Jun-mi Lee, director of the Ministry of Health and Welfare’s Regenerative Medicine Division
The advanced regenerative medicine treatment system has produced its first approved treatment plan since its implementation, marking a shift from a clinical research-centered framework to one that enables actual patient care. The government said the case could expand treatment access for patients with rare and intractable diseases while supporting growth in the advanced biopharmaceutical industry.
“Under the existing drug approval system, it was difficult to fully reflect the characteristics of advanced regenerative medicine, which uses living cells and genes,” Jun-mi Lee, director of the Ministry of Health and Welfare’s Regenerative Medicine Division, said during a briefing with medical reporters on May 6. “A separate clinical research framework was introduced to reflect these characteristics, and this case is significant as the first example of the treatment system leading to actual patient care.”
The treatment plan was submitted by Catholic University Yeouido St. Mary’s Hospital. It involves relapse-prevention treatment for patients in complete remission from EBV-positive extranodal NK/T-cell lymphoma, a rare blood cancer with a high relapse rate and poor prognosis.
“This system will allow a new treatment approach, similar in concept to CAR-T therapy, to be provided to patients with rare lymphoma to help prevent relapse,” Lee said. “It will also serve as a reference case for how researchers can design and obtain approval for treatment plans going forward.”
Clinical data on efficacy and safety were key to the approval. According to Lee, a clinical trial comparing 21 patients in the treatment group with 25 in the control group found no serious adverse events or deaths in the treatment group, while some occurred in the control group. During two years of follow-up, one patient in the treatment group relapsed, compared with eight patients in the control group who either relapsed or died.
Under the law, high- and medium-risk treatments are generally required to go through advanced regenerative medicine clinical research. However, this case became the first to use clinical trial data under the Pharmaceutical Affairs Act through a regulatory sandbox.
Lee emphasized that consistency with prior research and the validity of the study design are important in treatment plan reviews. “The purpose and content must be identical to those of existing research, and approval is unlikely if the design is inappropriate or if efficacy and safety are insufficient,” she said.
The cost structure was also viewed positively. Since advanced regenerative medicine treatment is legally classified as non-reimbursed, the patient burden can be high. In this case, an outcome-based payment model will be applied. Of the total treatment cost of about $50,000, patients pay an initial $27,000. Additional payment is required only if the patient does not relapse within five years. If relapse occurs, the amount already paid will be refunded.
“This structure shares part of the risk to reduce patient concerns over high-cost treatment,” Lee said. “The review committee also viewed this approach positively.”
Because the system is designed to expand treatment access before product approval, safety monitoring and adverse event reporting are critical. Medical institutions providing advanced regenerative medicine must enter patient registration, treatment administration details, research results and adverse events into the regenerative medicine safety management system.
“Safety monitoring and adverse event reporting are carried out under a separate safety management framework established by law,” Lee said. “The Advanced Regenerative Medicine Act requires a safety management agency to conduct monitoring and receive adverse event reports. The Regenerative Medicine Safety Management Division of the National Institute of Health under the Korea Disease Control and Prevention Agency is currently designated as the safety management agency.”
Future tasks include linking the treatment system with health insurance and defining its relationship with the product approval pathway. “There are currently no clear criteria connecting the advanced regenerative medicine treatment system with health insurance coverage,” Lee said. “This will need to be reviewed as the system develops based on operational experience.” Related details are expected to be included in the second basic plan, scheduled for release in June.
On the need to connect the treatment track with the Ministry of Food and Drug Safety’s product approval system, Lee said, “Real-world data accumulated during treatment should hopefully be used as reference material in future product approval reviews. We need to explore ways to link the two systems through discussions with the MFDS.”
“Advanced regenerative medicine is important not only for its public value in addressing rare and intractable diseases, but also as a field leading the future shift in medical treatment,” Lee said. “Active participation by medical institutions and expansion of infrastructure will be essential. The government will continue strengthening institutional support so that advanced regenerative medicine can deliver meaningful outcomes in both expanding patient treatment opportunities and supporting industry growth.”
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