Pharmac’s clinical experts recommend funding Trikafta

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Pharmac today announces its clinical advisory committee PTAC has recommended funding cystic fibrosis treatment Trikafta with medium priority for over 12s.

“Our Pharmacology and Therapeutics Advisory Committee (PTAC) has recommended, with medium priority, that Pharmac fund Trikafta for those over the age of 12,” says Pharmac’s Director of Operations Lisa Williams. “PTAC are waiting to make a recommendation about funding for people aged between six and 11 until further evidence has been provided.”

“PTAC has assessed the evidence and provided additional advice to us, which will help determine where the application is ranked, against all other medicines we would like to fund.”

PTAC’s recommendation is different to Pharmac’s Respiratory Advisory Committee’s recommendation received in August 2021. The Respiratory Subcommittee recommended, with high priority, that Pharmac fund Trikafta for all those 6 years of age and older.

“We now have two options to assess for funding Trikafta. The recommended clinical criteria are similar, but one option is to fund those 6 years and over, and the other is to fund those aged over 12,” says Ms Williams.

In providing advice to Pharmac, PTAC and the Specialist Advisory Committees each bring different expertise and perspectives.

“Both committees bring an evidence-based clinical perspective. Specialist Advisory Committees offer incredibly valuable insight about how new medicines would impact real people in New Zealand that need them. PTAC provides advice to help Pharmac make decisions across the broad range of illnesses and diseases that medicines can treat. It's made up of senior health practitioners from a range of specialities, who also regularly work with patients and their families.”

“PTAC and Specialist Advisory Committees may assess applications and proposals differently, and occasionally provide different recommendations. Pharmac takes the advice of all its experts into consideration when assessing, comparing and making decisions about which medicines to fund.”

“We have already determined that Trikafta is a medicine we would like to fund. We’ll now use the recommendations from both our Respiratory Advisory Committee and PTAC, to review the ranking of funding for all those 6 years of age and older on our Options for Investment (OFI) list and at the same time we’ll add and rank a new option – funding for those aged 12 and over.”

“We can’t shy away from the fact that it’s a very expensive medicine. While Pharmac can negotiate some of the best prices for medicines in the world, when faced with a medicine which has a patent with such a long patent life, such negotiations can be challenging.”

“Before it could be funded by Pharmac, we would need a deal with the supplier and enough money in our budget to fund it.”

“As of today, there are 115 applications on our OFI list. They would all benefit patients if funded. This means we need to make some difficult choices and why prioritisation of these medicines is incredibly important,” concludes Ms Williams.

How prioritisation works at Pharmac

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Cystic fibrosis is an inherited life-threatening disorder that damages the lungs and digestive system.

Trikafta is a combination of three different medicines - elexacaftor, tezacaftor and ivacaftor. It is supplied by pharmaceutical company Vertex.

In July last year, Vertex applied to Pharmac for Trikafta to be funded for people aged over 6 with cystic fibrosis.

We have followed our usual process – we sought clinical advice from our expert advisors, and we undertook an economic health assessment.

Media release: Cystic fibrosis medicine added to the Options for Investment List

Our Respiratory Specialist Advisory Committee recommended in August 2021 that Pharmac fund Trikafta for those aged six years and over with high priority. PTAC looked at the application in November 2021 and recommended that Pharmac fund Trikafta for those aged 12 years and over with medium priority. We’ve published the clinical advice we’ve received on our website.

Check the Application Tracker entry for Trikafta(external link)

Trikafta was granted regulatory approval by Medsafe in December 2021.

Product detail sheet for Trikafta - Medsafe website(external link)