With ultra-high-priced treatments from global pharmaceutical companies newly entering the benefit range this year, human "risk management" is emerging as an issue at clinical sites. Since the treatment is so expensive, "risk" management issues such as damage and loss that may occur during administration are acting as a key issue.

#Kymriah, the CAR-T treatment of Novartis Korea, which entered the benefit range this year, and Zolgensma, the treatment of Spinal Muscular Atrophy (SMA), are representative. According to the pharmaceutical industry and the medical community, Novartis Kymriah Korea has been applied as a health insurance benefit since April, and it has been confirmed that it has been administered not only at university hospitals in the Seoul metropolitan area but also at university hospitals in provincial areas.

Zolgensma, who has newly entered the health insurance benefit zone since August, has passed the Drug Committee (DC) of major Big 5 hospitals, including Seoul National University Hospital and Seoul Asan Hospital, and is preparing to administer patients in earnest. As expensive treatments, which cost hundreds of millions of won alone, are administered in earnest at the clinical site, "risk management" that may occur is emerging as a key issue. For example, the question is who will be responsible for the damage or loss of the treatment in the process of handling patients with expensive treatments. This is because the price of the treatment is expensive, so both hospitals and pharmaceutical companies can be burdened depending on where they are responsible. Moreover, in Korea, it is recognized as a bigger problem because there is no insurance system that can be called a safety device in preparation for the human risk of such expensive treatments.

However, there is a difference if Kymriah and Zolgensma are directly compared in terms of risk management. First of all, Kymriah, which entered the benefit range, is a pair due to the nature of the treatment, that is, a system that prepares two treatments in case of an emergency.

An official from Novartis said, "In the case of Kymriah, due to the nature of the treatment, two treatments are prepared in case of an emergency." He explained the background, "As we make a treatment with T cells collected from the patient's blood, we can make two treatments with the same sauce." Kymriah reached up to 500 million won in the United States for a single administration, but in Korea, the patient's burden was lowered to up to 5.98 million won as it was set at 360.4 million won through drug price negotiations.

Against this background, Um Ki-sung, a professor of blood medicine at Seoul St. Mary's Hospital, said, "In the process of introducing Kymriah, Novartis said that he would not receive drug costs if it was not actually administered, regardless of who did it wrong." Professor Um Ki-sung explained, "If a patient receives a lot of chemotherapy during the three-week administration, lymphocytes may not be extracted or cells may not come out." Professor Um said, "This means that pharmaceutical companies will not take issue with this and will only receive the drug price when the administration is completed."

The problem is Zolgensma, which domestic drug price has been set at nearly 2 billion won. This is because the characteristics are different from Kymriah itself and there is no insurance system to prepare for risks that may occur during the administration process. In particular, in the case of Zolgensma, it is also worrisome that it differs from Kymriah in that the process is different, such as putting genes in vectors and transporting them so that they do not die. For this reason, there are opinions among large hospitals that are concerned about this.

On top of that, Evrysdi, which is considering salaries as SMA treatments following Zolgensma, is also said to have to come up with a safety device for human risk. In this regard, the HIRA, which is considering benefits, reportedly asked pharmaceutical company Roche to come up with a plan.

As Evrysdi is an oral drug, it means that safety devices should be prepared to prevent risk of patient loss. This is because Evrysdi will not have a high drug price compared to Zolgensma and Spinraza, but it is widely expected that it will be set as a burdensome drug price for patients.

Novartis is in a position to discuss the plan with hospitals as it is scheduled to be administered in earnest according to the application of Zolgensma benefits. "Since it is an expensive treatment, it is not easy for hospitals and pharmaceutical companies to pay for it," a Novartis official said. "In the near future, Seoul National University Hospital is discussing how to manage risks and has been partially coordinated." "I think we will need to continue discussions in the future," he explained. He said, "I checked the insurance company in terms of human risk safety, but there is no insurance company that covers it." He added, "There is no internal determination of responsibility at the moment, but since it is the first time, we have to continue to discuss it in the future.".