Life Saving Drugs Program – Mucopolysaccharidosis type I (MPS I) – 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 mucopolysaccharidosis type I (MPS I) in Australia
- management of MPS I 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 MPS I
- use of the LSDP MPS I medicines
- developing technologies that may impact future access.