ATMPs: What are they and why does real-world evidence matter?

In this summary article, we explore advanced therapy medicinal products (ATMPs) – taking a look at what they are, their role in the context of rare diseases and the place of real-world evidence. Individually rare, collectively common: explaining rare diseases Rare diseases, also called orphan diseases, affect a small percentage of the population yet are of significant interest to a diverse range of stakeholders. These include researchers, healthcare professionals, advocates, and the general public – all with the shared goal of advancing knowledge and improving outcomes for patients with conditions such as cystic fibrosis, Huntington’s disease, and Ehlers Danlos syndrome....