Research News – Cure SMA Canada

Government of Canada improves access to affordable and effective drugs for rare diseases

ebesso — Mon, 27 Mar 2023 21:20:07 +0000

Government of Canada improves access to affordable and effective drugs for rare diseases

In Canada, one out of twelve people—many of whom are children—has a rare disease. There are thousands of different rare diseases that affect patients, their families and caregivers across the country. Unfortunately, treatments are only available for a small percentage of these conditions. Even when treatments that might dramatically improve the quality of a patient’s life are available, they are often unaffordable and out of reach.

Today, the Honourable Jean-Yves Duclos, Minister of Health, 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. Through this, we will help increase access to, and affordability of, effective drugs for rare diseases to improve the health of patients across Canada, including children.

As part of this overall investment, the Government of Canada will make available up to $1.4 billion to provinces and territories through bilateral agreements. This funding will help provinces and territories improve access to new and emerging drugs, as well as support enhanced access to existing drugs, early diagnosis, and screening for rare diseases. This will help patients with rare diseases, including children, have access to treatments as early as possible, for better quality of life.

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. Similarly, to further support eligible First Nations and Inuit patients living with rare diseases, $33 million from this funding will go to Indigenous Services Canada’s Non-Insured Health Benefits Program.

In addition, we will also invest $68 million for various initiatives to help improve consistent access to drugs for rare diseases across the country, including:

$20 million over three years to the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Canadian Institute for Health Information (CIHI) to improve the collection and use of evidence to support decision-making;
$32 million over five years to the Canadian Institutes of Health Research (CIHR) to advance rare disease research with a focus on developing better diagnostic tools and establishing a robust Canadian rare disease clinical trials network; and,
$16 million over three years to support the establishment of national governance structures, such as a Health Canada secretariat and a stakeholder Implementation Advisory Group, to support the implementation of the Strategy.

Today’s announcement is the result of feedback from extensive consultation that garnered diverse perspectives from over 650 individuals and organizations, including patients with lived experience, family members, and caregivers. To create this Strategy, lived experiences were taken into careful consideration to ensure Canadians living with rare diseases have access to the drugs they need and receive better treatments and services, regardless of where they live. In addition, this Strategy will improve the collection and use of evidence and strengthen investments in critical research and innovation in rare diseases.

The Government of Canada will continue to work with provinces, territories, and key partners to deliver on these improvements so those living with rare diseases can have better health outcomes and access to cutting-edge treatment options.

Quotes

“Access and affordability should never be a barrier to care. Through this new Strategy, Canadians living with a rare disease will soon have improved screening and diagnostics, and better access to affordable and effective treatments they need, no matter where they live. We will keep working with provinces and territories and all relevant partners so those living with a rare disease, including children, have the best health outcome possible while their caregivers get the support they need and deserve.”

The Honourable Jean-Yves Duclos
Minister of Health

“As the Federal Government launches the National Strategy for Drugs for Rare Diseases, the Non-Insured Health Benefits Program (NIHB) at Indigenous Services Canada will receive new investments of $33 million. This investment will help ensure that First Nations and Inuit eligible for NIHB prescription coverage will have the support needed to better access drugs for rare diseases.”

The Honourable Patty Hajdu
Minister of Indigenous Services and Minister responsible for the Federal Economic Development Agency for Northern Ontario

“This investment will enable improved access to new and emerging drugs for Canadians with rare diseases, including children, as well as support to early diagnosis and screening. This is a first step in looking at drugs for rare diseases coverage from a national perspective – consistently ensuring that patients can access the drugs they need regardless of where they live in the country.”

