Life Saving Drugs Program – Pompe disease – Medicines review protocol
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The reviewers will use data sources specified in this document and results of stakeholder consultation to describe:
- prevalence of Pompe disease in Australia
- management of Pompe 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 Pompe disease
- use of the LSDP Pompe disease medicines
- developing technologies that may impact future access.