PHARMAC’s Rare Disorder Subcommittee

13 February 2019

The Subcommittee met on 5 and 6 November 2018 and has recommended that four medicines be funded, and five be declined for funding. One medicine was deferred to be considered at a later date.

The recommendations were that the four medicines be funded for use in hyperammonaemia (with two different patient groups), hereditary tyrosinaemia type 1, Fabry disease, and cystic fibrosis in patients with a G551D mutation.

You can read the Subcommittee’s full discussion on PHARMAC’s website:

The next step for these funding applications is for all of the Subcommittee’s recommendations to be reviewed by PHARMAC’s main group of expert clinical advisors, the Pharmacology and Therapeutics Advisory Committee (PTAC) at its February 2019 meeting. Following this, PHARMAC will consider the positive recommendations using their Factors for Consideration framework, and will rank them against other funding applications received.