Optimisation strategies for registries from the perspective of UK and EU regulatory and HTA decision-makers
Gene, cell, and tissue-engineered therapies are Advanced Therapy Medicinal Products (ATMPs); which offer the potential for significant disease modification. However, they often enter the market with limited clinical evidence due to small patient populations, and short trial follow-up.
These factors create significant gaps in understanding long-term safety, durability, and real-world effectiveness. Real-world data (RWD) and real-world evidence (RWE) are increasingly critical for addressing these gaps, supporting regulatory and health technology assessment (HTA) decision-making, and informing post-authorisation monitoring, pricing, reimbursement, clinical practice and patient choice.
This Centre of Excellence in Regulatory Science and Innovation for Advanced Therapies (CERSI-AT) whitepaper presents the position of registries within the broader scope of RWD sources, highlighting the existing challenges and distinct opportunities for optimisation.
