HIRA rejects reimbursed use of Spinraza on 14-year-old

HIRA posts online October Treatment Review Committee deliberation results

Preliminary application result on Spinraza, 11 maintenance dose approved, 4 failed

Approval on reimbursed use of Spinraza (nusinersen) on a 14-year-old male patient with 5q spinal muscular atrophy (SMA) was denied.

 

The application submitted by the hospital was rejected as it did not clarify if the patient had developed clinical symptoms of SMA before 36 months.

 

On Nov.

 

29, Health Insurance Review and Assessment (HIRA, President Kim Seung-taek) posted seven cases of Treatment Review and Evaluation Committee deliberation in October.

 

Although Spinraza was listed on the Drug Reimbursement List from last April 8, a healthcare institute planning to use the treatment has to submit a preliminary drug use approval application due to the ultra-expensive maximum reimbursement price reaching 92,359,131 won per 5 ml vial.

 

In last month, 15 preliminary drug use approval applications on Spinraza, including 13 first loading doses and two maintenance doses (application submitted once every four months), were submitted.

 

But the committee disapproved only one case, which involved a 14-year-old boy.

 

Other 11 first loading doses and two maintenance doses were approved, and one first loading dose was approved with conditions.

 

The conditional approval was granted to a five-month-old female infant trying to get detached from ventilator.

 

The committee approved of reimbursed use of Spinraza as long as the patient submits medical profession’s statement on use of ventilator prior to Spinraza administrations and medical record of respiratory function.

 

Before the Spinraza review, the committee reviewed 34 preliminary applications on use of Soliris (eculizumab), including 23 cases of paroxysmal nocturnal hemoglobinuria (PNH) and 11 cases of atypical hemolytic uremic syndrome (aHUS).

 

For the first loading dose, one PNH case was accepted and other one was rejected.

 

Whereas three cases of aHUS patients were accepted and other three were rejected.

 

Use of Soliris on a PNH case denied as the patient’s medical record was missing repetitive abdominal pain-induced hospital administration, but showed a recent increase in use of narcotic analgesic.

 

The patient’s record did not meet scope of reimbursed use of the treatment.

 

An aHUS case was rejected because the patient’s status was not clear cut as stated in the criteria.

 

The patient’s symptom was considered as a secondary thrombotic microangiopahty induced from rheumarthritis and its treatment, and from multiple myeloma and its treatment.

 

And also the patient showed rise of CEA level, delayed prothrombin time and activated partial thromboplastin time (PT/aPTT), and fall of fibrinogen level.

 

The committee also decided the patient cannot expect positive effect from the treatment as the patient needs dialysis for end-stage renal disease.

 

A preliminary approval review on reimbursed ventricular assist device (VAD) treatment was conducted as well.

 

A male patient aged 64 years was registered on the heart implant waiting list with dilated cardiomyopathy (DCM), who was diagnosed with end-stage heart failure by an echocardiography with dobutamine substance and motor function test.

 

The patient did not recover from medication treatment and is dependent on intravenous cardiotonic agent.

 

HIRA approved reimbursement on the patient’s VAD treatment, because the case met the reimbursement standard of ‘indication for VAD implant on end-stage heart failure patient, who has been registered on heart implant waiting list as a makeshift treatment’.

 

Also the patient not showing any contraindication helped the decision.

 

Other details of the Treatment Review and Evaluation Committee’s October meeting can be found on HIRA website (www.hira.or.kr).