HIRA clears 4 out of 5 Spinraza reimbursement pre-approvals

HIRA request one additional data submission and approves 10 monitorings

Passed all 2 Soliris use on PNH, denied 1 aHUS case

Korean health authority has passed four out of five preliminary applications submitted last month to treat spinal muscular atropy (SMA) with reimbursed use of Spinraza.

 

Even the one denied case would likely to be cleared, if the applicant submits additional evidential data of patient’s onset symptoms of SMA.

 

In every four months, Spinraza users receiving reimbursement have to submit monitoring report prior to maintenance administration.

 

And the health authority has approved all ten monitoring reports submitted.

 

On Mar.

 

31, Health Insurance Review and Assessment Service (HIRA, President Kim Seung-taek) posted the results of pre-administration approval on four listed items—Spinraza, Soliris, ventricular assist device (VAD) and hematopoietic stem cell transplantation (HSCT)—Treatment Review and Assessment Committee deliberated in February.

 

Spinraza’s reimbursed use standard stipulates patients are eligible for reimbursement when qualifying all conditions—positive genetic testing of a deletion or mutation in the survival motor neuron 1 (5q SMN-1) gene, onset of SMA symptoms from age three or less and not on permanent ventilator (more than 16 hours a day for over 21 consecutive days).

 

The reimbursed use of the drug has to be assessed before the initial administration, after administering initial dosage (four doses) but before the fifth dose, and every maintenance dose then after based on clinical evaluation (development stage, motor function and respiratory function).

 

Before the Spinraza review, the committee reviewed pre-administration applications on reimbursed use of Soliris (eculizumab).

 

All three to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) were cleared, but one case of atypical hemolytic uremic syndrome (aHUS) treatment was denied.

 

The rejected aHUS case was to treat 67-year-old patient, who visited an emergency room for nausea and vomiting and was hospitalized due to acute renal failure and observation of symptoms like thrombocytopenia, anemia and schistocyte but continued to have thrombotic microangiopathy (TMA) despite receiving steroid and therapeutic plasma exchange (TPE).

 

Nevertheless, it was ultimately rejected, as the doctor’s record of the patient’s schistocyte and thrombocytopenia did not coincide with the TMA described in the reimbursement standard notice.

 

Other details of the Treatment Review and Evaluation Committee’s deliberation can be found on HIRA website.