Government – Muscular Dystrophy Canada

Funding for first-ever National Strategy for Drugs for Rare Diseases announced

FOR IMMEDIATE RELEASE March 22, 2023

Toronto, Ontario – Muscular Dystrophy Canada applauds the Government of Canada for committing $1.5 billion over three years in support of a National Strategy for Drugs for Rare Diseases to help increase access to, and affordability of, promising and effective drugs for rare diseases to improve the health of patients across Canada.

“MDC works with Canadian Fire Fighters, donors, volunteers and other like-minded organizations to break down barriers for the neuromuscular community. The major barriers that exist for our diverse community, which consists of individuals with rare and ‘ultra-rare’ neuromuscular disorders, are lack of cures and delayed – out of reach treatments. So this announcement is great news for our community,” said Stacey Lintern, CEO, Muscular Dystrophy Canada. She added, “we are excited that this announcement has been made but time is of the essence for many rare diseases and the sooner we can move this from strategy to action, the better it is for our community.”

This long-awaited announcement identified four areas of focus for the national strategy and funding support for provinces and territories to improve access to new and emerging drugs, enhance access to existing drugs, early diagnosis, and screening for rare diseases.

“The areas of focus align perfectly with the work that MDC is already doing such as ensuring early diagnosis and clinical genetic testing. We know access to an accurate diagnosis, coupled with early access to treatments, are important for optimal outcomes and better quality of life,” said Lintern.

“MDC welcomes a strategy where the patient and family are at the forefront and that supports an evidence-based care journey. We are committed to working with our partners like the Neuromuscular Disease Network for Canada (NMD4C) to help make that happen, and bring about better diagnostic tools, support for clinical trials and evidence that supports decision-making. We look forward to a time when the neuromuscular community has access to accurate and early diagnoses, life-changing treatments, and supports regardless of where they live, their age or the rarity of the condition.”

The full Government of Canada announcement can be read here

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ABOUT MUSCULAR DYSTROPHY CANADA

Muscular Dystrophy Canada’s mission is to enhance the lives of persons affected by neuromuscular disorders by continually working to provide ongoing support and resources while relentlessly searching for cures through well-funded research. To learn more about MDC, please visit muscle.ca or call our toll-free number at 1-800-567-2873.

FOR MORE INFORMATION CONTACT:

Heather Rice
Muscular Dystrophy Canada
Heather.Rice@muscle.ca
902-440-3714

Muscular Dystrophy Canada and Independent Living Canada partner to help lead the development of Canada’s first ever Disability Inclusion Action Plan

FOR IMMEDIATE RELEASE

Toronto, ON – January 27, 2021 – Muscular Dystrophy Canada (MDC) and Independent Living Canada (IL Canada) are partnering to equitably consult with individuals, organizations and stakeholders across disabilities to help inform the development of Canada’s first Disability Inclusion Action Plan (DIAP).

Beginning in January 2022, this 15-month collaboration will both inform the development of the DIAP in advance of its release, and continue to assess and improve the Plan throughout its implementation. The DIAP will focus on reducing poverty among persons with disabilities, getting more persons with disabilities employment opportunities, and realizing the goal of the Accessible Canada Act to achieve a barrier-free Canada by 2040, making it easier for persons with disabilities to access federal programs and services and fostering a culture of inclusion. Our goal is to ensure all Canadians have the opportunity to provide feedback, express concerns, and share their experiences to make this vital action plan as inclusive and impactful as possible.

With over 100 disability organizations invited to collaborate on this engagement process, IL Canada and MDC were selected to co-lead this project with support and funding from Employment and Social Development Canada (ESDC).

“For The Disability Action Plan to make a real impact in the lives of persons with disabilities, it has to be informed by lived experience. I’m grateful that Muscular Dystrophy Canada and Independent Living Canada will be working with the disability community to determine what barriers are still preventing persons with disabilities from participating in society, and how Canada can remove them to create a more equal, inclusive, and prosperous country” says The Honourable Carla Qualtrough, Minister of Employment, Workforce Development and Disability Inclusion.

