Why a purposeful approach to the PIP pays dividends

Efficiently adapting regulatory compliance requirements from the Food and Drug Administration (FDA) to the European Medicines Agency (EMA) across nonclinical, clinical, and quality sections of the Paediatric Investigation Plan (PIP) is essential. While the FDA has strict requirements around studies in children – through the initial Paediatric Study Plan (iPSP) and, more recently, the Paediatric […]

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 […]