The approval of Korea’s first advanced regenerative medicine treatment plan is ushering in a new medical paradigm of “pre-approval therapy,” allowing patients to receive cutting-edge treatments before formal product approval.
As advanced regenerative medicine moves beyond institutional framework-building into actual patient care, attention is growing over its potential impact on both clinical practice and the broader biotech industry.
Lee Jun-mi, director of the Regenerative Medicine Policy Division at the Ministry of Health and Welfare, emphasized the importance of the advanced regenerative medicine treatment plan system during a briefing for journalists from medical and pharmaceutical media outlets. (KBR photo)
Lee Jun-mi, director of the Regenerative Medicine Policy Division at the Ministry of Health and Welfare, explained the significance of the newly approved advanced regenerative medicine treatment plan during a briefing for journalists from medical and pharmaceutical media outlets on Wednesday.
The first approved treatment plan targets patients with Epstein–Barr virus (EBV)-positive extranodal natural killer/T-cell lymphoma (ENKL) who achieved complete remission after anticancer therapy but remain at high risk of relapse. The treatment involves administering autologous EBV antigen-specific immune cells (T cells) derived from the patient.
Lee emphasized that the approval marks the first real-world application of Korea’s advanced regenerative medicine system for patients, adding that it goes beyond symbolic significance by offering a practical model for clinical implementation.
“Once a treatment plan is approved, treatment can usually begin immediately after the medical institution enrolls in liability insurance,” Lee said. “However, this first case also falls under the Ministry of Trade, Industry and Energy’s regulatory sandbox program, requiring additional procedures.”
The first approval is significant because it signals a shift from treatment centered solely on approved drugs to treatment approaches grounded in clinical data.
“Until now, there had been persistent questions from the field about why advanced regenerative medicine treatments were not actually being implemented in clinical settings,” Lee said. “This case has opened a path for patients to receive real treatment within the institutional framework. It will also serve as a benchmark for researchers on how to submit and obtain approval for treatment plans.”
Clinical data reviewed during the approval process also showed promising results. In a clinical trial conducted under the regulatory sandbox program, no serious adverse events or deaths occurred among the 21 patients in the treatment group, while some adverse events were reported in the control group. In terms of efficacy, two-year follow-up data showed relapse in one patient in the treatment group, compared with eight in the control group, demonstrating meaningful clinical benefit.
“To gain approval for an advanced regenerative medicine treatment plan, prior studies demonstrating safety and efficacy are critically important,” Lee said. “Maintaining the same objectives and study design as previous research is also a key factor in determining approval.”
She explained that appropriate clinical trial design, including randomization, and the reliability of the data are also major evaluation criteria.
Another notable aspect of the first approved case is its “risk-sharing payment structure.” The total treatment cost is approximately 70 million won ($48,265). Patients initially pay 40 million won in installments, and if no relapse occurs over five years, they pay the remaining 30 million won. If the disease recurs, however, the previously paid amount is refunded.
“The structure was designed to reduce patient burden and uncertainty associated with non-reimbursed treatment,” Lee said. “The fact that researchers also shared part of the risk was viewed positively during the review process.”
Regarding the broader review status of advanced regenerative medicine treatment plans, 18 applications have been submitted to date. Fourteen were deemed unsuitable, while several others remain under review. The review committee meets monthly, with approval decisions made separately for each case.
Lee said that although no clear standards currently exist for incorporating approved treatment plans into the national health insurance system, the government plans to examine the issue based on accumulated operational experience. Related policies are expected to be included in the “Second Five-Year Master Plan for Advanced Regenerative Medicine and Advanced Biopharmaceuticals,” scheduled for release in June.
She also noted that data accumulated through the treatment track could later serve as important evidence during the formal product approval process.
“There is a need to actively utilize real-world data by linking different regulatory systems,” she added.
Lee described the greatest significance of the first approval as the “practical realization of the system.” While the government had focused on establishing the institutional framework following the law’s implementation in 2025, it has now entered a stage where patients can actually receive treatment.
“Advanced regenerative medicine is a key field that can help overcome rare and intractable diseases and drive a transformation in future medical paradigms,” Lee said. “Now we must move beyond building institutional foundations and toward providing real treatment opportunities for patients.”
She added that although researchers are highly motivated, many still struggle due to insufficient infrastructure at medical institutions.
“As the government plans to expand support, we hope medical institutions will actively participate and support related research,” Lee said. “Advanced regenerative medicine holds both public and industrial value, and active participation by healthcare institutions will ultimately contribute to the success of the system and greater benefits for patients.”
Kwak Sung-sun
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