Regulatory landscape for CAR-T therapy development in the Brazilian context.

Abstract

Introduction: Chimeric antigen receptor T (CAR-T) therapy represents a significant advance in the treatment of hematologic malignancies, yet its global implementation remains limited by regulatory, logistical, and manufacturing challenges. Understanding differences among international regulatory frameworks is essential for expanding safe and equitable access, particularly in emerging academic programs.

Areas covered: This review examines the regulatory pathways, manufacturing requirements, and quality standards governing CAR-T therapies in Brazil, comparing them with those in the United States and the European Union. A focused literature search encompassed peer-reviewed articles, institutional guidelines, and regulatory documents from 2016 to 2025. Key elements assessed include GMP expectations, analytical method validation, biosafety considerations, and infrastructure requirements. The analysis also explores expanded-access mechanisms and summarizes clinical experiences that have used these pathways in the real world.

Expert opinion: Despite structural differences, all jurisdictions converge on core principles emphasizing product quality, long-term safety, and traceability. However, Brazil faces unique multi-agency coordination requirements and infrastructural constraints that may delay national deployment. Global evidence indicates that strengthening GMP facilities, analytical validation strategies, and harmonized regulatory processes, particularly for academic and point-of-care manufacturing, will be crucial to broaden access and sustain innovation in CAR-T therapies.