Life Saving Drugs Program – Gaucher disease (type 1) – Medicines review protocol

This final protocol describes the methodology reviewers will follow to assess use of enzyme replacement medicines under the Life Saving Drugs Program medicines for Gaucher disease (type 1).

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Life Saving Drugs Program – Gaucher disease (type 1) – Medicines review protocol

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Publication date:
Publication type:
Procedure
Audience:
General public
Language:
English
Description:

The reviewers will use data sources specified in this document and results of stakeholder consultation to describe:

  • prevalence of Gaucher disease (type 1) in Australia
  • management of Gaucher disease in comparison to Life Saving Drugs Program (LSDP) guidelines
  • clinical and comparative effectiveness and safety of medicines
  • relevant patient-based outcomes
  • value for money of LSDP treatment for Gaucher disease
  • use of the LSDP Gaucher disease medicines
  • developing technologies that may impact future access.

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