Following Kymriah, Zolgensma is also about to be reviewed

Jang Yong-myung, executive director of HIRA, mentioned in a briefing

Conditions for refunding expenses for ineffective patients

Jang Yong-myung, executive director of HIRA development, briefing with reporters at Korea Special Press Association on the 8th

So-called "one-shot treatments" that treat specific rare diseases with one administration are speeding up the reimbursement.

 

Following Kymriah, which treats blood cancer such as leukemia and lymphoma, Zolgensma (Onasemnogene Abeparvovec), which treats a rare disease called SMA, is also set to be reviewed by HIRA's Drug Reimbursement Evaluation Committee.

 

These drugs were difficult for patients to access because of their high price instead of high therapeutic effects, but if they are reimbursed, the burden of drug prices is expected to be relieved.

 

Jang Yong-myung, executive director of HIRA Development, said at the Korea Special Press Association meeting on the 8th that Zolgensma will deliberate on the Drug Reimbursement Evaluation Committee to evaluate the adequacy of medical care benefits.

 

Zolgensma is a drug that can treat spinal muscular dystrophy with a single dose.

 

Spinal muscular dystrophy is a fatal rare genetic disease in which muscles are gradually contracted due to deficiency or mutation of normal SMN1 genes, and if not treated, 90% of SMA type 1 dies before the age of 2 or requires permanent respiratory aids.

 

More than 30% of SMA type 2 patients can also die before the age of 25, requiring lifelong treatment.

 

Zolgensma replaces genes that can prevent SMA progression, so it is expected to improve the quality of life of patients and their families as it can be fundamentally treated only once in a lifetime with intravenous administration.

 

It is an ultra-high-priced new drug worth about 2 billion won, and it is too expensive for patients to handle, so being paid is the only hope for patients and their families.

 

Accordingly, the solution found by the insurance authorities allows pharmaceutical companies to refund the amount paid to patients who was not effective.

 

Executive Director Jang said, "Kymriah and Zolgensma are one-shot treatments that expect therapeutic effects from single administration, but there is a limit to proving cost effectiveness through existing evaluation methods due to high price and long-term uncertainty." "In the case of Kymriah, all patients who have been administered will be obligated to collect patient conditions for a certain period of time, and according to the results of the patient-level performance evaluation, pharmaceutical companies will refund a certain amount of money after administration," he explained.

 

He also said, "If we claim more than a certain amount, we will pay on the condition of a contract that returns all excess amounts," adding, "We will be subject to risk-sharing revaluation after the fourth year of registration." Kymriah passed the Reimbursement Evaluation Committee on the 13th of last month under these conditions.

 

As the adequacy of benefit has been recognized, it will now be possible if negotiations between the NHIS and pharmaceutical companies proceed smoothly.

 

The HIRA plans to evaluate the adequacy of benefit in the same way as Kymriah or Zolgensma.

 

Novartis applied for a decision on medical care benefits for Zolgensma in May last year and is currently being reviewed by the HIRA.

 

It has been collecting opinions from the society since July last year, and at the end of last year, an expert advisory meeting was held to discuss clinical usefulness.

 

At the end of January, the Drug Benefit Standards Subcommittee also reportedly set the benefit criteria.

 

Executive Director Jang said, "Zolgensma is also considering a therapeutic performance-based refund mechanism to overcome the limitations of proving uncertainty and cost-effectiveness about long-term effects, just like Kymriah, as a drug that is effective with one administration."