The Global Approach to Cell Therapy

Cell therapy is a global science governed by national law. A preparation that is a routinely administered product in one country may be an unapproved investigational agent a border away — not because the biology changed, but because the regulatory philosophy did.

Three regulatory postures

The United States distinguishes between products regulated solely under Section 361 of the Public Health Service Act — minimally manipulated, homologous use, meeting defined criteria — and those requiring biologics licensure under Section 351. The classification determines almost everything that follows: what testing is required, what claims may be made, and what pathway to market exists.

The European Union classifies most cell-based products as Advanced Therapy Medicinal Products, centrally assessed by the EMA, with a hospital exemption permitting limited non-routine preparation under national oversight. Japan introduced a conditional, time-limited approval pathway that permits earlier market access on demonstrated safety and probable benefit, with efficacy confirmation required subsequently — a genuinely different bet about how to balance access against certainty.

Where divergence becomes a clinical problem

Regulatory asymmetry creates arbitrage. Patients travel to jurisdictions with lighter oversight, and a market has grown around clinics offering procedures unavailable at home. Some operate responsibly. Many do not, and the field's public credibility has absorbed real damage from advertised interventions with no characterisation, no follow-up and no adverse-event reporting.

The physician's exposure here is direct. A patient who received an uncharacterised preparation abroad may present with a complication and no documentation of what was administered.

Where convergence is happening

Standards are internationalising faster than statutes. The ISCT minimal criteria, MISEV reporting guidance, ISO 20387 for biobanking and ISO/IEC 17025 for laboratory competence are recognised across borders. Multinational registries and shared adverse-event reporting are slowly building the evidence base that fragmented national systems could not.

The practical filter

Jurisdiction is a weak proxy for quality; documentation is a strong one. A preparation accompanied by a certificate of analysis specific to its lot, an accredited quality framework, declared donor screening and traceable custody is verifiable regardless of where it was made. One without those is unverifiable regardless of what any regulator has or has not said about it.