IL Canada and MDC look forward to leading this process while ensuring representation from within all areas of disability, including racialized and LGBTQ2S+ communities.

“Independent Living Canada is thrilled to be leading this consultation process in partnership with Muscular Dystrophy Canada, the pillar leads, and individuals affected by disabilities from coast-to-coast to coast. This is a once-in-a-lifetime opportunity to ensure that the voices of marginalized and hard-to-reach people affected by disabilities are provided the opportunity to have their voices heard about what should be included in the first-ever federal Disability Inclusion Action Plan,” says Anne MacRae, National Executive Director, Independent Living Canada.

“Muscular Dystrophy Canada is honoured to work in partnership with IL Canada to consult Canadians affected by disabilities and share these experiences in a method that will drive the implementation of the much needed Disability Inclusion Action Plan,” says Stacey Lintern, CEO, Muscular Dystrophy Canada. “This is a vital action plan which will impact each and every Canadian in some way. We look forward to expanding our person-centred approach and ensuring all voices are heard throughout this process.”

MDC and IL Canada look forward to working with Inclusion Canada, Canadian Council on Rehabilitation and Work, Canadian Association of the Deaf, and Eviance who have been designated as leads for the four respective thematic pillars of the DIAP: Financial Security, Employment, Accessible and Inclusive Communities and a Modern Approach to Disability.

“At Inclusion Canada we are extremely excited about hearing from persons with a disability, their representative organizations and stakeholders across the country as the lead on the financial security pillar of the Disability Inclusion Action Plan,” says Krista Carr, Executive Vice-President, Inclusion Canada. “Hundreds of thousands of people with disabilities in Canada live in poverty and this urgently needs to change. We are looking forward to getting started right away.”

“We are pleased and excited at the funding support for this project. We really look forward to working with our partner organizations in building new bridges to hard-to-reach components of the Deaf, Deaf-blind and disabled communities across Canada,” says Jim Roots, Executive Director, Canadian Association of the Deaf.

“The Canadian Council on Rehabilitation and Work (CCRW) recognizes the capability of persons with disabilities, and supports their choices for equitable and meaningful participation in work. We are proud to be a pillar of change and build up the community by sharing our values, knowledge and commitment to Disability Inclusion and ensuring that there is an equitable approach to disability inclusive design,” says Maureen Haan, President and CEO, CCRW. “We are honoured to be involved in the development of the DIAP as the lead agency of the Employment Pillar. We are excited and motived to ensure this project will bring change and a voice to those with lived experience that remain hard-to-reach and marginalized populations with disabilities.”

“Eviance looks forward to collaborating on this important piece of work, and bringing together the knowledge and diverse experiences of persons with disabilities, to strengthen the federal Disability Inclusion Action Plan,” says Dr. Susan L. Hardie, PhD, Executive Director, Eviance.

About Muscular Dystrophy Canada

Muscular Dystrophy Canada’s mission is to enhance the lives of those affected with neuromuscular disorders by continually working to provide ongoing support and resources while relentlessly searching for a cure through well-funded research. To learn more about Muscular Dystrophy Canada, please explore our website at muscle.ca or call our toll-free number at 1-800-567-2873.

About Independent Living (IL) Canada

Founded in 1986, Independent Living Canada is a national non-profit association of Independent Living Centres led by and for people with disabilities who promote the Independent Living Philosophy. IL Canada supports the development of Independent Living Centres, and provides education and leadership for its member organizations, partners and communities.

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Media Contact

Heather Rice
Director, Marketing and Communications
Muscular Dystrophy Canada
heather.rice@muscle.ca
902-440-3714

Response to PMPRB Draft Guidelines Consultation

February 14, 2020

Dr. Mitchell Levine
Chairperson
Patented Medicines Prices Review Board
333 Laurier Avenue West, Suite 1400 Ottawa, Ontario K1P 1C1
Email: PMPRB.Consultations.CEPMB@pmprb-cepmb.gc.ca

Dear Dr. Levine:

Muscular Dystrophy Canada is thankful for the invitation to attend the PMPRB Consultation meeting in Ottawa on December 10, 2019 and for the opportunity to respond to the Draft Guidelines Consultation.

