Novartis nears reimb for its 2 ultra-high-priced new drugs

Agreement reached during drug pricing negotiations for Zolgensma... Kymriah listed in April

Both applied the prior approval system… opens door to the era of one-shot treatments

With Novartis Korea nearing reimbursement listing for its 2 ultra high-priced drugs, how the two drugs will progress is receiving attention.

 

The two Novartis drugs that have continuously raised issues since its domestic approval are ‘Kymriah (tisagenlecleucel) and Zolgensma (onasemnogene abeparvovec-xioi).

 

Among the two drugs, Kymirah was first listed for reimbursement in April, and the drug pricing negotiations with the National Health Insurance Service for Zolgensma were also completed recently.

 

When Zolgensma passes deliberation by the Health Insurance Policy Deliberation Committee (HIPDC), the drug will also be applied the prior approval system like Kymriah.

 

If approved, it will be the first time in the history of Korea’s insurance reimbursement system that two drugs were consecutively applied the prior approval system for reimbursement benefit.

 

The reimbursement process for the drugs was far from smooth.

 

And when considering the long wait endured by the patients, it wasn’t even that ‘quick.’ However still, given the unprecedented cost of administration and the immense fiscal investment required, as well as its ‘one-shot’ concept, the results achieved by the two drugs are quite encouraging.

 

The gov't and pharmaceutical companies’ rapid response shone through for Kymriah The reimbursement of the ultra-high-priced one-shot CAR-T new drug Kymirah was made possible through the combined efforts of the government, pharmaceutical companies, and patients.

 

Kymriah is indicated for the treatment of ▲ adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL) after two or more lines of systemic therapy, and ▲pediatric and young adult patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (B-ALL) that is refractory or in second or later relapse.

 

The company had submitted a reimbursement application for both indications.

 

The drug started its reimbursement listing process after it was approved by the Ministry of Food and Drug Safety in March last year through the ‘approval-reimbursement review linkage system.’ Six months later, Kymriah was first put up as an agenda for NHIS’s Cancer Disease Deliberation Committee (CDDC) review, but the committee deferred its decision.

 

Immediately upon news of the deferral, the Korea Leukemia Patients Organization among others strongly criticized the government and the pharmaceutical company.

 

The KLPO had before criticized the authorities over the delay in Kymriah’s agenda being put up for deliberation by the CDDC.

 

In the end, Kymriah’s reimbursement agenda passed the CDDC review in October of the same year.

 

This was also the first day HIRA disclosed the results of CDDC deliberations to the public.

 

Also, Kymriah's reimbursement agenda passed the first Drug Reimbursement Evaluation Committee (DREC) meeting held in 2022.

 

After then, the company successfully completed drug pricing negotiations and officially listed Kymriah for insurance benefit in April.

 

Slower but fast enough results derived for 'Zolgensma' reimbursement The progress made for Zolgensma was slower than that of Kymriah.

 

Although both drugs are high-priced one-shot treatments developed by the same company, Zolgensma is a rare disease treatment.

 

Novartis submitted its reimbursement application for the spinal muscular atrophy (SMA) treatment Zolgensma in May last year, but the agenda was put up for deliberations a year later, in May of this year.

 

As Zolgensma is a rare disease drug, the pharmaceutical reimbursement standard subcommittee must first set a reimbursement guideline for the drug before undergoing DREC review.

 

However, discussions were delayed due to repeated requests for data supplements made by the government and their submission by the pharmaceutical company.

 

As a result, the reimbursement progress for Zolgensma progressed at a much slower speed than Kymirah, which had applied for reimbursement approximately a month before Zolgensma.

 

However, the process sped up after DREC deliberations.

 

After passing DREC deliberations on May 12th, Zolgensma entered drug pricing negotiations in the same month and reached a drug pricing agreement without period extensions before the 60-day negotiation period expired.

 

As a result, Zolgensma will likely be reimbursed as early as next month (August), or in September at the latest given HIPDC’s schedule.

 

Zolgensma is a gene therapy that contains genetic material that functionally replaces defective genes.

 

The Ministry of Health and Welfare had approved the drug as the second advanced biopharmaceutical after Zolgensma.

 

Advanced biopharmaceuticals are cell therapies or gene therapies that use live cells, tissues, or genetic material as ingredients.