Advanced Therapy Medicinal Products (ATMPs), including gene, cell, and tissue-engineered therapies, are gaining increasing prominence in regulatory submissions and health technology assessment (HTA) as they often offer the potential for significant disease modification. Despite this promise, the evidence available at market entry often does not meet the level of robustness typically expected by regulators and HTA bodies.
ATMPs frequently target very small and highly specific patient populations where the viability of traditional randomised controlled trial (RCT) designs is reduced, increasing reliance on single-arm studies. Factors such as these create significant gaps in understanding long-term safety, durability of effect, and real-world effectiveness.
In this Centre of Excellence in Regulatory Science and Innovation for Advanced Therapies (CERSI-AT) white paper, we provide a landscape review of regulatory and HTA guidance, demonstrating how Real-World Data (RWD) can be used to strengthen ATMP evidence and highlighting opportunities for optimisation.
