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

Current version - Collaborative approach

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

AdoptedReference Number: EMA/237265/2017Summary:

This Strategic Collaborative Approach document is not a formal guidance; the purpose of this document is to facilitate paediatric drug development particularly in the field of Gaucher disease. The general principles presented should be viewed as suggestions only.

Overview of comments received on 'Gaucher disease: a strategic collaborative approach from the European Medicines Agency and Food and Drug Administration' (EMA/44410/2014)

Reference Number: EMA/752905/2016Summary:

This Strategic Collaborative Approach document is not a formal guidance; the purpose of this document is to facilitate paediatric drug development particularly in the field of Gaucher disease. The general principles presented should be viewed as suggestions only.

Gaucher disease: A strategic collaborative approach from European Medicines Agency and Food and Drug Administration

Reference Number: EMA/44410/2014Summary:

The purpose of this Collaborative Approach document is to increase - the chances of rapid and smooth agreement of the Paediatric Investigation Plan (EMA) / Pediatric Study Plan (FDA). In addition, this document 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.