Adam van Koeverden
Parliamentary Secretary to the Minister of Health and to the Minister of Sport

Quick facts

Innovative treatments for rare disease can cost anywhere from $100,000 to more than $2 million per year. Budget 2019 provided funding up to $1 billion over two years, with up to $500 million per year ongoing, to help Canadians with rare diseases access the drugs they need.
In July 2021, Health Canada released a What We Heard Report from national engagement on the National Strategy. The engagement garnered diverse perspectives from over 650 individuals and organizations, including patients with lived experience, family members, and caregivers.
In December 2022, Health Canada pre-published proposed amendments to the Food and Drug Regulations that would introduce regulatory flexibilities such as the option of a rolling review, which may facilitate earlier market access for certain drugs, including drugs for rare diseases. The proposal would also allow for terms and conditions for all drugs, enabling Health Canada to include obligations on the manufacturer to optimize the benefits and manage any risks and uncertainties associated with the drug. This would be particularly important in the cases of drugs for rare diseases where patient populations are small.
Additionally, Health Canada’s Pediatric Drug Action Plan aims to improve access to safe and effective medicine for children in Canada, including drugs for rare diseases, which tend to be more prevalent in the pediatric population.

SPACES

mollyocean — Mon, 15 Aug 2022 14:30:13 +0000

SPACES is a first-of-its-kind musical collaboration created for everyone with a disability by the SMA community. Each creative component of the program – song, music video and album art – was led by someone with SMA. It uses the universal language of music to elevate the many voices of this community and celebrate our individuality

Learn more

August is Spinal Muscular Atrophy (SMA) Awareness month!

mollyocean — Mon, 15 Aug 2022 14:13:43 +0000

August is Spinal Muscular Atrophy (SMA) Awareness month!

Cure SMA Canada spreads awareness throughout Canada to bring hope to a future where SMA is a curable disease.

This year, the CN Tower and the Montreal Tower will be lit up in purple and orange, the Cure SMA Canada colours, on August 19 from 6:30pm until 7:00am August 20, in honour of those living with SMA, and in precious memory of those we have lost. Together we are strong in our commitment to supporting families affected by Spinal Muscular Atrophy.

Untreated, SMA is a degenerative neuromuscular disease, causing weakness throughout the body. A person affected by SMA has normal cognitive ability. Lets stand strong together in remembrance of those we have lost to this disease and for those who still wait for access to treatment.

Cure SMA Canada is Canada’s national charity supporting families affected by SMA through funding critical research projects, advocacy and support initiatives such as camps, newly diagnosed packages and informational conferences. Cure SMA Canada provides Help for Today and Hope for Tomorrow.

Thank you to the Montreal Tower and the CN Tower management teams for supporting Cure SMA Canada to spread awareness for Spinal Muscular Atrophy.

You can view the CN Tower on the live cam https://www.cntower.ca/#earthcam

Better yet! Take a family photo in front of it!! We’d love to see it, bring family and friends!

Activist Sammy Cavallaro awarded Quebec National Assembly Medal of Honour

mollyocean — Mon, 18 Jul 2022 20:28:18 +0000

We are so so proud of the achievements of Sammy Cavallaro! Congratulations on your award Sammy! Thank you to the Cavallaro family for your dedication to improving life for people affected by SMA in Canada!

You teach us all to make a change where one is needed. Very deserving of the award, we are so proud of you!!

A young West Island activist and advocate for disabilities is being awarded the Medal of Honour of the National Assembly.Sammy Cavallaro, who suffers from Spinal Muscular Atrophy, has raised more than $2 million to find a cure for the disease. His advocacy work has lead to major changes for people who suffer from SMA in Quebec. Global’s Gloria Henriquez reports.

Cure SMA Canada Conference August 10-14th in Hope, BC.

mollyocean — Wed, 06 Jul 2022 20:42:06 +0000

Agenda

‘This disease will slowly rob me of everything’: Why are Canadians living with rare diseases waiting for treatment?

ebesso — Tue, 01 Mar 2022 16:53:20 +0000

COVID has proven we can quickly set up infrastructure without long-term clinical data. It’s time to do the same with rare diseases, says Durhane Wong-Rieger, president and CEO of Canadian Organization for Rare Disorders.
Vanessa Hrvatin,

Feb 28 2022

Sasha Kirkland, pictured with her husband, Mark and son Maks, lives with spinal muscular atrophy (SMA).

