Perspectives

Q&A with Pharma Progress 2021 panelist: Daniel De Schryver, Patient Engagement & Advocacy Lead, Europe, Middle-East and Africa, Janssen

We’ve asked some of our experts to share insights on the challenges posed by rare diseases in healthcare, particularly in the wake of the ongoing COVID-19 pandemic. Each has given their own take based on their experience of working in the rare disease space, based on the impact on payers, pharmaceutical companies and, most importantly, patients.

COVID-19 has intensified many existing trends. One that is particularly relevant to this public health crisis is the need to accelerate the approval of innovative technologies. Even before the pandemic, regulatory initiatives by the FDA and the EMA sought to provide earlier conditional approval of innovative therapies for some rare, chronic and terminal conditions. Similarly, health systems have sought to increase access to new therapies via mechanisms such as the Temporary Use Authorizations in operation in France, the compassionate use programs (Härtefallprogramme) in Germany and the Cancer Drugs Fund in England.