From Laboratory to Patient: The Evolving Path to Become a Medicine

Gene editing and other laboratory developments promise to revolutionize treatment of many diseases that currently have no cure. The process to become a medicine is long and complex and requires many years. Regulation of medicines (permission to market a product and monitoring of its performance) is evolving to adapt to emerging new approaches in development. Current issues and new ideas needed to navigate the difficult waters from laboratory to patient are discussed here: novel approaches for narrow evidence base, earlier discussions of evidence with regulators, earlier planning for post-authorization requirements and better patient access.