Evaluation different after Kymriah·Zolgensma benefits?

Concern about passing on 300 million drug costs to patients if reimbursement is not recognized later

Kymriah is not subject to prior approval, and performance evaluation is not disclosed

The ultra-expensive treatments Zolgensma and Kymriah are supposed to go through a performance evaluation, but whether or not they are disclosed is different.

 

As a pre-approved drug, Zolgensma, which is subject to review by an expert review committee, has performance evaluation results disclosed to the public, whereas Kymriah does not.

 

Therefore, unlike Zolgensma, Kymriah, which received a benefit earlier than Zolgensma, does not disclose performance evaluation results.

 

According to the industry on the 5th, The HIRA recently disclosed the first performance evaluation of Zolgensma on its website.

 

Zolgensma requires clinical evaluation before administration and every 6 months after administration for up to 5 years, and objective data such as medical records for clinical evaluation must be submitted.

 

In addition, ▲ permanent respiratory use or death, ▲ CHOP-INTEND score not improved by 4 points or more compared to the baseline before drug administration, ▲ even if the previous items were improved, CHOP-INTEND 4 points or more in two consecutive times in the subsequent response evaluation Or, if it falls under any of the cases of a decrease of 3 points or more on the HFMSE, it is defined as drug administration failure.

 

Reflecting this, as a result of the first performance evaluation, clinically meaningful improvement was confirmed in 5 out of 6 cases and marked as successful.

 

The remaining case was judged to be a failure because a patient with respiratory problems due to SMA died of suspected acute respiratory failure.

 

5 out of 6 were successful reviews.

 

Then, how was Kymriah, which was listed for benefit in April last year, 4 months ahead of Zolgensma, evaluated?

 

Zolgensma is an ultra-expensive new drug worth 2 billion won and Kymriah is about 360 million won, and the Ministry of Health and Welfare is managing the performance of both.

 

However, Kymriah cannot know the results of the evaluation.

 

Because there are no disclosure rules.

 

An official from the HIRA said, "Zolgensma is a pre-approved drug that goes through an expert review committee, so the review results are disclosed according to regulations, but Kymriah has no disclosure regulations." “At this time, we are not considering making it public,” he explained.

 

Kymriah was not subject to the prior approval system because the patient's condition could deteriorate due to medication delay.

 

For this reason, performance evaluations are not made public.

 

Regarding this, an industry insider pointed out, "It is appropriate to disclose the results of the performance evaluation to the outside in order to enhance transparency and reliability in the context of the decision to evaluate the performance as part of the management of super-expensive drugs." The HIRA went through a professional review committee and disclosed two cases of the recognition of Kymriah's benefits in May, but both cases were rejected because they did not meet the standards.

 

If the reimbursement is rejected, the patient has no choice but to pay 360 million won for non-reimbursement drugs.

 

If benefits are applied, the drug price will be reduced to 6 million won.

 

For this reason, some are suggesting that a plan to reduce the burden on patients should be prepared in the event of a post-reimbursement issue by reflecting the characteristics of an ultra-expensive new drug.

 

Regarding this, Novartis said, "The reduction in benefit is a problem that occurs between the nursing institution and the patient and has nothing to do with the manufacturer.

 

There is no separate patient support program."