Te Arai Rare Disorders Medicines recommended for PHARMAC funding
The PHARMAC Rare Disorders Subcommittee met November 5 and 6 2018 to consider the funding of Medicines for the treatment of Rare Disorders. Rare Disorders are defined as those affecting less than 1 in 50,000 New Zealanders, often these Medicines are referred to a Orphan Medicines. The subcommittee can recommend a Medicine is funded, decline funding or defer a decision to a later date.
Of the total 11 Medicines considered two Te Arai BioFarma Medicines received a recommendation for funding with a high priority: Carglumic acid (for the treatment of metabolic disorders CPS1 and NAGs) and nitisinone (for the treatment of hereditary type 1 tyrosinaemia). Two further Medicines were recommended for funding with a medium priority for the treatment of cystic fibrosis and fabry disease. The remaining Medicines were declined (6) or deferred (1).
The complete subcommittee minutes are available here : Nov 5 and 6 2018