May 9, 2019, 6:00 PM

Kia ora Gill,

I wanted to touch base to let you know that we have published the advice and recommendations from the February 2019 Pharmacology and Therapeutics Advisory Committee (PTAC) meeting.

As you know, a number of medicines for rare disorders were considered by our expert clinical advisors at this meeting. These were initially assessed by our Rare Disorders Subcommittee in November 2018.

PTAC’s recommendations are below. You will note that some of their recommendations are different from those of our Rare Disorders Subcommittee. This means that the priorities for those medicines have changed. I have provided some information as to why, but you are welcome to read the full summary on our website

I can assure you that we are still taking these funding applications through our funding process.

Recommendations from clinical advisors are one just one input to PHARMAC’s decision-making. We have also compared and ranked these funding applications against other funding applications we have received. We do not generally undertake further assessment of funding applications that have been recommended for decline. We will, however, consider any new evidence and information that we may receive.

We cannot give a timeframe for if, or when, we may make a funding decision about any of these funding applications. We will continue to stay in contact with you.

Applications with a changed funding priority

  • Agalsidase alfa (Replagal) for Fabry disease was recommended for decline. Our advisors recommended the priority change from ‘medium’ to ‘recommended for decline’ due to low quality evidence consistent with only modest long-term health benefits.
  • Ivacaftor (Kalydeco) for cystic fibrosis with G551D mutation was recommended for funding with a low priority. Our advisors recommended the priority change from ‘medium’ to ‘low’ due to moderate quality evidence of a health benefit, the limited availability of long-term data, concerns regarding markers of surrogacy, and the high cost.

Recommended for funding with a high priority

  • Carglumic acid for hyperammonaemia due to urea cycle disorders
  • Nitisinone for Tyrosinaemia type 1

Recommended for funding with a medium priority

  • Carglumic acid for organic hyperammonaemias

Recommendation deferred

  • Nusinersen (Spinraza) for spinal muscular atrophy was deferred, pending longer-term follow-up analyses of two trials. We are expecting to receive updated data from the supplier by the end of 2019.

Recommended for decline

  • Alglucosidase alfa (Myozyme) for late-onset Pompe disease
  • Elosulfase (Vimizim) for mucopolysaccharidosis (MPS) type IVA
  • Migalastat (Galafold) for Fabry disease
  • Miglustat for Gaucher disease
  • Miglustat for Niemann Pick Type C
  • Teduglutide (Revestive) for short bowel syndrome intestinal failure.

You may also be interested to know that we have recently called for more funding applications for medicines for rare disorders. These applications will be assessed by our Rare Disorders Subcommittee in September 2019.

I hope this information is helpful for you and your networks.

Ngā mihi nui


Alison Hill| Director Engagement and Implementation

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