DRUG DEVELOPMENT FROM THE REGULATORY STANDPOINT: EXPERIENCES FROM THE INDUSTRY

This thesis explores the role of regulatory affairs in pharmaceutical drug development, with a focus on pre-marketing authorization procedures in the European Union. The research is based on case studies of two biological medicinal products, riliprubart, a monoclonal antibody targeting complement-mediated diseases and BIVV003, a gene therapy for Sickle Cell Disease, both developed by Sanofi to address unmet medical needs in rare diseases. The thesis underscores the significance of regulatory science in guiding drug development and balancing the attempts to follow innovative pathways with compliance. Regulatory frameworks, such as waivers in Paediatric Investigation Plan, Scientific Advice and Protocol Assistance procedures, and Orphan Drug Designation applications, play an essential role in determining a drug’s approval trajectory. By analyzing these regulatory pathways and their impact on drug development, the thesis aims to provide insights into how a multinational pharmaceutical company navigates the regulatory landscape in the EU.