A medicine heralded for its efficacy in managing cystic fibrosis, often branded as Trikafta, has now been added to Pharmac's priority list for funding, the agency announced on Wednesday morning.
In July this year, American biopharmaceutical company, Vertex Pharmaceuticals, applied to Pharmac for its triple combination modulator therapy to be funded for people aged six and over with cystic fibrosis, an inherited, life-threatening disorder that damages the lungs and digestive system. Cystic fibrosis affects the cells that produce mucus, sweat and digestive juices.
The drug, either branded as Trikafta or Kaftrio, is a combination of three different medicines - elexacaftor, tezacaftor and ivacaftor. It is currently not funded in New Zealand.
"We've added this medicine to our options for investment list," Pharmac's director of operations, Lisa Williams, said on Wednesday. "We followed our usual process – we sought clinical advice from our Respiratory Subcommittee, and we undertook an economic health assessment."
Next month, Pharmac's Pharmacology and Therapeutics Advisory Committee (PTAC) will assess the evidence and provide additional advice to determine where the application is ranked against the other medicines the agency would like to fund.
"Our clinical advisers tell us if a medicine has the evidence to back up the claims made by pharmaceutical companies. Our respiratory experts have reviewed this evidence and recommended that Pharmac fund Vertex's triple combination modulator therapy. They gave this a high priority," Williams said.
"We've published their clinical advice on our website. Using this information and our own economic health assessment, we have determined this is a medicine we would like to fund."
Before Trikafta could be funded by Pharmac, the agency would need a deal with the supplier and enough money in its budget for funding. Pharmac would also need to ask New Zealanders for their thoughts before any final decision to fund the medicine is made.
"We can't shy away from the fact that it's a very expensive medicine," Williams said. "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.
"As of today, there are 114 applications on our options for investment (OFI) list, and we would like to fund them all. 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."
Vertex applied to New Zealand's medicines and medical devices regulator, Medsafe, for market approval of their medicine in June 2021. Medsafe's website shows they issued their first request for information to Vertex on October 15, 2021. It has been granted priority assessment status.
Cystic Fibrosis New Zealand has been campaigning for public funding for Trikafta. A petition to the Health Select Committee has amassed more than 26,000 signatures and is set to close on October 31, 2021.
"Trikafta is a breakthrough treatment widely heralded as having the potential to turn cystic fibrosis from a life-threatening condition, to a manageable condition," it says.
The genetic condition currently affects more than 540 people in New Zealand.
