FDA/EMA and Rare Diseases

Regulatory science is frequently criticised for not keeping up with innovation. As technology evolves at a rapid change, at the same time that patient demands for early access to safe and efficacious treatment - it is a big ask. The European Medicine Agency (EMA) are proactively supporting processes to enable these scenarios. European regulators are only one stakeholder in a global eco-system however; which also involves payers, HTAs, pharmaceutical companies, physicians and patients themselves. The adoption of new innovation by one stakeholder therefore needs to be done in consideration of the impact of all the other. This talk will explore some of those concepts.