Muscular Dystrophy Canada leads Canada’s neuromuscular community as the central axis in a complex health services, policy, and information ecosystem that surrounds and support our core concern: Canadians with neuromuscular disorders. Muscular Dystrophy is the umbrella name used to cover the 160-plus known neuromuscular disorders today. While the disorders we represent are individually rare, they are collectively common, impacting more than 50,000 Canadians from coast to coast. These disorders weaken the body’s muscles, or the communication between the nerves and the muscles. The causes, symptoms, age of onset, severity and progression vary depending on the individual and the exact diagnosis. For many individuals, the disorder is fatal. Currently, there is no cure for these disorders. We believe that neuromuscular disorders will one day be cured (with treatments and therapies), and that our organization will be a positive contributor to that outcome by: investing in innovative research that has potential for real-world impact; providing programs and support services for those impacted by neuromuscular disorders, their caregivers and families, health professionals and researchers; and remaining committed to advocating for fair and patient-centric policies that improve the lives of all Canadians with neuromuscular disorders.

Currently, the range of treatments and therapies available to support impacted individuals can come with a heavy financial burden and a significant economic impact. As an organization, we are passionate in the belief that every Canadian with a neuromuscular disorder deserves to benefit from leading-edge discoveries in the research field in a timely manner and equal access to the best treatments. Muscular Dystrophy Canada together with members of its Medical and Scientific Advisory Committee are of the strong belief that, if PMPRB guidelines are enacted as proposed, the amendments may result in unfavorable consequences as described below:

Significant decreases in price will result in delays in manufacturers launching their product in Canada and this will have a negative impact on the overall length of time that it takes for Canadians to have access to new medicines in Canada, if at all. We are seriously concerned that Canadians with neuromuscular disorders will, in fact, see a decline in innovative medicines access. Members of our Medical and Scientific Committee Dr. James Dowling (Hospital for Sick Children, Toronto), Dr. Ronald D. Cohn (Hospital for Sick Children, Toronto), and Dr. Craig Campbell (Children’s Hospital London Health Sciences Centre, London) together published an article in April 2018 in the Journal of Medical Genetics that emphasizes: “many exciting treatment approaches are currently in clinical trial, and several have achieved conditional or full market approval in various regions of the world. Many other treatments are in the pipeline, and we predict that over the next decade meaningful therapies will become widespread across the neuromuscular disease spectrum.”

In addition to compromising timely access to new therapies for patients with neuromuscular disorders, Muscular Dystrophy Canada is profoundly concerned about the impact the pricing changes will have on the health research infrastructure of Canada. Although PMPRB confidently notes that pricing is not a significant determinant to bringing clinical research to Canada, the Life Sciences Ontario (2019) survey says otherwise. In that survey, 91% of pharmaceutical executives said the changes would have a negative effect on clinical research in Canada, with 44% saying the negative effect would be “significant.” There will be significant adverse impact of the proposed changes on research incentives and investments, which is important as we are on the verge of life-changing therapies and treatments for individuals with neuromuscular disorders.

While we support the efforts to lower the costs of prescription drugs for Canadians, we strongly believe that this must be done in a way that ensures timely access by Canadians to new medicines and to clinical studies of new medicines. Our recommendations are in line with those proposed by Health Charities Coalition Canada (HCCC) and we strongly echo the discussion outlined in their thorough and carefully well thought-out submission. We suggest you carefully review our submissions and take into account these four key recommendations:

Recommendation #1:

That the PMPRB undertake a stepwise approach to its proposed changes by initially enacting only the changes to the comparator countries. Once the impact of this change is fully understood and if the objective of lowering Canadian prices sufficiently has not been met, then other new elements could be considered.

Recommendation #2:

That a multi-stakeholder dialogue be established to evaluate the impact of the changes on availability of medicines and specifically to inform any decision on whether and how to implement the use of the new economic criteria.

Recommendation #3:

That the Federal Government require PMPRB to hire a third party to conduct a formal assessment of the potential and real-time impacts of the reforms on research investment and activity in Canada (including clinical trials).

