Joint European Medicines Agency / Food and Drug Administration workshop for paediatric Gaucher disease type I: exploring the way forward

• Date:
  Monday, 17 September 2012, All day
• Location:
  European Medicines Agency, Amsterdam, the Netherlands

Food and Drug Administration colleagues joined forces with the European Medicines Agency to host industry- and patient-organisation representatives for a unique workshop to discuss the current situation of medicine development in paediatric Gaucher disease. Participants were encouraged to consider collaborative and innovative thinking to address the current therapeutic needs in this rare disorder. Registration closed.

Documents

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.

Minutes of the joint European Medicines Agency / Food and Drug Administration workshop for paediatric Gaucher disease type I: exploring the way forward, 17-18 September 2012

AdoptedReference Number: EMA/610397/2012

An alternative approach to drug development in children with Gaucher disease: a collaborative proposal from the European Medicines Agency and the Food and Drug Administration

Reference Number: EMA/754506/2012

Presentation - European Medicines Agency-Food and Drug Administration Gaucher's disease workshop: Opening remarks (Andrew E. Mulberg)

Presentation - Orphan medicinal products in the European centralised procedure: current marketing authorisations for Gaucher disease (Andrea Taft)

Presentation - Orphan medicinal products in the United States: current marketing authorisations for Gaucher disease (Carla Epps)

Presentation - Background to the meeting: paediatric investigation plans for Gaucher (Elin Haf Davies)

Presentation - Patients' and families' priorities: Gaucher disease (Tanya Collin-Histed)