Funding of Spinraza for all Canadian patients affected by Spinal Muscular Atrophy (SMA)
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Why is SMA so challenging?
Imagine a disease that destroys the motor neurons controlling the arms, legs, chest, face, throat and tongue. That’s SMA, or Spinal Muscular Atrophy. About 1 in 6000 newborns are affected, and about 1 in 40 people carry the gene for SMA. Only a year ago, it was expected that a child, like Nina, born with severe SMA would never be able to sit or breathe on her own; she would most likely not survive beyond her second birthday. Children, like Mark, with a less severe form might gradually lose his ability to walk and to use his hands...
Why do all Canadians need to sign this petition?
Funding is being negotiated by yet another government agency, the panCanadian Pharmaceutical Alliance (pCPA).
AND THIS IS THE TRAP.
If governments agreed to fund only those SMA patients recommended by the review agencies, INESSS and CADTH, only those infants who are most severely affected will get coverage. And of course we do not want NO delay for these patients. BUT if negotiations are concluded too hastily, we are at high risk of excluding all other SMA patients who could benefit from Spinraza. This is a situation that is equally abhorrent, probably unethical, and undoubtedly devastating for the SMA community.
Which is why Cure SMA Canada and the Canadian Organization for Rare Disorders are calling upon all Canadians to sign this petition, demanding governments include all SMA patients in the funding of Spinraza and to make the drug available immediately.
WHEREAS clinical evidence has demonstrated that Spinraza significantly reduces the risk of dying and improves motor and respiratory functions;
WHEREAS patients with a broad spectrum of Spinal Muscular Atrophy (SMA) receive Spinraza treatment in 15 other countries, including the United States, France, Germany, Sweden and Italy;
WHEREAS interim results from clinical trials (with less severe or late onset patients) demonstrated such clear benefit that it was deemed unethial to keep patients on placebo; hence trials were discontinued and patients switched to actual therapy;
WHEREAS Spinraza’s health benefits would considerably alleviate provincial medical and social costs;
WHEREAS funding has been granted for the treatment of Canadian patients with other rare diseases on the basis of clinical trials smaller in size, with similar or lesser outcomes, and without conclusive data indicating positive long-term impact on survival. Denial of treatment would essentially discriminate against SMA patients;
Whereas the very small number of SMA patients would result in a very low overall budget impact, and. the drug’s net impact should take into account the potential to delay or avoid more invasive and costly procedures;
WHEREAS Spinraza treatment for SMA of any type should be started as early as possible to prevent disease progression and loss of motor and respiratory functions, ultimately saving life as well as improving quality of life.
We, the undersigned, request that the decision-makers and/or governing bodies urgently grant coverage and/or formulary listing of Spinraza to make this drug available to all Canadian patients with Spinal Muscular Atrophy.
TICK TOCK!!! The clock continues to move relentlessly forward for a patient affected by SMA. Every day without treatment is taking something from us we can’t get back. Some have paid the ultimate price of losing a child, all SMA patients have lost abilities and worry about losing more. Cure SMA Canada, CORD and the SMA community are counting on you to ensure our governments make the right decision. Our lives depend on it!
What is the hope?
So what has changed in a little more than a year? In December 2016, the US Food and Drugs Administration granted approval for Spinraza, the very first treatment for 5q-SMA (representing 95% of SMA patients). In June 2017, the European Medicines Agency (EMA) authorized use across a broad range of SMA patients based on strong interim results from clinical trials with less severe (late onset) SMA patients. Because of the rapid progression and debilitating consequences of this disease, 15 countries are already funding patients with a broad range of SMA.
What is the problem in Canada?
In July 2017, Health Canada approved Spinraza for all patients with 5q-SMA. In December 2017, following months of review, the Quebec review agency, L’Institut national d’excellence en santé et en services sociaux in Québec (INESSS), recommended Spinraza NOT be funded for ANY SMA patients. Just before Christmas 2017, the review agency for all other provinces and territories, the Canadian Agency for Drugs and Technologies in Health (CADTH), recommended funding to only a tiny fraction of those with the most severe form of SMA. Basically any child over the age of two will be denied treatment. So contrary to the conclusions of the EMA and Health Canada that there was strong clinical evidence demonstrating the effectiveness of Spinraza for all SMA patients, contrary to the decision of 15 other countries to make treatment broadly available, most Canadians with SMA, unless they have private drug coverage, will not be treated.
About Cure SMA Canada
Cure SMA Canada is the national registered charity; supporting Canadian families and individuals affected by Spinal Muscular Atrophy from the point of diagnoses, through the life course and even after loss of life. Cure SMA Canada also funds critical Canadian research projects with the aim of affecting accessible treatments for SMA. Cure SMA Canada provides advocacy, information and resources to families, communities and health professionals.
For more information about Spinal Muscular Atrophy, please go to our website at curesma.ca
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