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Life Saving Drugs Program – Gaucher disease (type 1) – Miglustat review plan

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Publication date: 
2 April 2019
Publication type: 
Procedure
Intended audience: 
General public
Description: 

As the uptake of miglustat through the LSDP has been considerably lower than predicted, this separate review will focus on:

  • the place of miglustat in the treatment pathway for patients with Gaucher disease at the time it was added to the LSDP compared to now
  • whether any new evidence for the safety and efficacy of miglustat is available compared to what was considered at the time it was recommended to be added to the LSDP
  • the actual observed usage of miglustat in the time it has been available through the LSDP compared to the predicted usage.