Comparing rare disease applications in the EU and the US

Innovators from markets outside the EU often have many questions about what to expect with the European Medicines Agency (EMA). For US sponsors with rare disease products, both significant and subtle differences can be a minefield when seeking EMA approval. 1. Navigating 27 markets and standards of care First and foremost, while there have been […]

Sampling the buffet of scientific advice with rare disease product development

Sponsors of products for rare diseases must manage a complex environment and often in a much shorter timeframe to meet high unmet need. However, they have one significant advantage: access to free scientific advice from the European Medicines Agency (EMA). Orphan Drug Designation, the PRIME (PRIority MEdicines) scheme, conditional marketing approval – these and other […]

Key steps to proper management of CMC with rare disease products

Meeting requirements for Chemistry, Manufacturing and Control (CMC) is complex for any drug developer, but is even more so in the rare disease space. These challenges are further compounded for non-EU sponsors seeking to initiate clinical trials in Europe where the differences in requirements and expectations from their home market are often not well-understood. Manoeuvring […]