Recommendation #4:

That the Federal Government require that PMPRB, along with other appropriate agencies, immediately establish a formal mechanism for meaningfully and continuously engaging patient representatives in its decision-making and processes to ensure patient voice, choice and representation.

Thank you for the opportunity to review the guidelines and provide comments. We remain committed to working with the Federal Government and the broader stakeholder community in defining an implementation and evaluation process that will address both cost savings and access

to innovation that will optimally serve the needs of individuals with neuromuscular disorders in Canada. We wish our feedback will be taken into consideration as you prepare the final guidelines and we would be happy to answer any questions you might have.

Kind Regards,
Muscular Dystrophy Canada

Alberta Fourth Province to Expand Access to Spinraza™ for Patients Impacted with Spinal Muscular Atrophy

Alberta fourth province to expand access to SPINRAZA™ for patients impacted with Spinal Muscular Atrophy.

Muscular Dystrophy Canada (MDC) commends the Government of Alberta for joining Quebec, Saskatchewan, and Ontario in expanding access to SPINRAZA™, a life-changing treatment for individuals impacted with Spinal Muscular Atrophy (SMA).

In Alberta, the following patients will now be eligible for reimbursement of SPINRAZA™, in addition to Type 1 patients:

patients who are pre-symptomatic with two or three copies of SMN2, or
have had disease duration of less than six months, two copies of SMN2, and symptom onset the first week after birth and on or before seven months of age, or
are under the age of 18 with symptom onset after six months of age, regardless of the ability to walk.
Other patients who do not meet the expanded funding criteria may be considered in exceptional cases.

More Patients Impacted with Spinal Muscular Atrophy in Ontario to Gain Access to Spinraza™

Toronto, Ontario – Muscular Dystrophy Canada (MDC) applauds the Government of Ontario for expanding access to SPINRAZA™, a life-changing treatment for individuals impacted with Spinal Muscular Atrophy (SMA). In Ontario, expanded coverage of SPINRAZA™ will include the following, in addition to existing coverage for Type 1 patients:

patients who are pre-symptomatic with two or three copies of the SMN2 gene;
patients with a disease duration of less than six months, two copies of the SMN2 gene, and symptom onset the first week after birth and on or before seven months of age;
patients under the age of 18, with symptom onset after six months of age and who have never achieved the ability to walk independently.

Patients Living with Spinal Muscular Atrophy (SMA) in the Province of Saskatchewan Gain Access to Spinraza™

April 22, 2019

Dear members of the SMA community,

In response to the requests received by patients living with Spinal Muscular Atrophy (SMA), Biogen Canada would like to provide this latest progress regarding coverage of SPINRAZA™ (nusinersen) in the province of Saskatchewan.

Biogen Canada is delighted to inform you that on April 12, 2019, the Government of Saskatchewan informed treating physicians in their province that patients living with Spinal Muscular Atrophy (SMA) have access to SPINRAZA™. The Saskatchewan Ministry of Health has made the decision to expand coverage of SPINRAZA™ to include the following, in addition to existing Type I patients:

Patients who are pre-symptomatic with two or three copies of the SMN2 gene;
Patients up to age 18, with symptom onset after six months of age and who have never achieved the ability to walk independently;
Patients that may have achieved the ability to walk independently (type III) as well as type II and type III patients over the age of 18 are encouraged to talk to their treating physician to apply for a case by case coverage.

Federal Government Commits to National Strategy to Improve Access to Rare Disease Drugs

Toronto, Ontario – Muscular Dystrophy Canada commends the Government of Canada for committing $35 million, in its 2019 budget, to develop a Canadian Drug Agency to oversee the development of a new national formulary of prescribed drugs and a strategy to provide support for Canadians with rare diseases.

A national strategy to alleviate the high-cost of drugs for rare diseases will ensure more Canadians are able to access effective, life-changing treatments.

“This is an important first-step towards ensuring individuals diagnosed with a rare disease, such as a neuromuscular disorder, are able to access treatment without cost as a barrier,” said Barbara Stead-Coyle, CEO, Muscular Dystrophy Canada. “We call on all Federal parties to focus on this very real issue facing Canadians.”