For Sasha Kirkland, it’s hard to look back over the last decade of her life and not feel discouraged. The 36-year-old lives with type 3 spinal muscular atrophy (SMA), a rare progressive disease that causes muscles to weaken over time. Over the last 10 years her condition has gotten progressively worse; she can no longer walk without assistance and was forced to leave her full-time employment.

But two years ago, there was a glimmer of hope. Alberta, where Kirkland lives, expanded access to the drug Spinraza — the first drug approved by Health Canada to treat SMA which slows progression of the disease — beyond just those living with type 1 SMA. Although she didn’t meet the new criteria, the province stated that patients not meeting the expanded funding criteria would be considered on a case-by-case basis.

To date, Kirkland’s neurologist has submitted an application for her to access Spinraza six times but each one has been denied.

“It feels like a blow to the chest,” says Kirkland. “It makes me feel like my life isn’t worth as much as other people’s, and that my ability to contribute to society isn’t as great, either.”

Kirkland is one of roughly three million Canadians living with a rare disease, many of whom can’t access treatment. There are several barriers for rare disease patients to access innovative therapies in Canada, including the price tag associated with these drugs, which ranges from $100,000 to more than $2 million per patient each year. This price is too steep for most people to pay out of pocket, leaving the onus on provincial and territorial governments or private insurers to cover the cost. If not, the only way patients can get access is through clinical trials, patient-support programs sponsored by pharmaceutical companies, or Health Canada’s special access program.

‘Rare disease drug strategy should focus on infrastructure’

This challenge in accessing treatment is what the federal government is looking to address in a national rare disease drug strategy , backed by a $1 billion proposed budget over two years starting in 2022. President and chief executive officer of the Canadian Organization for Rare Disorders (CORD) Durhane Wong-Rieger says the strategy must focus on building infrastructure that will make it possible for clinical trials to be done effectively and efficiently in Canada so more companies will be inclined to market their products here. Currently, only 60 per cent of treatments for rare diseases make it into Canada, and most get approved up to six years later than in the USA and Europe.

Another barrier is the health technology assessment a new drug goes through after receiving Health Canada approval, which looks at the drug’s value and overall effectiveness. Many drugs that treat rare diseases are tested in small clinical trials with high uncertainty in terms of which patients they work for and how well they work, so they don’t pass the assessment.

“This is where we think the biggest change will happen with this rare disease drug strategy,” says Wong-Rieger. “We need to approve these drugs, make them available now, and then have the infrastructure to monitor patients in real time to see how they’re working. The drugs are there and could make a profound difference in peoples’ lives, so we don’t need to wait until we have all the evidence or until we’ve done these big clinical trials.”

Wong-Rieger points to the roll-out of COVID-19 vaccines — which was coordinated quickly despite not being backed by years of clinical trial data — as exactly what the rare disease space has been advocating for.

“We got the vaccines and learned they were waning after four months just by studying patients in real time,” she says. “We’re not asking for money to be put toward drugs — that’s a given, just like having the money for vaccines was a given. We need the $1 billion to be invested in setting up clinics, communications and long-term monitoring, just like we’ve seen with COVID.”

“I can’t say things like, ‘our 10-year anniversary is coming up so let’s save money and go on a trip,’ because I don’t know what life is going to look like for me in 10 years,” says Sasha Kirkland.

Wong-Rieger says the rare disease drug strategy likely won’t consist of a fully fleshed out framework; it needs to be flexible as new infrastructure is put in place and more innovative therapies become available. And if Canada gets it right, she’s hopeful this strategy could act as a blueprint for other diseases.

“The idea here isn’t let’s just open the doors and make every drug available to everyone,” she says. “We want to make sure the drugs coming in are the right drugs and that they’re going to the right people. We need to have a process in place to ensure they’re coming in at prices and in circumstances that are affordable and sustainable in the long term.”

Despite the rare disease drug strategy being announced in 2019, CORD developed a broader strategy back in 2015 focused on many aspects of rare diseases, ranging from diagnosis to community support, but it never gained federal attention.

