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On Behalf of the ISCT Asia Pacific (APAC) Industry Committee

Discussion Topic: Should We Be Treating Patients with Unapproved Cell and Gene Therapies?

Panelists

• Dr. Shuji Terai, Gastroenterology/Regenerative Medicine, Professor and Chairman of the Division of Gastroenterology at Niigata University; Vice President of the Japanese Society for Regenerative Medicine (JSRM)
• Dr. Christopher Bravery, EU/EMA Regulatory Frameworks, Independent Regulatory Consultant (Cell and Gene Therapy), Former Evaluator/Reviewer with MHRA and EMA.
• Dr Wilson Bryan, U.S. Regulatory Frameworks, Independent Regulatory Consultant; Former Director of the Office of Tissues and Advanced Therapies (OTAT), U.S. FDA.
• Kunihiko Suzuki, APAC Regulatory Frameworks, Representative at Forum for Innovative Regenerative Medicine (FIRM); VP-Elect of ISCT Asia Regional and ISCT Ethics Committee for CGT.

Moderator

• Bill Milligan, Business Development Consultant (APAC Region), Independent Consultant focused on Japan, Taiwan, and the U.S.; Former Chair of the ISCT APAC Industry Committee

Date and Time: 7th May 2026, 08:00 a.m. 

Location: ISCT 2026 Scientific Annual Meeting in Dublin

Key Takeaways and Summary

Main Discussion Points

• Regulatory frameworks for access to unapproved cell and gene therapies vary significantly across the U.S., EU, Japan, and other APAC nations, ranging from tiered risk-based exemptions to hospital exemption systems and conditional approvals.
• Equity and affordability are major unresolved concerns; patient-pays models under compassionate use or expanded access disproportionately favor wealthier patients and populations.
• Enforcement of rules governing unapproved therapies, particularly for unregulated stem cell clinics, is weak in the U.S. and inconsistent globally; no single agency has the capacity or political will to regulate the full landscape.
• Smaller and lower-income markets face structural disadvantages in accessing approved therapies, making alternative access mechanisms (e.g., Singapore’s Special Access Route) essential.
• Japan’s 10-year experience with ASRM highlights both the value of tiered access systems and the need for robust outcome data collection; the absence of systematic data capture was an acknowledged shortcoming that is now being addressed through new registries.
• Company obligations to patients enrolled in investigational programs remain an under-addressed ethical and regulatory issue.
  

Key Conclusions and Recommendations

• Unapproved cell and gene therapies may appropriately be used, but only within legal, regulated frameworks; patients alone should not be the sole arbiters of whether access is appropriate.
• Institutional oversight, ethics committee review, and clinical governance are essential safeguards for any access pathway outside full market authorization.
• Outcome data registries (as developed by JSRM in Japan) should be considered a best practice and adopted more broadly to support informed consent and future regulatory decisions.
• Regulatory systems should include risk-tiered classification of products to proportionately match oversight intensity to actual risk.
• Greater attention to equitable access is needed across all frameworks; mechanisms to ensure that access to investigational therapies is not limited to affluent patients or countries should be developed.

Future Outlook and Actions

• FIRM’s comparative APAC regulatory survey (covering nine APAC countries plus the U.S. and EU) is available in abbreviated form on the ISCT conference platform, with a full report expected later in 2026.
• Japan, South Korea, and Taiwan are expected to continue refining their regulatory frameworks for medical practice with cell and gene therapies, with design improvements informed by Japan’s decade-long ASRM experience.
• The EU legislative revision process may introduce mandatory marketing obligations for rare disease products across all member states, improving equitable access within Europe.
• In the U.S., regulation of stem cell clinics is likely to evolve through state-by-state legislation rather than federal action in the near term.
• Consumer litigation (as documented in the Cytotherapy article “Litigating the Hype”) may emerge as a meaningful, if imperfect, accountability mechanism in jurisdictions with limited regulatory enforcement.
• ISCT and Regional Partners, such as FIRM, are expected to continue international dialogue on regulatory harmonization and ethical standards for access to unapproved therapies through forums like the Asia Partnership Conference for Regenerative Medicine.
  

You may find the Cytotherapy Article “Litigating the Hype: The Lung Health Institute Class Action and Its Implications“ here: https://doi.org/10.1016/j.jcyt.2026.102902