BOX 5-2Gaucher Disease: A Strategic Collaborative Approach from EMA and FDA

Based on Pediatric Cluster meetings, the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) published a strategic collaborative approach on Gaucher disease, a rare metabolic disorder that is passed down from parents to their children. Given the number of drugs in the pipeline and based on work of the Pediatric Cluster, EMA and FDA undertook a collaborative approach to lay out the necessary parameters for demonstrating the safety and efficacy of drug products to treat Gaucher disease in pediatric populations. In addition to discussing opportunities for extrapolation of efficacy and use of nonclinical models to strengthen development plans, EMA and FDA explored the possibility of establishing a multi-arm, multi-company development program (EMA, 2017).

This collaborative approach has the potential to streamline the testing of drugs to treat Gaucher disease by avoiding unnecessary pediatric studies and reducing burden on patients. Although developed specifically for Gaucher disease, EMA notes that this type of approach could be applied toward drug development for other rare diseases and conditions (EMA, 2024a).