For Sasha Kirkland whose condition deteriorates a little more each day, expanding access to drugs and support for rare diseases can’t come soon enough.

“I can’t say things like, ‘Our 10-year anniversary is coming up so let’s save money and go on a trip,’ because I don’t know what life is going to look like for me in 10 years,” she says. “Will I be able to hold my own head up on a plane? Will I even enjoy travelling? I can’t plan for the future. This disease will slowly rob me of absolutely everything.”

Vanessa Hrvatin is a B.C.-based freelance writer.

Interested in other stories about rare disease advocacy? Read about Catherine Boivin’s life as an advocate for people living with spinal muscular atrophy, John Clark’s work in the myeloproliferative neoplasms community, and why Riyad Elbard is fighting for Canadians living with thalassemia.

February 28 is Rare Disease Day, a global movement focused on improving equity in social opportunity, health care, and access to diagnosis and therapies for people living with a rare disease. To find out how you can support Canadians living with rare diseases, visit the Canadian Organization for Rare Disorders.

Health Canada approves Risdiplam!

mollyocean — Fri, 16 Apr 2021 16:15:58 +0000

Treatment potential for our pediatric and adult patients!

Dear Canada’s SMA community,

Cure SMA Canada is happy to share that approval has been issued by Health Canada for Risdiplam for SMA patients 2 months of age and older.

Risdiplam is an oral treatment from Roche that treats SMA by increasing the SMN levels. The convenience and benefits of this oral treatment has proven to be life changing for Canadian patients that are already accessing through clinical trials or through Canada’s Expanded Access Program.

Cure SMA Canada looks forward to working with stakeholders to provide justification and support for the various government levels of approval. Our ultimate goal is to ensure that Canada’s SMA patients will have the opportunity to access Risdiplam in a timely manner. Many of Canada’s SMA patients are still living without access to treatment, Risdiplam offers a solution to the unmet need.

We look forward to engaging the SMA community to support our initiatives as we continue our advocacy for access to treatment.

Health Canada’s approval is a wonderful first step on our way to access Risdiplam, we look forward to ensuring Canada’s SMA patients will benefit from this life changing treatment.

Sincerely,
Susi Vander Wyk
Executive Director
Cure SMA Canada

Interim access to Zolgensma for Alberta children

ebesso — Thu, 28 Jan 2021 10:00:55 +0000

Families of Alberta children suffering from spinal muscular atrophy (SMA) may now be eligible to receive funding for gene replacement therapy treatment.

Alberta’s government is working with Novartis Pharmaceuticals Canada Inc. to provide interim patient access to Zolgensma.

SMA is a rare and progressive genetic disorder that causes muscle wasting. Alberta children with SMA and who may become ineligible for the treatment while waiting for the final approval processes to be completed, will be considered for funding on a case-by-case basis.

“Access to this treatment will mean a world of difference for these few brave children and their families. Alberta’s government recognizes the urgency families are experiencing in getting treatment for their children diagnosed with this disease and we are pleased to be able to provide interim funding to help them out.”

Tyler Shandro, Minister of Health

“We are so thrilled about the funding approval in Alberta for Zolgensma. This is huge for the SMA community and we feel a great sense of joy that no other family will be faced with raising money for the world’s most expensive medication.”

Lana Bernardin, mother of Kaysen

“Thank you to the Alberta government for making a life-saving decision that will change the course of this devastating disease. We are thrilled knowing the impact of this decision on the patients and their families.”

Susi Vander Wyk, executive director, Cure SMA Canada

Specialist physicians who provide care to children with spinal muscular atrophy are able to submit an application on behalf of their patients for coverage to be considered.

“This is a positive step forward in providing better care for young children with spinal muscular atrophy and wonderful news for those families who will have access to this gene therapy.”

