Some users of spinal muscular atrophy (SMA) treatment Spinraza (nusinersen) failed to get reimbursement because their motor function was not maintained or improved after its administration.

As a result of the Health Insurance Review and Assessment Service's deliberation on whether Spinraza is eligible for medical benefits, three out of eight applications for reimbursement were disapproved. (Credit: Getty Images)
As a result of the Health Insurance Review and Assessment Service's deliberation on whether Spinraza is eligible for medical benefits, three out of eight applications for reimbursement were disapproved. (Credit: Getty Images)

The Health Insurance Review and Assessment Service (HIRA) released the results of the recent Medical Review and Assessment Committee meeting, which reviewed various applications from Spinraza users for insurance benefits, on its website.

Patients eligible for Spinraza with coverage are those with 5q spinal muscular atrophy (SMA) with a confirmed genetic diagnosis of a defect or mutation in the 5q SMN-1 gene if they have three or fewer copies of the SMN2 gene even before the onset of symptoms and are less than six months old at the time of treatment, and if they are type 1-3 with SMA-related clinical symptoms and signs and are not on a permanent ventilator.

As a result of the committee's deliberations, three of the eight applications for Spinraza's medical benefits were rejected because they failed to prove whether they could maintain their motor function according to the results of the motor function assessment.

For example, a 32-year-old man was rejected because his HFMSE (Hammersmith Functional Motor Scale-Expanded) scores were zero before and after receiving Spinraza, which cannot be considered as maintaining or improving motor function. Another man, 35, was also rejected for failing to demonstrate maintenance or improvement of motor function on two consecutive occasions, as his motor function was not identified in the video.

Besides, all 33 cases submitted as Spinraza administration monitoring reports were approved for reimbursed treatment because they confirmed that motor function was maintained or improved.

In contrast, all five applications for the oral SMA treatment Evrysdy (risdiplam) were disapproved.

While switching or combining SMA treatments is not authorized as reimbursable, a single switch is allowed only if the improvement is confirmed while taking Spinraza and there are clinical reasons (such as scoliosis) that do not meet the discontinuation criteria but require a switch to Evrysdi.

However, in the disclosed cases, it was confirmed that the replacement dose of Evrysdi was not approved because the results of the motor function assessment did not show that motor function was maintained or improved.

Further details of the committee's deliberations can be found on the HIRA website.

 

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