementation Advisory Group is comprised of individuals drawn from a range of perspectives and roles, including patients, caregivers, clinicians, and industry.
IF PRESSED ON FUNDING TO HEALTH SYSTEM PARTNERS
• We are working with our partners, including the Canadian Agency for Drugs and Technology in Health (CADTH), the Canadian Institute for Health Information (CIHI), and the Canadian Institutes of Health Research (CIHR) to focus on collection and use of evidence to support decision making and to advance rare disease research.
• For example, CADTH’s new newborn screening expert panel will develop guidance to foster greater consistency and timelier access to treatments.
• CIHR is providing $20M over 5 years to the Maternal Infant Child and Youth Research Network (MICYRN) to build a National Pediatric Rare Disease Clinical Trials and Treatment Network.
IF PRESSED ON WHEN PATIENTS WILL SEE BENEFITS FROM THE STRATEGY
• Funding for the bilateral agreements with willing provinces and territories will start on April 1, 2024 and run for three years until March 31, 2027
• Funding to health system partners has already begun, and is now helping to improve the collection and use of evidence to support decision-making, and advancing rare disease research and treatment networks.
IF PRESSED ON HOW THE STRATEGY IS ALIGNED WITH THE PHARMACARE ACT
• Bill C-64, An Act respecting pharmacare, commits to maintaining long-term funding for provinces, territories and Indigenous Peoples to improve the accessibility and affordability of pharmaceutical products, beginning with the funding announced for the National Strategy for Drugs for Rare Diseases.
• The National Strategy is intended to be part of continuing progress towards national universal pharmacare and is a concrete expression of the principles set out in the pharmacare bill.
• Through the National Strategy we are addressing the issue of access and universal coverage by addressing the issue of the “postal code lottery” for DRD identified in national consultations. We are also focusing on affordability and high costs of DRD that leave patients financially vulnerable, and addressing appropriate use through supporting improvements in the use and generation of real world evidence.

CANADIANS LIVING WITH RARE DISEASES
Rare diseases are life-threatening, seriously debilitating and sometimes chronic in nature. They are often genetic conditions, with onset either at birth or early childhood.

Rare diseases are not rare as a group: it is estimated there are between 6,000 and 8,000 in the world. They are considered rare because they each affect small numbers of people, ranging from a handful of individuals to a few thousand. It is estimated that one out of twelve Canadians has a rare disease.

DRUGS FOR RARE DISEASES
Patients with rare diseases generally have few treatment options, resulting in unmet clinical need. Accordingly, treatments are in high demand and can be high-cost. Treatments for rare diseases are often referred to as orphan drugs, or expensive drugs for rare diseases, or drugs for rare diseases.

High prices are often attributed to factors such as the high cost of research, limited number of patients, small market size, and lack of competitors.

The Government of Canada is working with Provinces and Territories as an active member of the pan-Canadian Pharmaceutical Alliance (pCPA) to combine the governments’ collective buying power to negotiate lower prices on brand name drugs for all public plans, including drugs for rare diseases.

ACCESS TO DRUGS FOR RARE DISEASES
Currently, Canadians with rare diseases can access the drugs they need through government drug plans, through private drug plans, or by paying out of pocket. Because these drugs are so expensive, patients generally cannot afford to pay by themselves.

Canadians have been able to gain access to drugs for rare diseases through participation in clinical trials, or as drugs are approved under Division 8 of the Food and Drug Regulations. In cases where criteria are met, Health Canada’s Special Access Program (SAP) considers requests for access to drugs that are unavailable for sale in Canada. About 150 of the drugs accessed through the SAP are for the treatment of rare diseases.

LAUNCH OF THE FIRST PHASE OF THE NATIONAL STRATEGY FOR DRUGS FOR RARE DISEASES
The Minister of Health announced the launch of the first phase of the National Strategy for Drugs for Rare Diseases on March 22, 2023, with an investment of up to $1.5 billion over three years, to improve access to treatments for people living with a rare disease.

As part of this first phase, the Government of Canada will provide up to $1.4 billion over three years to provinces and territories through bilateral agreements. This federal investment will increase access to safe and effective drugs for Canadians with rare diseases. Provinces and territories will be able to add new drugs to their formularies and increase coverage of existing drugs. The new funding will also enable provinces and territories to improve screening and diagnostics so that patients with a rare disease have a better chance of getting access to effective treatments at the right time, which can mean significantly better health and overall quality of life for patients and their families.

In addition, the Government of Canada is providing up to $33 million over three years to Indigenous Services Canada’s Non-Insured Health Benefits Program to support eligible First Nations and Inuit clients with rare diseases.

The Government of Canada is also making key investments of up to $68 million to support collaborative governance, data infrastructure, and research for drugs for rare diseases. $20 million over three years is being provided to the Canadian Agency for Drugs and Technologies in Health and the Canadian Institute for Health Information to improve the collection and use of evidence to support decision-making. The Canadian Institutes of Health Research are receiving $32 million over five years to advance a rare disease research agenda with a focus on developing better diagnostic tools and establishing a robust Canadian rare disease clinical trials network. $16 million over three years, starting in 2023, is being invested to support the establishment of national governance structures, such as a Health Canada secretariat and a stakeholder Implementation Advisory Group.

The Government of Canada will continue working with provinces and territories, health system partners, and stakeholders, including patients, families, and clinicians, to develop future actions based on lessons learned as part of these actions included in the first phase of the Strategy.

NATIONAL PHARMACARE LEGISLATION
The 2019 Final Report of the Advisory Council on the Implementation of National Pharmacare recommended for the Canadian government to develop a formal National Strategy for Drugs for Rare Diseases.

Budget 2019 announced federal investments to move forward on a number of foundational elements of national pharmacare, including the creation of the National Strategy, as well as the development of a national formulary, and the creation of the Canadian Drug Agency.

The National Strategy is intended to be part of continuing progress towards a national universal pharmacare program. It continues to be a crucial part of the government’s commitment to long-term funding to improve the accessibility and affordability of drugs.

Bill C-64, An Act respecting pharmacare, commits to maintaining long-term funding for provinces, territories and Indigenous Peoples to improve the accessibility and affordability of pharmaceutical products, beginning with the funding announced for the National Strategy for Drugs for Rare Diseases.

• Rare diseases are life-threatening, seriously debilitating and sometimes chronic in nature. They are often genetic conditions, with onset either at birth or early childhood.
• Currently, Canadians with rare diseases access the drugs they need through government drug plans, through private drug plans, or by paying out of pocket. Many drugs for rare diseases are very expensive and patients generally cannot afford to pay by themselves.
• The 2019 Final Report of the Advisory Council on the Implementation of National Pharmacare recommended for the Canadian government to develop a formal National Strategy for Drugs for Rare Diseases.
• Bill C-64, An Act respecting pharmacare, commits to maintaining long-term funding for provinces, territories and Indigenous Peoples to improve the accessibility and affordability of pharmaceutical products, beginning with the funding announced for the National Strategy for Drugs for Rare Diseases.