Funding Trikafta for kiwifruit with cystic fibrosis – Setting the record straight

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On Monday, March 28, Carmen Shanks presented her parliamentary petition to MP Shanan Halbert, urging the government to fund Trikafta, the first drug capable of treating the cause of cystic fibrosis for the majority of sufferers.

Since the presentation of Carmen’s petition, cystic fibrosis and Trikafta have been the subject of numerous reports on various channels. Although this has raised awareness, Cystic Fibrosis NZ is disappointed that some of the discussions include statements that are simply incorrect.

“If Trikafta is to get a fair hearing, it’s essential that the correct information is shared,” says Lisa Burns, chief executive of Cystic Fibrosis NZ. “To ensure that we are all working from the same facts, Cystic Fibrosis NZ has verified the statements that have been made and described our understanding of the true position.”

What is the total cost of Trikafta?

Several different figures have been mentioned in relation to the total cost of Trikafta and not all of them are correct. The total cost of Trikafta for one person for one year in New Zealand is approximately $330,000 NZD excl. GST.

Will Pharmac have to pay the full cost of Trikafta?

Pharmac consistently claims that it can negotiate some of the best drug prices in the world. Vertex has already brokered deals for Trikafta with 31 other countries, including Australia.

Pharmac and Vertex have brokered a deal for Kalydeco, a drug that treats a small percentage of people with cystic fibrosis in New Zealand who have certain genetic mutations. Pharmac confirmed when announcing Kalydeco’s listing that a confidential discount applied which reduced the net price to the funder.

It therefore seems entirely plausible that Pharmac and Vertex could broker a deal to fund Trikafta at less than the full cost of the drug.

There are therapeutic treatments for cystic fibrosis

There are over 540 people in New Zealand with cystic fibrosis. Only about 36 of them (7%) have publicly funded access to Kalydeco, a therapy that addresses the cause of their condition.

This means the majority of Kiwis with cystic fibrosis only have access to treatments that help manage symptoms, such as severe lung infections. These are primarily nebulized saline solution, IV antibiotics, and dornase alfa (Pulmozyne). While some were able to access Trikafta through private funding, only a very small number participate in Vertex’s managed access program, and their health must have been severely compromised before they were eligible.

Existing treatments that manage the effects on their own, and which take three to four hours a day, do not prevent the inevitable decline and in some cases early death. As complications develop, lung function declines, organ damage increases, and the burden or treatment increases dramatically. The current situation without Trikafta means that a lung transplant is their last hope. A transplant is not guaranteed and, for some, not even an option.

Expert clinical advisors from Pharmac, its respiratory subcommittee, have called Trikafta “a paradigm-shifting treatment for patients with cystic fibrosis, in that it treats the cause of cystic fibrosis rather than its symptoms.”

Are all costs included in Pharmac’s cost-benefit analysis for drugs?

Pharmac uses cost-utility analysis to assess which drugs to fund. This analysis considers benefits such as improved quality and length of life, and costs such as pharmaceutical costs, hospitalization of inpatients, and other health system costs such as blood tests. laboratory and diagnostic services, specialist visits and primary care services.

However, Pharmac’s cost-utility analysis does not provide a complete picture of the costs that a disease like cystic fibrosis imposes on patients and their families. For example, the Pharmac Economic Analysis Handbook states:

“Direct patient healthcare costs do not include:

  • salary lost due to illness
  • cost of premature mortality
  • non-government subsidized costs such as private hospital, physiotherapy or non-subsidized pharmaceuticals”.

“Examples of indirect patient costs that should be excluded from costs are:

  • cost of patient or caregiver downtime (i.e. lost wages) and reduced productivity costs
  • cost of premature mortality
  • intangible costs (e.g. pain and suffering experienced as a result of treatment)”

People with cystic fibrosis, their families and whānau incur significant financial costs in these excluded cost categories, for example lost wages for people with cystic fibrosis and their carers and loss of work opportunities. Costs such as hours spent on daily treatments, trips to and from hospital, frequent hospitalizations, and the fact that a parent or caregiver has to give up work to care for a sick family member are all excluded.

Uncounted costs are not considered in the cost-utility analysis undertaken by Pharmac when evaluating a drug for funding, but are borne by people with CF and their families.

Pharmac indicates that it considers these impacts in its Consideration Factors, but this is only qualitative and can in no way adequately represent the heavy financial and economic burden borne by people with cystic fibrosis and their families. As a result, Pharmac’s analysis significantly underestimates the true benefit of funding a drug like Trikafta.

Are Pharmac’s negotiations with Vertex to finance Trikafta underway?

The true status of trade negotiations between Pharmac and Vertex over Trikafta remains unclear.

Vertex says, “it is ready to negotiate and explore creative and innovative financing solutions,” while Pharmac says, “discussions to secure funding for the drug are ongoing.”

However, Pharmac’s own process requires that a drug be prioritized and then identified as part of the list that Pharmac wishes to advance, before price negotiations can be entered into with a supplier.

Cystic Fibrosis NZ understands that Trikafta has currently been prioritized for funding and included in the list of investment options, but the next steps in Pharmac’s process – Identify and Negotiate – have yet to commence.

Following the presentation of her petition to Parliament, Carmen Shanks called for an urgent hearing by the Health Select Committee into the funding of Trikafta.

Cystic Fibrosis NZ endorses Carmen’s appeal and has submitted a request for support to the Committee calling on the government to hear firsthand from people living with cystic fibrosis about Trikafta’s urgent need for public funding.

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