Advanced Therapy Medicinal Products: an introduction to risk management

Abstract

Introduction: Advanced therapy products include gene therapy, somatic cell therapy, and tissue engineering products that promise important health benefits. These products contain active cells or genetic constructs that exert a mechanism of metabolic, immunological, genetic or pharmacological action. Objective: To discuss main risks involved in advanced therapy products to understand risk management regulatory models and practices. Method: Review in the scientific literature and in official documents of the regulatory agencies of the United States and Europe. Results: Advanced therapy products can be difficult to
define, particularly cell-based products. Completely elucidating the mechanisms of action contributes to mitigate risk of development and characterization, including through the development of disease models or other functional assays. Disease severity, predicted benefit level and safety profile will affect the number of participants and other design
aspects of each test. They present a high degree of technical complexity and substantial challenges to their manufacture. Conclusions: Major regulatory agencies demonstrate efforts to establish clear rules for the preparation of advanced cellular products compatible with Good Manufacturing Practices and the conduct of clinical trials in order to rationalize requirements adapted to the specific characteristics of advanced therapy products.