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◆ Biotech (Basel (Switzerland))2026-09-16

Regulatory Convergence in Cell and Gene Therapy: Harmonizing Quality, CMC, and Approval Pathways Across the FDA, EMA, PMDA, and Emerging Markets.

Nagendra Verma, Swati Arora

原始摘要(英文原文)· Original abstract
Cell and gene therapies (CGTs) have progressed from proof of concept to an established commercial pipeline, yet global translation remains constrained by fragmented regulatory frameworks; heterogeneous Chemistry, Manufacturing, and Controls (CMC) requirements; and divergent approval pathways. This narrative review compares CGT quality, CMC, and approval-pathway regulation across the US Food and Drug Administration (FDA), European Medicines Agency (EMA), and Japan's Pharmaceuticals and Medical Devices Agency (PMDA), together with five emerging-market agencies: Brazil, Russia, India, China, and Mexico (BRIC-M), current to June 2026. Four convergence gaps recur across all eight jurisdictions: unstandardized potency assay validation, inconsistent post-change comparability expectations, uneven ICH guideline implementation, and divergent evidentiary thresholds for small-population trials. Convergence readiness varies sharply within the emerging-market group, from ICH Regulatory Membership and internationally benchmarked CMC guidance in China and Brazil to reference-country reliance in Mexico and observer status in Russia and India. On this basis, we propose the Global CGT Regulatory Convergence Framework (GCRC-F), a reference architecture of four independently adoptable pillars: (i) Unified CMC Standards, (ii) a Data Harmonization Layer for long-term follow-up and real-world evidence, (iii) an Adaptive Approval Layer linking accelerated designations across agencies, and (iv) a Manufacturing Standardization Layer that is built on existing regulatory precedents rather than novel instruments, with participation tiered by demonstrated regulatory-science maturity. The framework is offered as a structured proposal for discussion; it has not been evaluated by regulators or industry stakeholders, and its feasibility remains to be tested through the consultation and case-study methods identified.
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Regulatory Convergence in Cell and Gene Therapy: Harmonizing Quality, CMC, and Approval Pathways Across the FDA, EMA, PMDA, and Emerging Markets. — 科研速览 Science Skim