FDA and EMA Collaborate on Expandable Model for Rare Pediatric Drug Development

The FDA and the European Medicines Agency released a draft joint plan Monday to support the development of pediatric treatments for Gaucher disease — an approach the agencies say can apply to rare diseases in general.

The draft plan proposes using data extrapolation to avoid unnecessary studies and improve the allocation of resources, and encourages sponsors to make better use of clinical data modelling and simulation techniques to predict how a drug will perform in pediatric populations.

The draft plan proposes safety and efficacy testing of products from different companies in a single clinical study — a multi-arm, multi-company trial, using a single control to reduce the total number of children enrolled. The FDA is expected to publish its own copy of the document in the next few months.