Question Period Note: Drugs for Rare Diseases

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• 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.
KEY MESSAGES
• 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 now working with provinces and territories on 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.
• Together, with investments in Indigenous Services Canada’s Non-Insured Health Benefits Program, these bilateral agreements will help increase access to, and affordability of, effective drugs for rare diseases to improve the health of patients across Canada, including children.
• The bilateral agreements with provinces and territories will also support enhancements in early diagnosis and screening, and other elements of the Strategy will support improvements in the collection and use of evidence, and critical research and innovation in rare diseases.
• We will also establish the Implementation Advisory Group by the summer to support the implementation of the Strategy.
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…
• Our Government is working with provinces and territories to achieve outcomes together that build national consistency for patients, while also providing flexibility to address their own circumstances.
• Health Canada officials are now engaging with provinces and territories on the development of bilateral agreements, beginning 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, for the benefit of patients. Similarly, funding will go to Indigenous Services Canada's Non-Insured Health Benefits Program to support eligible First Nations and Inuit patients living with rare diseases.
• An Implementation Advisory Group will also be launched by this summer to ensure that patients and clinicians, public and private drug plans, the pharmaceutical industry and others remain at the centre of the Strategy.
• We are also working with our other 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 advance various initiatives focused on collection and use of evidence to support decision making and the advancement of rare disease 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. This improved information about the safety and effectiveness of drugs for rare diseases will support decision-makers and clinicians, which will in turn benefit patients.
• 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 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 in the next critical step of jointly determining a small set of new and emerging drugs that would be cost-shared and covered in a consistent way across the country, 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 WHEN PATIENTS WILL SEE BENEFITS FROM THE STRATEGY
• The investment that I announced on March 22 will help increase access to, and affordability of, effective drugs for rare diseases to improve the health of patients across Canada, including children.
• 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.
• The multi-stakeholder Implementation Advisory Group (IAG) announced on March 22 will be established this summer to support implementation of the Strategy.
• 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 issued two notices of upcoming funding opportunities, for a National Pediatric Rare Disease Clinical Trials and Treatment Network and Bringing Rare Disease Gene Therapies to Clinical Trial Readiness. These opportunities are anticipated to launch this summer.

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.

Patients generally have reduced quality of life and shortened life span and are high-end users of the public health care system.

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
Having timely access to safe and effective treatments can significantly improve the health and overall quality of life of a person with a rare disease.

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.

IMPACT OF COVID-19 ON DRUGS FOR RARE DISEASES
Despite the pandemic, attention continues to be focused on drugs for rare diseases, including on new drugs for specific patient cases, and general calls emphasizing the need for a national strategy.

To date, private drug plans have reported minimal changes to the coverage status of patients taking drugs for rare diseases, but it is unknown if this will change over time.

STAKEHOLDER ENGAGEMENT ON DRUGS FOR RARE DISEASES
There is broad consensus amongst political parties, PT governments, private insurers and other health system partners for federal funding for drugs for rare diseases.

Reaching an agreement on the scope/details of the strategy – and the balance of access vs. affordability vs. appropriate use – will require a comprehensive engagement approach. This will also help manage expectations around the limits of federal funding.

In December 2020, federal, provincial and territorial (FPT) Ministers of Health mandated their officials to establish a FPT ADM-level group to guide the development and implementation of the national strategy.

A comprehensive engagement process commenced in late 2020 and continued through early 2021, starting with PTs, health technology assessment organizations, the pan-Canadian Pharmaceutical Alliance and national Indigenous health partners. Key stakeholders include patients and patient groups, clinicians, academics and researchers, private payers, and pharmaceutical manufacturers. Canadians were also invited to provide their views through virtual public town halls and an online questionnaire, which concluded on March 26, 2021. The perspectives gathered from this process are helping to inform the design of the national strategy.

Over the summer 2021, as a follow up to the What We Heard Report, Health Canada continued discussions with patients and caregivers, and other stakeholders and partners to build a strategy that will work for all Canadians. Based on input received, a draft framework for the national strategy was developed in spring of 2022. Further targeted consultations to seek feedback on this draft framework and further inform a comprehensive model for the national strategy took place throughout April and May 2022, and the Minister of Health hosted three stakeholder roundtables on pharmaceutical initiatives, including drugs for rare diseases. This extensive engagement helped to inform the government’s approach to developing and implementing the National Strategy for Drugs for 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.

Minister Dubé wrote to you on April 18, 2023, to request Quebec’s share of the funding, without conditions, for the National Strategy for Drugs for Rare Diseases. Quebec has not participated in the Drugs for Rare Diseases process since stating in December 2020 that they have, and intend to keep, their own drug insurance plan.

INVESTMENT IN RESEARCH
Through the Canadian Institutes of Health Research (CIHR), the Government is supporting research on rare diseases and has taken a leadership role by joining important international research initiatives, such as the International Rare Disease Research Consortium, which aims to accelerate medical breakthroughs for people affected by rare diseases and includes 58 organizations from 22 countries. As of 2017, this international Consortium has yielded 279 new medicinal products and therapies for rare diseases. Budget 2021 also proposed to provide $250 million over three years, starting in 2021-22, for the CIHR to implement a new Clinical Trials Fund to increase clinical research capacity.

Additionally, Budget 2021 proposed to provide $400 million over six years, starting in 2021-22, in support of a Pan-Canadian Genomics Strategy. This funding would provide $136.7 million over five years, starting in 2022-23, for mission-driven programming delivered by Genome Canada to kick-start the new Strategy and complement the government’s existing genomics research and innovation programming.

Budget 2021 proposed to provide a total $2.2 billion over seven years towards growing a vibrant domestic life sciences sector. This support would provide foundational investments to help build Canada’s talent pipeline and research systems, and support the growth of Canadian life sciences firms, including: $500 million over four years, starting in 2021-22 for the Canada Foundation for Innovation to support the bio-science capital and infrastructure needs of post-secondary institutions and research hospitals, and $45 million over three years, starting in 2022-23, for the Stem Cell Network to support stem cell and regenerative medicine research. These various initiatives have potential linkages to research and innovation for drugs for rare diseases.

To build on these past investments the National Strategy for Drugs for Rare Diseases, announced on March 22, 2023, will provide $32 million over five years to CIHR to further advance a rare disease research agenda with a focus on developing better diagnostic tools and establishing a robust Canadian rare disease clinical trials network. This research will help inform better clinical care for all patients with rare diseases.

• On March 22, 2023, the Government of Canada announced the launch of the first-ever National Strategy for Drugs for Rare Diseases, supported by investments of up to $1.5 billion over three years. This first phase aims to increase access to and affordability of effective drugs for rare diseases, which will contribute to improving the health of patients across Canada.
• As part of the overall $1.5 billion investment, the federal government will make available up to $1.4 billion over three years to provinces and territories to improve access to new and emerging drugs, as well as to support enhanced access to existing drugs, early diagnosis, and screening for rare diseases.
• Similarly, to ensure that eligible First Nations and Inuit clients with rare diseases will also benefit, the Government is providing up to $33 million over the first three years to Indigenous Services Canada’s Non-Insured Health Benefits Program.
• The Federal Government is also making key investments of a total of up to $68 million for various initiatives to help improve consistent access to drugs for rare diseases across the country.