Innovative Medicine: The Science and the Regulatory Framework
Scientific progress is providing new opportunities to respond to medical needs as yet unmet, in particular with regard to novel and more efficacious treatments for neglected pathologies such as genetic and rare disorders. In Europe, developments have been delayed in the past because of regional differences in the regulatory environment and the consequent market fragmentation this causes. Not only advances in treatment have dramatically outpaced changes in the financial and social structure of healthcare, but medicines that tackle much less common pathologies are often not developed due to the risks involved with regard to investment return. Stimulated by the intensive debate surrounding these disparities, this book (which is a collection of presentations from the II Foresight Training Course organised by the Gianni Benzi Foundation) examines the issues of developing innovative medicines in Europe. It describes the initiatives being implemented by EMA to facilitate the activities in this complex environment, such as the Advanced Therapies Initiative, the Orphan Drugs Initiative, and includes presentations covering other topics such as the promotion of innovation at a national level, the role of pharmaceutical companies in health research and the balance between treatment opportunities and budget control. Thanks to the contribution of experts and regulatory operators from the Task Force in Europe for Drug Development for Young (TEDDY), the book also presents the most recent advances in Paediatric Medicines. Of interest to medical professionals, policy makers, regulators and those working in pharmaceutical research, this book provides a valuable overview of the situation with regard to the development of innovative medicines, in Europe and beyond.