Don’t we just love the way PHARMAC puts a price on our heads and our government keeps a hands off approach even though PHARMAC is a government entity? Welcome to the merry-go-round!
Medicines for rare disorders
In June 2018, PHARMAC called for supplier funding applications for medicines for rare disorders.
By the 3 September 2018 deadline, we had received 13 supplier applications. In some cases, more than one application was received for the same medicine and indication from different suppliers (such applications will be considered together). See below for a full list of medicines, indications and supplier applications.
These applications will be considered at the Rare Disorders Subcommittee’s inaugural meeting in November 2018.
The Subcommittee will make recommendations to PHARMAC about whether the treatments should be funded or not. Minutes of the Subcommittee’s discussion will be published on our website and Application Tracker. PHARMAC may decide it also needs advice from PTAC on the applications.
Following clinical advice and completion of our assessment processes, including prioiritisation, PHARMAC will then determine the next appropriate steps for the application, such as commercial processes.
Applications received
As you can see, there is an application in for Myozyme for Pompe disease. Of course we are in competition with all of the other people who desperately need medicines.
Who will the “lucky winner” be this year?
| Application | Indication | Supplier |
| Agalsidase alfa (Replagal) | Fabry Disease | Shire |
| Alglucosidase alfa (Myozyme) | Late-onset Pompe disease | Sanofi Genzyme |
| Carglumic Acid | Hyperammonaemia due to urea cycle disorders | Max Health and Te Arai BioFarma |
| Elosulfase (Vimizim) | Mucopolysaccharidosis (MPS) type IVA | BioMarin |
| Ivacaftor (Kalydeco) | Cystic fibrosis – with G551D mutation | Vertex |
| Migalastat (Galafold) | Fabry disease | Amicus |
| Miglustat | Gaucher disease | Te Arai BioFarma |
| Miglustat | Niemann Pick Type | Te Arai BioFarma |
| Nitisinone | Tyrosinaemia type 1 | Max Health and Te Arai BioFarma |
| Nusinersen (Spinraza) | Spinal muscular atrophy | Biogen |
| Teduglutide (Revestive) | Short bowel syndrome intestinal failure | Shire |