Only 6 out of 9 reimbursed use of Spinraza approved

HIRA officially disclosed all reviewed cases and outcomes

1 out of 6 reimbursed use of Soliris for treating aHUS approved

Only 66.6 percent of preliminary approval applications for reimbursement submitted last month was cleared for the use of spinal muscular atrophy (SMA) treatment Spinraza (nusinersen).

 

Six out of nine applications were approved, and other three applications were rejected with conditional approval, disapproval and required supplementary data.

 

On Dec.

 

31, Health Insurance Review and Assessment Service (HIRA, President Kim Seung-taek) officially disclosed outcomes of reimbursement review on four items—Spinraza, Soliris, ventricular assist device (VAD), and hematopoietic stem cell transplantation—deliberated by Healthcare Review & Assessment Committee in November 2019.

 

Spinraza has been listed for reimbursement since last Apr.

 

8, but a healthcare institute intending to use super-high-cost treatment priced at 92.36 million won per 5 ml has to apply for a preliminary approval for reimbursement.

 

For reimbursement on the treatment, a patient has to qualify for all conditions of lacking 5q SMN-1 gene or being diagnosed with gene mutation; SMA related clinical symptoms and signs onset from age younger than three; and not in use permanent ventilator.

 

A well-known unapproved case was a 14-year-old male patient with SMA, who could not clearly prove his age of SMA-related symptom and sign onset was younger than 36-month-old according to the submitted data.

 

The committee did not approve the application without proper objective data.

 

A conditional approval was granted on 24-year-old female patient with a record of scoliosis surgery as her myelogram result proved she is able to take continuous administration into her dura mater with lumber puncture.

 

However, the approval condition was submission of the confirmed number of SMN2 copy with the monitoring report.

 

Soliris (eculizumab) was available for preliminary approval for reimbursement prior to Spinraza, and had six applications submitted for treating atypical hemolytic uremic syndrome (aHUS).

 

But only one was approved out of all.

 

A 49-year-old male patient was approved with reimbursed use of Soliris as he had recurrent thrombotic microangiopathy and kidney dysfunction, despite he had conservative treatment after ABO-incompatible kidney transplantation.

 

Although HIRA found, reviewing his medical record, thrombotic microangiopathy has occurred after the kidney transplantation and using immunosuppressant, it approved of the reimbursed use because the patient had clinical symptoms of aHUS and also continued to show thrombotic microangiopathy symptoms with no apparent response to hemodialysis and therapeutic plasma exchange.

 

But the patient has been ordered to submit genetic testing result and two-month monitoring report.

 

His feasibility of maintained reimbursement would be deliberated depending on the clinical result.