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South Korea expands access to regenerative medicine for serious illnesses

February 2025
Frances Verter PhD

 

Starting on February 21, South Korea will activate a “Regenerative Medicine Law” which will allow patients to receive cell and gene therapies that do not yet have market approval, if the patients have been diagnosed with conditions that are “severe, rare, or incurable”1. The full name of the new South Korean directive is the Act on the Safety of and Support for Advanced Regenerative Medicine and Advanced Biological Products2.

The key things to know about the Regenerative Medicine Law are listed here:

  • South Korea passed the law in August 2020 and it becomes effective February 2025.
  • The law allows patients outside clinical trials to access “new advanced regenerative medical technologies”.
  • Qualifying patients must have a diagnosis that has no approved treatment or the condition is serious, rare, or incurable.
  • The treatment must have already demonstrated safety and efficacy in clinical research.
  • The manufacturer of the treatment must follow strict quality standards and be subject to inspections.
  • There are 112 hospitals and clinics designated as advanced regenerative medicine (ARM) implementation institutions.
  • Qualifying medical institutions must be active in clinical research in the field of advanced regenerative medicine.
  • To initiate a treatment plan, the medical institution submits an application to the national review committee for ARM.
  • The national review committee operates under the Ministry of Health and Drug Safety.
  • The committee has up to 21 members that are scientific and medical experts and patient advocates, and more members will be recruited.
  • The national review committee meets monthly.
  • This regulatory framework, with applications submitted by hospitals and reviewed by a panel of experts, is designed to filter out more speculative regenerative therapies.
  • There are no restrictions on patient diagnoses. It is reported that the pathway will be interpreted to include treatments for arthritis, Parkinson’s, and auto-immune diseases.
  • There are restrictions on therapeutic modalities. The pathway excludes minimally manipulated cells such as cord blood, which have been stored under the “Cord Blood Law” and do not follow the new “Regenerative Medicine Law”.
  • Detailed patient data (real world evidence) must be collected under the law, including long-term follow-ups.
  • Health insurance will not cover these treatments because they are not yet approved for market.
  • Patients may be billed for the treatments.
  • The law requires participants to recuse themselves from any decisions or activities where they have a conflict of interest.
  • The law includes fines and penalties for violations of the regulatory requirements.

There are many similarities and differences between this new patient access program in South Korea versus compassionate use pathways for patients in other countries. Based on the full text of the law that is published online, the South Korea program most resembles pathways that are called “Expanded Access” in the United States (US) and “Hospital Exemptions” in the European Union (EU)2. In the US, manufacturers are allowed to charge patients that participate in expanded access programs, although in the EU the payment system varies by country3,4. One notable difference is that in most countries it is the patient’s doctor that initiates the request for compassionate use, whereas in South Korea it is the responsibility of designated medical institutions that have been approved to offer these treatments2-4. A very important distinction is that South Korea’s Regenerative Medicine Law excludes any therapy with minimally manipulated cells which are controlled by existing law2. In other words, infusions or injections of umbilical cord blood cells are excluded from compassionate use in South Korea, whereas they are allowed under compassionate use programs in all other countries2-4.

The media has been quick to cast the South Korea program as a response to Japan’s relatively liberal access to regenerative medicine1. However, a careful reading of the two frameworks shows that the regulations in South Korea are much stricter than those in Japan2,5,6. There are many differences, but some of the most obvious are that Japan stratifies cell and gene therapies into three tiers of risk, and there are multiple approval committees. In South Korea there is only one pipeline for approval by one centralized committee.

We requested comment from Min-Young Kim, MD PhD, who is a leading expert on the use of cord blood for regenerative medicine and is also a professional member of South Korea’s national review committee for ARM7. She replied, “In brief, while the news of Korea looks very positive, … there are many, many, hurdles to overcome, especially regarding cord blood use”.  We have seen that different countries are taking different approaches to speeding patient access to regenerative medicine, and it will be interesting to follow this newly enacted program in South Korea8-10.

 

References

  1. Heo J-Y. Korea permits stem cell therapies for arthritis, autoimmune diseases, and Parkinson's patients. Chosun Biz. Published 2025-01-19
  2. South Korea Government. ACT ON THE SAFETY OF AND SUPPORT FOR ADVANCED REGENERATIVE MEDICINE AND ADVANCED BIOLOGICAL PRODUCTS. Law Viewer. Act No. 16556, Aug. 27, 2019. Amended by Act No. 17472, Aug. 11, 2020. Effective Feb. 21, 2025.
  3. Verter F. Expanded Access Programs for Advanced Cell Therapies. CellTrials.org News. Published 2021-11-06
  4. Pachocki J, Verter F. Polish regulatory system regarding ATMP hospital exemptions. Frontiers Immunology. 2024; 15:1379134.
  5. Cyranoski D. Stem Cells 2 Go. The potent effects of Japan’s stem-cell policies. Nature Magazine. Published 2019-09-25
  6. Takashima K, Morrison M, Minari J. Reflection on the enactment and impact of safety laws for regenerative medicine in Japan. Stem Cell Reports. 2021; 16(6):1425-1434.
  7. Verter F. Cord Blood plus Erythropoietin is Better for Cerebral Palsy than Either Alone. Parent's Guide to Cord Blood Foundation Newsletter Published 2021-02
  8. Kim DS, Bae SJ. Impact and challenges of enactment for advanced regenerative medicine in South Korea. Frontiers Bioengineering Biotechnology. 2022; 10:972865.
  9. Yoon J, Lee S, Kim MJ, Kim J-H. Brief summary of the regulatory frameworks of regenerative medicine therapies. Frontiers Pharmacology. 2025; 15:1486812.
  10. Park S. World-First Approval, but Lagging Commericialization: Bridging the Gap. Hit News. Published 2024-12-03