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Life Saving Drugs Program – Paroxysmal nocturnal haemoglobinuria – Medicines review protocol

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Publication date: 
4 July 2019
Publication type: 
Procedure
Intended audience: 
General public
Description: 

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

  • prevalence of paroxysmal nocturnal haemoglobinuria (PNH) in Australia
  • management of PNH 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 PNH
  • use of the LSDP PNH medicines
  • developing technologies that may impact future access.