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 command high prices, which pose significant challenges to patients, caregivers, and the health care system, including the sustainability of public and private drug plans that pay for these drugs.
• Nearly 75% of rare diseases present from early in utero to late childhood, and nearly 95% of the rare diseases affecting children are currently without treatment. It is estimated that one in 15 children are born with a rare disease, and many of these children die before their first birthday: rare diseases account for 30-40% of neonatal deaths in Canada. One in four pediatric hospital beds are currently being occupied by a child with a rare disease.

• We recognize that for many Canadians who require prescription drugs to treat rare diseases, the cost of these medications can be astronomically high.
• To help Canadians get better access to effective treatments, we are working with provinces, territories and other partners willing to move forward to develop a national strategy for drugs for rare diseases to be launched in 2022.
• Budget 2019 proposed to invest up to $1 billion over two years, starting in 2022-2023, with up to $500 million per year ongoing, to help Canadians with rare diseases access the drugs they need.

IF PRESSED ON COLLABORATION WITH STAKEHOLDERS…
• We held a virtual public and stakeholder engagement in early 2021, which concluded on March 26th, 2021. Canadians - especially patients with rare diseases, their families and their parents and caregivers - were invited to provide their views on the national strategy by participating in a public town hall or completing the online questionnaire. A “What We Heard” report summarizing key themes and feedback that emerged during the public and stakeholder engagement has been published on the engagement webpage.
• Building on what we heard from stakeholders and partners thus far and recognizing the importance of ongoing engagement, a second phase of targeted engagement comprising stakeholder roundtables is taking place over April and May 2022 to seek feedback on a draft framework for the strategy and further inform the development of the national strategy.
• Productive discussions are also ongoing with provincial and territorial counterparts.

IF PRESSED ON STATUS OF THE NATIONAL STRATEGY…
• Despite the pandemic, our Government recognizes the pressing need to improve access and affordability of treatments for rare diseases.
• In collaboration with willing provinces, territories and other partners, we continue to work toward delivering a national strategy for launch in 2022.
• Based on input to date, Health Canada has now developed a draft framework for the national strategy for drugs for rare diseases.
• Building on what we heard from stakeholders and partners thus far and recognizing the importance of ongoing engagement, Health Canada is hosting targeted roundtables with stakeholders throughout April and May 2022 to help finalize the national strategy.
• The Government of Canada is working to launch the national strategy for drugs for rare diseases in 2022 with the support of all those involved.

IF PRESSED ON MORE DETAILS ON THE DRAFT FRAMEWORK FOR THE NATIONAL STRATEGY …
• Based on what we have heard from stakeholders and partners to date through previous engagement, Health Canada has developed a draft framework for the national strategy.
• The draft framework is comprised of four pillars and eight principles, as well as potential activities that could be part of each pillar.
• Health Canada is now holding targeted consultations to seek feedback on the draft framework, explore how stakeholders could contribute to achieving the strategy objectives, discuss implementation considerations, and understand stakeholder preferences for involvement and engagement in the implementation.
• The implementation of a strategy will provide people across Canada with access to drugs for rare diseases, while enabling federal, provincial, and territorial governments, stakeholders, and experts to work together to enhance coordination across Canada’s drugs for rare diseases landscape.

IF PRESSED ON MORE DETAIL RELATED TO THE CANADIAN ORGANIZATION FOR RARE DISORDERS’ (CORD) RESPONSE …
• The Canadian Organization for Rare Disorders (CORD) is a key stakeholder supporting Health Canada’s development of a national strategy for drugs for rare diseases.
• CORD has been very engaged with Health Canada on the development of the national strategy. They participated in the initial broad public and stakeholder engagement in early 2021, follow-up meetings in the summer of 2021 after the publication of the What We Heard Report, and the second phase of targeted engagement to provide feedback on the draft framework in spring of 2022.
• Health Canada is appreciative of CORD’s feedback and perspectives and looks forward to continuing to work with CORD throughout the development and implementation of the national strategy for drugs for rare diseases.

IF PRESSED ON THE SPECIAL ACCESS PROGRAM…
• Our Government recognizes the importance of Canadians having access to the treatments they need.
• Health Canada's Special Access Program considers requests for access to drugs that are unavailable for sale in Canada.

