Socio-legal considerations in advanced therapy medicinal product regulation: a review of the evaluation literature

Advanced Therapy Medicinal Products (ATMPs) represent a pivotal advancement in healthcare, offering potential solutions for severe and life-threatening diseases. However, the dynamic landscape of ATMP development poses challenges in regulatory frameworks, necessitating comprehensive evaluation. This study aims to explore socio-legal perspectives on ATMPs, assessing risks and benefits within regulatory paradigms. A thorough literature review spanning 2009 to 2023 was conducted, synthesising empirical research to comprehend ATMP utilisation and regulatory approaches' impact on patient health. Employing multiple sampling strategies, 58 relevant studies were identified, with eight meeting inclusion criteria for qualitative analysis. Perspectives from clinicians, researchers, and industry experts underscored ATMPs' potential to address high unmet medical needs while highlighting concerns regarding clinical safety, efficacy demonstration, and long-term effects. Risk assessments highlighted uncertainties surrounding clinical safety, treatment benefits, and manufacturing consistency, underscoring the multifaceted nature of ATMP risk evaluation. The study advocates for a nuanced regulatory approach, allowing sufficient time to address uncertainties and ensure patient safety. In conclusion, while ATMPs offer promising therapeutic avenues, their complexity necessitates hard regulatory frameworks informed by empirical evidence and stakeholder perspectives to optimise patient outcomes and mitigate potential risks associated with ATMP utilisation. The article proposes amendments to EU legislation regarding the manufacturing practice of ATMP, and further incorporation of these changes into Ukrainian legislation.

advanced therapy medicinal products, patient health impact, orphan diseases, risk assessment methodology, clinical safety uncertainties