Global harmonization in advanced therapeutics: balancing innovation, safety, and access.

Introduction: Advanced Therapy Medicinal Products (ATMPs), which include gene therapies, somatic cell therapies, and tissue-engineered products, are a new paradigm for treating previously intractable diseases. Their regenerative and personalized approach makes them, unlike conventional treatments, require changing regulatory systems to adjust to their intricacies.

Areas covered: This review gives a comprehensive critique of international regulatory programs that include the FDA's RMAT designation, EMA's PRIME program, and Japan's Sakigake program intended to bring ATMPs to patients faster while ensuring patient safety. It also considers innovation-led strategies like adaptive licensing, rolling reviews, and real-world evidence (RWE) led decision-making for pre-market authorization and post-market monitoring. In addition, it discusses problems like regulatory divergence, intricate manufacturing standards, price constraints, and the transformative role of digital technologies such as artificial intelligence and blockchain in traceability and regulatory compliance. Patient-centric models and early access schemes are also extensively debated as part and parcel of the future of regulatory science.

Expert opinion/commentary: To achieve the maximum potential of ATMPs across the world, regulatory systems need to be harmonized and responsive, involving real-time data analysis, flexible approval processes, and improved stakeholder cooperation. New technologies, coupled with more patient engagement and global convergence efforts, are crucial for providing equal access to effective and safe advanced therapies.