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Pharmac Simplifies Access To A Medicine For The Rare Disorder, Wilson Disease

Pharmac – Te Pātaka Whaioranga is making it easier for people with Wilson disease to get the medicine they need.

From 1 May, trientine will be listed on the Pharmaceutical Schedule. At the moment people access it through one of Pharmac’s exceptional circumstances pathways, specifically the

Named Patient Pharmaceutical Assessment (NPPA) pathway.

“We want to make it easier for people to get access to the medicines they need where we can,” says Adrienne Martin, Pharmac’s Manager Pharmaceutical Funding.

“Shifting access to trientene from our exceptional circumstances pathway to the Pharmaceutical Schedule means people and prescribers will have clarity on who its funded for and simplifies how people get it. It will also mean their approvals for funding will be life-long. This might seem like a small thing to change but we know it’ll help the people taking and prescribing this medicine.”

Pharmac’s Rare Disorders Advisory Committee have provided advice on eligibility criteria for trientene for the small group of people who have Wilson disease, so the medicine can be listed on the Pharmaceutical Schedule.

Wilson disease is a rare, inherited condition that causes a build-up of copper in a person’s body. This build-up in the liver and other organs causes fatigue and abdominal pain, and the person may eventually require a liver transplant. The medicine, trientine helps remove copper from the body.

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Under the exceptional circumstances pathway, prescribers apply for trientine on behalf of their patients, specifying how the person’s circumstances meet the

NPPA Policy principles

. This takes time for prescribers and time for Pharmac to assess. Funding trientine through the Pharmaceutical Schedule removes these delays in accessing treatment.

Pharmac has not previously had a contract with a supplier for this medicine. Trientine was included in an Annual Tender process, which involves suppliers bidding to be the main contracted suppliers of various funded medicines. Having a contracted trientene product means it can be made available through the Pharmaceutical Schedule so pharmacies and people who need it will have certainty of supply.

“This change is going to reduce the load on prescribers and the stress on people with Wilson disease and their whānau, as the whole process will be more straightforward to deal with,” says Martin.

A very small number of people currently accessing trientine through NPPA will need to change brands. We will be in touch with their prescribers directly, to make sure they have the support needed to help with the change.

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