Gaucher disease: a strategic collaborative approach from the European Medicines Agency and Food and Drug Administration - Scientific guideline

This document aims to increase the chances of rapid and smooth agreement of the paediatric investigation plan (EMA)/pediatric study plan (FDA). It also discusses the possibility of a multi-arm, multi-company clinical trial for the treatment of Gaucher disease, as one approach to address the feasibility of developing multiple products for a rare disease in a limited timeframe.

Keywords: Gaucher disease, lysosomal storage disorder, enzyme replacement therapy, substrate-reduction therapy