Year in Review: Improving access to rare disorders medicines

PHARMAC is committed to improving New Zealanders’ access to medicines for rare disorders. To help with this, we have adjusted our funding application process for rare disorders treatments.

30 to 35 people to access ivacaftor

Unlike other medicines, rare disorders medicines are not required to have gained Medsafe approval before they can be considered by PHARMAC for funding. This makes it easier for pharmaceutical suppliers to apply to PHARMAC for their medicines to be funded. We are continuing to engage with people who have rare disorders and their advocates to understand their views. The insights we gain help inform our work to deliver the best outcomes for all New Zealanders. 

New medicine for people with a rare form of cystic fibrosis

In 2019/20 we were delighted to announce that an estimated 30 - 35 adults and children with a rare form of cystic fibrosis would be able to access ivacaftor (marketed as Kalydeco) for the treatment of their condition.

“This medicine treats the disease rather than the symptoms and has the potential to make a real difference to the lives of New Zealanders living with this rare type of cystic fibrosis,” explains PHARMAC director of operations Lisa Williams.

New medicine for people with severe haemophilia A

10 to 15 people to access emicizumab

Haemophilia A is a hereditary, life-long bleeding disorder. People with haemophilia A cannot form blood clots effectively and bleed more often. This puts them at risk of permanent damage from bleeding into the joints or death from severe internal bleeding.

“Emicizumab is expected to reduce the frequency of bleeding episodes and as a result, hospital visits, which will have important impacts on the quality of life for people who use it,” explains deputy medical director Dr Pete Murray.

This year our clinical advisory committees recommended that emicizumab be funded with a high priority. We were able to fund this medicine for patients from 1 December 2020.

One of the reasons it was given a high priority recommendation was because of the significant impacts this disorder has on the everyday lives of people with severe haemophilia A, and their whānau. This is especially true if the patient is a young child.

Emicizumab is a high-cost medicine. Without PHARMAC funding, if a person paid for this medicine privately, it would cost them over $600,000 a year.