Successful registration of new therapies in the EU and USA

摘要: Abstract. This chapter summarises the main regulatory frameworks operating in major pharmaceutical markets as operated by Regulatory Authorities EU, USA and with a brief reference to Japan. There is high degree of convergence requirements for marketing authorisation under International Convention on Harmonisation but there are still important differences content procedures that need be taken into account. A huge amount detailed information now available on-line from agencies. Considerable time care required navigate through different processes. constantly evolving light science, experience novel treatments new insights assessment techniques quality, safety or efficacy emerge. Clinical trial design also process constant evolution. It vital sponsoring companies discuss their development programmes relevant authorities prior embarking them. Likewise, regulators looking ways facilitate path clinic treatments, while bearing mind primary duty protect public unproven unsafe treatments. Taking lead other countries, EU has introduced orphan legislation incentivises develop drugs rare diseases. On occasion, normally due scarcity patients, these medicines can approved reduced data package although same regulations guidelines product apply conventional products. The biosimilars comprehensive ‘Advanced Therapies’. Other changes recently include those relating centralised pharmacovigilance. Food Drug Administration been going rather turbulent period an increasingly closer relationship European Medicines Agency, sometimes resulting having global agreed both agencies almost simultaneously. FDA seen providing better options fast track approval some cases. Engagement best ongoing dialogue which plans discussed than end-of-term examination where submission either passed rejected. In summary, affairs wide-ranging discipline well advised take early advice internal advisors external experts.