A committee of the Canadian Agency for Drugs and Technologies in Health (CADTH) has issued a draft recommendation advising Spinraza (nusinersen) should not be reimbursed for the treatment of adults with spinal muscular atrophy (SMA). 

CADTH is an independent, nonprofit organization that provides evidence-based advice to policymakers about healthcare technologies in Canada. The draft recommendation comes from CADTH’s Canadian Drug Expert Committee (CDEC).

“The committee was unable to conclude, from the available evidence, that [Spinraza] provides any clinically meaningful benefits in adults with type II or III SMA, regardless of ambulatory status,” the draft states.

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The draft recommendation was met with disappointment from the SMA community and from Spinraza’s manufacturer, Biogen, which said the decision “effectively denies adult patients with access to life-altering treatment.”

“Cure SMA Canada is devastated to hear of the negative draft recommendation from CADTH,” Susi Vander Wyk, executive director of Cure SMA Canada, said in a press release.

“The Canadian Organization for Rare Disorders feels betrayed by CADTH with this devastating recommendation NOT to provide access to adults with SMA despite the compelling real-world evidence that other countries have accepted and used to expand coverage. We desperately need a national Rare Disease Drug Strategy that is patient-centred,” added Durhane Wong-Rieger, president and CEO of the Canadian Organization for Rare Disorders.

Spinraza, which works to boost levels of the SMN protein whose deficit causes SMA, was approved in Canada in 2017. According to Biogen, the therapy is widely reimbursed in Canada for children with SMA; however, the only province where it is reimbursed for adults is Quebec.

Biogen says that, if implemented, this decision would put Canada out of step with more than 25 countries where Spinraza is reimbursed for SMA patients regardless of age. The company did not specify which countries.

“The Canadian community of adults with spinal muscular atrophy remains in a challenging position where they are unable to access a treatment approved for them by Health Canada except in Quebec where SPINRAZA is available to patients of all ages,” said Lawrence Korngut, MD, a neurologist at the University of Calgary.

In its draft decision, the CDEC acknowledged that there is an unmet need for treatments that can slow or stop disease progression in adults with SMA. However, the committee said that there is not currently enough evidence to say whether Spinraza actually provides benefits to adult patients.

The main issue is that there has never been a clinical trial to test Spinraza in adult patients. Instead, all of the available data on Spinraza come from real-world observational studies.

According to the CDEC, data from these studies “generally suggested that treatment with [Spinraza] had a positive effect on motor function,” but there were “key limitations including the uncontrolled nature of the studies and a high degree of selection bias, [resulting] in a patient population that was not considered representative of adult patients with SMA in Canada.”

The committee also noted that even without treatment, there is substantial variability in how SMA progresses. It also said that most patients in these studies were also receiving other interventions, like physical therapy.

“Therefore, CDEC could not draw any conclusions that the changes or maintenance in motor function scores were a result of [Spinraza] administration due to the considerable uncertainty associated with the evidence,” the draft states.

Xavier Rodrigue, MD, doctor of physical medicine and rehabilitation at Hôpital de l’Enfant-Jésus in Quebec, said the recommendation is “a very disappointing and unexpected response for the adult Canadian SMA community. All the more incomprehensible in the face of evidence of clinical effectiveness after three years of use in Quebec.” 

Wyk added, “Many adult patients globally and in Quebec have accessed SPINRAZA and had a life-changing impact on their quality of life, and we urge the members of CADTH to reconsider their decision.”

Korngut also voiced the opinion that real-world evidence supports the effectiveness of Spinraza for adults.

CADTH will accept feedback from stakeholders on its draft through June 3 and will then issue a final recommendation.

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