IF PRESSED ON RESEARCH….
• Our Government recognizes that supporting research is another important aspect of addressing rare diseases. Through the Canadian Institutes of Health Research (CIHR), Canada is playing an important role, both at the national and international levels, to tackle rare diseases.
• For example, Canada, through CIHR, is a founding member of the International Rare Diseases Research Consortium and is also engaged in E-Rare, the European Union’s main instrument for funding research in areas related to rare diseases.

IF PRESSED ON TRIKAFTA…
• Health Canada understands that patients with Cystic Fibrosis (CF) want faster access to new and promising drugs, particularly when limited treatment options are available.
• Recognizing the importance of this new treatment option for patients, following a priority review, on June 18, 2021, Health Canada authorized Trikafta for CF patients aged 12 years and older, with a gene mutation found in 90% of patients. It was approved in an aligned review with the Canadian Agency for Drugs and Technologies in Health (CADTH), which expedited the review and reimbursement recommendation process. Furthermore, on April 20, 2022, Health Canada approved Trikafta for children ages six to 11 with the same gene mutation.
• The pan-Canadian Pharmaceutical Alliance (pCPA) undertook negotiations to reach a mutual agreement with Vertex Pharmaceuticals for Trikafta and individual jurisdictions have begun to make decisions as to whether they will cover the drug under their drug plans.

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 command high prices. 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.

SPECIFIC CHALLENGES FOR CHILDREN WITH RARE DISEASES
The four main challenges that are exacerbated for Canadian children with rare diseases and their families are: (1) the inadequate receipt of paediatric data, particularly in comparison to counterparts in the US and Europe; (2) barriers to paediatric clinical trials; (3) economic challenges associated with developing, marketing, and maintaining a stable paediatric drug supply; and (4) current widespread use of off-label medications in children.

Economic, ethical, regulatory, and infrastructure considerations associated with the conduct of child health research have limited the numbers of clinical trials conducted with children. There are several challenges specific to conducting clinical trials with a pediatric population with rare conditions that have contributed to current gaps in the evidence base on medicines for children. These challenges include the difficulty to obtain a sufficient sample size to design and execute a well-powered clinical trial, and the lack of special methodological and analytic expertise required to conduct innovative and adaptive study types.

Despite these challenges, there is growing recognition of the undesired consequences of the vast gap in knowledge, such as a lack of information to guide dosing, inappropriate formulations, less than optimal clinical and patient-oriented outcomes, and issues related to transitioning into adulthood.

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 26th, 2021. The perspectives gathered from this process are helping to inform the design of the national strategy.

Two specific meetings with pediatric stakeholders including clinicians and researchers were held in April 2021, and a follow-up meeting to discuss next steps took place in June 2021. Pediatric stakeholders also reached out to Health Canada March 2022 to submit and discuss their proposal for the national strategy for drugs for rare diseases.

Over the summer, 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 to date, a draft framework for the national strategy was developed. Further targeted consultations to seek feedback on this draft framework and further inform a comprehensive model for the national strategy are taking place throughout April and May 2022. Pediatric stakeholders have been invited to the clinicians roundtable as well as the researchers roundtable. With the support of all those involved, the Government of Canada will work towards launching the national strategy for drugs for rare diseases in 2022.

THE CANADIAN ORGANIZATION FOR RARE DISORDER’S (CORD) RESPONSE TO THE DRAFT FRAMEWORK FOR THE NATIONAL STRATEGY
CORD recently published a public response to the draft framework. They validated the stakeholder views presented in the What We Heard Report, but they feel the draft framework uses vague language and lacks meaningful commitment to actions or timelines. CORD was underwhelmed by the strategy’s vision and principles presented in the draft framework. They also wanted more clarity around what the advisory structures could look like and how they would function as part of the strategy. CORD also proposed providing drug coverage for new transformative life-saving therapies, rather than focusing on an initial list of drugs of common concern.

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 2021-2022, 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 2021-2022, 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 2021-2022 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 2022-2023, 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.

KEY FACTS
• Budget 2019 proposed to invest up to $1 billion over two years, starting in 2022-23, with up to $500 million per year ongoing. As reaffirmed in the 2020 Fall Economic Statement and Speech from the Throne, as well as Budget 2021, this includes working with provinces, territories and stakeholders to establish a national strategy for drugs for rare diseases for launch in 2022.