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.

In March 2023, the Government of Canada announced the launch 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 now working with provinces and territories towards the development of bilateral agreements, starting with jointly determining a small set of new and emerging drugs that would be cost-shared and covered in a consistent way across the country.
This work will help increase access to, and affordability of, effective drugs for rare diseases to improve the health of patients across Canada.
IF PRESSED ON DETAILS OF THE IMPLEMENTATION, SUCH AS THE COMMON SET OF DRD, STAKEHOLDER CONSULTATION THROUGH THE IMPLEMENTATION ADVISORY GROUP, AND PROGRESS ON BILATERAL AGREEMENTS …
Health Canada officials are now engaging with provinces and territories toward the development of bilateral agreements. Similarly, funding will go to Indigenous Services Canada to support eligible First Nations and Inuit patients living with rare diseases.
An Implementation Advisory Group will also be launched soon to ensure that patients and other stakeholders remain at the centre of the Strategy.
We are also working with our health system partners to advance various initiatives focused on collection and use of evidence to support decision making and the advancement of rare disease research.
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, while also providing flexibility for jurisdictions to address their own unique circumstances.
All provinces and territories have been invited to engage in the next critical step of jointly determining a small set of new and emerging drugs for rare diseases, and to share in the benefits of collaborative approaches to the collection and use of evidence in rare diseases, and innovation and research.
IF PRESSED ON FUNDING TO HEALTH SYSTEM PARTNERS …
Our Government will be providing $20M over three years to the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Canadian Institute for Health Information (CIHI) to support the use of evidence in decision-making.
This work will improve the collection and use of real-world evidence to support decision-making, as well as patient registries.
An additional $32 million over five years to the Canadian Institutes of Health Research will advance a rare disease research agenda, developing better diagnostic tools and establishing a robust Canadian rare disease clinical trials network.
IF PRESSED ON WHEN PATIENTS WILL SEE BENEFITS FROM THE STRATEGY …
As a critical next step to advance the development of these bilateral agreements, the Government of Canada is engaging 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.
Our work with partners is also well underway; for example with the 32 million dollars provided to the Canadian Institutes of Health Research (CIHR) through the Strategy to advance rare disease research, CIHR has already launched four funding opportunities working to strengthen patient support services, including one to support clinical trial networks in pediatric 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. The pharmaceutical 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, at McGill University 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—many of whom are children. 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 $69 million to support collaborative governance, data infrastructure, and research for drugs for rare diseases. $20 million over three years will be 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 will receive $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, will be 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.

Rare diseases are life-threatening, seriously debilitating and sometimes chronic in nature.
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 but there are thousands of different rare diseases affecting people the country.
It is estimated that one out of twelve Canadians has a rare disease. They are often genetic conditions, with onset either at birth or early childhood.
Innovative treatments for rare disease can cost anywhere from $100,000 to more than $2 million per year. Sales of expensive drugs for rare diseases grew on average nearly 32% annually from 2011 to 2020, making this a key cost driver of pharmaceutical spending in Canada.