Dr. Jean K. Mah, Division of Pediatric Neurology, Alberta Children’s Hospital

The Government of Alberta will continue working with the Canadian Agency for Drugs and Technologies in Health and the pan-Canadian Pharmaceutical Alliance (pCPA) to complete the standard drug review and negotiation processes, and to reach a long-term agreement for access to the drug for other children and families.

“Novartis is proud to share Alberta’s commitment to the SMA community. We feel privileged to be a partner with the province to provide access to Zolgensma during this interim period and address the urgent treatment needs of children affected by this devastating disease.”

Andrea Marazzi, country head, Novartis Pharmaceuticals Canada

Quick facts

Spinal muscular atrophy or SMA is a rare motor neuron disorder that can affect the muscles used for head and neck control, sitting, crawling, walking and swallowing.
Zolgensma was approved by Health Canada in December 2020; a review by the Canadian Agency for Drugs and Technologies in Health is underway.
Zolgensma is administered by intravenous (IV) infusion to replace a missing or faulty gene.
A one-time treatment of Zolgensma costs US$2.125 million.

Multimedia

Watch the news conference

Media inquiries

Steve Buick

780-288-1735
Press Secretary, Health

SMA Data Update

ebesso — Mon, 06 Apr 2020 14:51:28 +0000

SMA Data update with Biogen.

Dear members of the SMA community,
Following your request for updates regarding new SMA data in adult patients, we wanted to share that a real-world study of nusinersen (SPINRAZA ) use in teens and adults with SMA was published in The Lancet Neurology on March 18, 2020 in a manuscript titled “Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study”. Results from this independent observational study conducted in Germany by Hagenacker T, et al. demonstrate the safety and efficacy of nusinersen in teens and adults with genetically confirmed 5q spinal muscular atrophy (SMA). The study examined the largest adult patient cohort to date treated with nusinersen and showed clinically meaningful improvements in motor function.
The study contains data from 124 adult SMA patients (mainly, types 2 and 3) from ten neuromuscular treatment centers in Germany. The results are based on the evaluation of several motor function scales, including the HFMSE (Hammersmith Functional Motor Scale Expanded), RULM (Revised Upper Limb Module) and the 6MWT (6-Minute Walk Test).

A summary of the study is available here.

If you have any questions about the data, please reach out to a treating SMA physician.

Sincerely,

Biogen Canada

Mise à jour des données sur l’AS.

Aux membres de la communauté de l’amyotrophie spinale (AS),
À la suite de votre demande de mises à jour concernant de nouvelles données sur l’AS chez les patients adultes, nous souhaitions vous informer qu’une étude sur l’utilisation du nusinersen (SPINRAZAMC) dans le monde réel chez des adolescents et des adultes atteints d’AS a été publiée dans la revue The Lancet
Neurology le 18 mars 2020. Le manuscrit est intitulé « Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study ».
Les résultats de cette étude observationnelle indépendante menée en Allemagne par Hagenacker T, et coll. démontrent l’innocuité et l’efficacité du nusinersen chez les adolescents et les adultes atteints d’amyotrophie spinale 5q confirmée génétiquement. L’étude a examiné la plus importante cohorte de patients adultes traités par le nusinersen à ce jour et a montré des améliorations cliniquement
significatives de la fonction motrice.
L’étude contient des données provenant de 124 patients adultes atteints d’AS (principalement des types 2 et 3) de dix centres de traitement neuromusculaire en Allemagne. Les résultats sont fondés sur l’évaluation de la fonction motrice au moyen de plusieurs échelles, dont les suivantes : Hammersmith étendue (HFMSE; Hammersmith Functional Motor Scale Expanded), test de fonction des membres supérieurs (RULM; Revised Upper Limb Module) et test de marche de six minutes (6MWT; 6-Minute WalkTest).

Un résumé de l’étude est accessible ici.

Si vous avez des questions au sujet des données, veuillez communiquer avec un médecin qui traite l’AS.

Cordialement,

Biogen Canada

Together in SMA with Biogen March Newsletter

ebesso — Sun, 29 Mar 2020 13:31:35 +0000

Together in SMA with Biogen.

Please click here to view or download the newsletter