Will a new reimbursement model be introduced for Zolgensma?

Will a new insurance benefit model be introduced for the ultra-high-priced Zolgensma?

Novartis, confident about the clinical efficacy and safety of Zolgensma, proposes a performance-based RSA model

Will require a huge fiscal spending for each approval… government is considering installment payments

Concurrent application of the expenditure cap type in RSA also possible

The move to list Novartis’s new drug ‘Zolgensma (onasemnogene abeparvovec-xioi)’ for reimbursement benefits is now underway.

 

The industry’s eyes are on which types of the RSA scheme will be combined for this ultra-high-priced ‘dream drug.’ Whether Zolgensma will be listed for insurance benefits is the focus of attention in the industry.

 

The industry-wide interest was approved in May in Korea as a single shot of Zolgensma costs over 2 billion won.

 

Of course, when compared to other existing drugs that require spending 300 to 500 million won every year for the rest of one's life, this ‘one-shot’ treatment cannot strictly be called an ‘ultra extensive' drug.

 

Nevertheless, it is true that the government burden would be immense, as it would have to bear billions of won as its cost each time.

 

The reasoning for Zolgensma’s high price is in that it can treat rare diseases that cause death with just a single injection.

 

Zolgensma treats spinal muscular atrophy (SMA), a rare condition in which the SMN1 gene is innately deficient or mutated to result in progressive muscle atrophy.

 

In the case of SMA Type 1, the most common and severe form of SMA, over 95% of the motor neurons are damaged within 6 months, and 90% die before the age of 2 if left untreated.

 

Zolgensma is a gene therapy that has a functional replacement for the missing or non-working SMN1 gene.

 

When the drug is administered to a patient, the replacement delivered with Zolgensma produces SMN proteins and radically cures the disease.

 

This completely different mechanism of action, unlike those of existing drugs that increase the production of SMN protein using the ‘backup’ SMN2 gene, is why Novartis calls Zolgensma an ‘innovation'.

 

In the Phase III SPR1NT study, all pediatric SMA patients with two SMN Type 2 gene copies (Cohort 1) that were treated prior to the onset of symptoms survived without requiring ventilatory or nutritional assistance and achieved sitting independently for 30 seconds or more.

 

Most (11/14) patients achieved age-appropriate motor milestones within the World Health Organization (WHO) window of normal development.

 

Also, in the real world, Zolgensma improved or maintained motor development scores in patients 6 months of age and older, regardless of their prior use of existing therapies.

 

The efficacy and safety of Zolgensam were confirmed for over 6 years, with treatment experience accumulated for patients aged 6 months to 2 years.

 

Novartis applied for Zolgensma's reimbursement benefit in June and is currently discussing ways to allow its reimbursement.

 

The key agenda lies in how to incorporate Zolgensma in the current reimbursement system.

 

As of now, the RSA scheme is being considered the most realistic alternative, and among the various types of RSA, it is likely that a mix of several models would be used to cover the drug.

 

The company has proposed a performance-based RSA system.

 

Miri Shin, Director of Zolgensma's Business Unit at Novartis Korea said, “We have reviewed various measures that can effectively reduce the burden on NHI finances and proposed the performance-based payment system to the government.” The performance-based payment system is an RSA conditional upon evidence development.

 

Under this RSA type, reimbursement is decided according to the results of a clinical trial that is conducted after listing.

 

The only drug listed with the RSA conditional upon evidence development was ‘Evoltra,’ a treatment for acute lymphoblastic leukemia.

 

If this performance-based RSA is applied for Zolgensma, setting the criteria for reevaluation will be important.

 

Also, new types such as installment payments can be incorporated into the system, under which the price of the single shot would be paid in installments.

 

The U.S.

 

has also adopted this method to minimize its financial burden.

 

In addition, finance-based RSA types such as the expenditure cap type may also be applied to Zolgensma.

 

“We are flexibly discussing various measures for Zolgensma’s reimbursement with the government," said Director Shin.

 

“We will continue discussions to work out the details.”