If it were not for bad news, the spinal muscular atrophy (SMA) community in Canada would have no news at all. At least in the past couple of months.
First, the Good News
Last summer, the situation was so promising—that was when the drug Spinraza (nusinersen) was approved by Health Canada for all 5q-SMA patients (which is the majority of SMA patients.) The speed at which that happened was quite impressive. Just 7 months earlier, the US Food and Drug Administration (FDA) approved the drug for all SMA patients.
The approval of the drug was based largely on a phase 3 study in which the drug was given to infants with the most severe form of the disease (SMA I). In the study, 40% of the patients given Spinraza were able to show motor milestones compared to none in the control group. And for this disease, those numbers are phenomenal. And the FDA and Health Canada understood that. They understood the pathophysiology of SMA (they have a genetic mutation on the SMA1 gene) and the mechanism of action of Spinraza (it can produce SMN protein from a normally silent SMA2 gene). And with that knowledge, the regulators cut the makers of Spinraza some slack when looking at data for SMA patients aged 2-12 years with the less severe forms (SMA II, SMA III). That data used a different dosing schedule than the one approved but still showed the drug to be effective.
So for the SMA community in Canada, all they had to was wait for the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) to approved the drug for distribution and used that approval to quickly get the provinces to approve the drug for their formularies as well. SMA is a truly devastating disease and the knowledge that a drug would soon be available had the Canadian SMA community thrilled.
Then the Bad News
Both the L’Institut national d’excellence en santé et en services sociaux in Québec (INESSS) and CDEC recommended the drug be reimbursed only for patients with the most severe form of the disease and be treated before they reached the age 7 months (the age limit in the phase 3 study that led to the drug’s approval).
The next step is to start discussions with the provinces and pan-Canadian Pharmaceutical Alliance (pCPA) to get the drug to the patients. And that is going to take a lot of time and effort, not only to get the drug be part of their participating drug plans but the even more difficult hurdle of having those drug plans include all SMA patients rather than just those under the age of 7 months.
Interestingly, while the regulatory bodies have focused on the efficacy data, it is likely that the major concern of allowing this drug be given to older SMA patients is the cost. In the U.S., the first year of treatment costs $750,000 and $375,000 for every subsequent year.
Cure SMA Canada Responds
And that more difficult hurdled race is what the Canadian SMA community is starting now. They are extremely disappointed by the recent decisions and also the slowness to which the provinces and the pCPA seem to be responding. In a news release, Cure SMA Canada noted that the pCPA does not list Spinraza on the drug products to be considered for recommendation for negotiation. Meanwhile, 15 other countries having secured broad access to Spinraza for all SMA patients.
Yesterday, we spoke with Susi Vander Wyk, Cure SMA Canada Executive Director about the predicament the SMA community finds itself.
Vander Wyk said, “we are very frustrated right now. We have the most to win and the most to lose. And it is simply taking too long for things to move forward.”
Vander Wyk added, “it seems like every step of the process is a fight. We have families used to fighting for advocacy but this is a new type of fight and one we thought would not be so difficult. The thing is we do not have any other options – this (Spinraza) is it.”
In addition to having to contact all provincial leads to plead their case for including Spinraza on a drug plan, Cure SMA is also concerned that the limited criteria for the drug will make it extremely difficult for infants, even with the right eligibility requirements, to have enough time to get the drug. “Its such a small subset of SMA patients that will be allowed to have the drug compared to the entire SMA population that would benefit. Meanwhile, 15 other countries have approved the drug for all SMA patients. They had the same data that was given to the Canadian government,” said Vander Wyk.
While frustrated by the bureaucracy, Cure SMA Canada continues to plead their case and families are being encouraged to post their testimonies about what access to this drug would mean to them. You can see those videos on their Facebook Page.
One of their more compelling videos is one involving Vander Wyk at last October’s Canadian Organization of Rare Disorders Conference in which she pleas with the regulators to stop looking solely at the data and look up to see the families willing to risk everything for a treatment. That video is shown below.
Posted by James Radke | Jan 24, 2018