Life-changing CF drug goes back to PBAC

5 minute read

The wait to access Trikafta just got much longer for children aged six to 11.

A life-changing cystic fibrosis drug recommended by the Pharmaceutical Benefits Advisory Committee (PBAC) for young children has been sent back to the advisory body after a dispute over uptake and compliance rates.

Cystic Fibrosis Australia said the dispute around the drug, which reduces exacerbations and lung function decline while improving nutrition and survival, appeared to be around metrics over the number of children who will use and continue to use the drug.

At its November meeting last year, the PBAC recommended Trikafta (elexacaftor/ tezacaftor/ ivacaftor) be added to the PBS for children aged six to 11 who have at least one F508del mutation in the CFTR gene.

But the PBAC also said the evidence presented could not accurately quantify the benefits of treating patients with Trikafta from a younger age. However, it acknowledged that treatment from a young age was likely to be beneficial.

In a statement, the manufacturer said they had been taking proactive steps to progress the PBS listing since the November PBAC recommendation.

A spokesperson for Vertex Pharmaceuticals said they submitted a simple, nine-page document on 20 January which included real-world evidence and PBS data demonstrating differences between children and older age groups in relation to uptake and compliance.

“In January, we submitted data to support our estimates of use of Trikafta in young children, with the hope this would be reviewed swiftly. We have now learned it has been referred back to the March PBAC meeting,” a statement by Vertex said.

Minutes from that meeting are due to be communicated to Vertex on 31 March and made publicly available on 21 April. It will then await PBS approval.

The Department of Health and Aged Care said the delay was because the company was not happy with certain details of PBAC’s recommendation. It told ARR in a statement that documentation had been lodged by Vertex and was under consideration.

“Unfortunately, the pharmaceutical company has not accepted the PBAC recommendation in full and is disputing a number of elements. This means that the PBAC has to reconsider the documentation lodged by Vertex, which is effectively an alternative proposal to what was recommended by the PBAC.

“The PBAC has expedited consideration of this alternative proposal to its March 2023 meeting,” it said.

Trikafta is currently only available on the PBS for people aged 12 and over. Without PBS funding the drug costs around $30,000 a month.

“Trikafta is reimbursed for children 6 years and older in 30 other countries around the world. Vertex is committed to continuing to work together with the PBAC and the Department of Health to come to a resolution quickly, without further delay so that children in Australia can also benefit from government funded access,” the manufacturer said in a statement.

The Department has said it will “continue to work with Vertex to move as quickly as possible to make Trikafta available for this age group.”

Cystic Fibrosis Australia chief executive Jo Armstrong said it was devastating news for the cystic fibrosis community.

“In the meantime, kids are getting sicker and they’re the ones who are suffering because bureaucracy just isn’t serving the people it’s meant to protect,” she told ARR.

Ms Armstrong said the timeline for medicines to be listed on the PBS after PBAC meetings was “a complete unknown”.

“This is one of the areas of angst in the community,” she said.

Ms Armstrong said if children had access to Trikafta earlier in life it greatly improved their survival and quality of life.

“The more we can eliminate the long-term damage, the better quality of life they’re going to have and the biggest impact is on younger kids … because they haven’t had as much lung damage as people who are older with cystic fibrosis. They should be the priority. No one should have to wait.”

According to Cystic Fibrosis Australia, there are around 500 children in Australia who would benefit from Trikafta.

“It’s 500 kids who are going to be able to get a full education because they’re not off school sick or in hospital for months on end on intravenous antibiotics, and then they can become citizens who contribute to the community and broader society.”

Ms Armstrong said parents were incredibly stressed about whether their young children with cystic fibrosis would be able to access the drug, such as the parents of a six-year-old.

“He’s really, really sick. He’s missing school and at home in bed and they’re really stressed out with this decision. He desperately needs the medication and they don’t know when, or if, he’ll be able to get it.”

Another family re-mortgaged their home last year so they could buy Trikafta for their daughter. “They were prepared to live in a tent because she was so sick,” Ms Armstrong said.

“When you see your child deteriorating each day, you do whatever you can.”

Ms Armstrong it was devastating for the community that the news came on the same day it was announced that Trikafta would become available in New Zealand to anyone aged six and above from April 1.

“There are 30 other countries with access to Trikafta for six-plus and we’re not one of them. We’re really lagging on a global perspective, unfortunately.”

A spokesperson for Health Minister Mark Butler said: “The Albanese Government will work with the sponsor, Vertex, to move as quickly as possible to make Trikafta available for this age group.”

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