31 March 2022
On Monday 28 March, Carmen Shanks presented her Parliamentary Petition to MP Shanan Halbert, urging the Government to fund Trikafta, the first medicine that can treat the cause of cystic fibrosis for the majority of those who have the condition.
Since Carmen’s petition was presented, cystic fibrosis and Trikafta have been the source of many news stories across various channels. While this has heightened awareness, Cystic Fibrosis NZ is disappointed that some of the discussions include statements that are simply incorrect.
“If Trikafta is going to get a fair hearing, it’s essential that the correct information is being shared, says Lisa Burns, Chief Executive of Cystic Fibrosis NZ. “To make sure we are all working from the same facts, Cystic Fibrosis NZ has checked the statements that have been made and outlined our understanding of the true position.”
What is the full cost of Trikafta?
Several different numbers have been mentioned in relation to the full cost of Trikafta and not all are correct. The full cost of Trikafta for a person for a year in New Zealand is approximately NZD$330,000 excl. GST.
Will Pharmac have to pay the full cost of Trikafta?
Pharmac consistently states it can negotiate some of the best prices for medicines in the world. Vertex has already negotiated agreements for Trikafta with 31 other countries, including Australia.
Pharmac and Vertex negotiated an agreement for Kalydeco, a medicine that treats a small percentage of people with cystic fibrosis in New Zealand who have certain genetic mutations. Pharmac confirmed when advising the listing of Kalydeco, that a confidential rebate applied, that reduced the net price to the funder. 
It therefore seems completely plausible that Pharmac and Vertex can negotiate an agreement to fund Trikafta at less than the full cost of the medicine.
There are existing therapeutic treatments for cystic fibrosis
There are more than 540 people in New Zealand with cystic fibrosis. Approximately only 36 of them (7%) have publicly funded access to Kalydeco, a therapy that treats the cause of their condition.
That means the majority of Kiwis with cystic fibrosis only have access to treatments that help manage the symptoms, like serious lung infections. These are primarily nebulised saline, IV antibiotics and dornase alfa (Pulmozyne). While some have been able to access Trikafta through private funding, only a very small number are on Vertex’s Managed Access Programme, and their health had to be severely compromised before they were eligible.
The existing treatments that only manage the effects, 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 rises significantly. The current situation without Trikafta means a lung transplant is their final hope. A transplant is not guaranteed and, for some, is not even an option.
Pharmac’s expert clinical advisers, its Respiratory Subcommittee, called Trikafta a “paradigm-shifting treatment for patients with CF, in that it treats the cause of CF rather than its symptoms”.
Are all costs included in Pharmac’s cost-benefit analysis for medicines?
Pharmac uses cost-utility analysis to assess medicines for funding. This analysis includes consideration of benefits such as improvements in quality and length of life, and costs such as pharmaceutical, inpatient hospital, and other health system costs like laboratory and diagnostic tests, specialist visits, and primary care services.
However, Pharmac’s cost-utility analysis does not provide a full picture of the costs that a condition like cystic fibrosis imposes on patients and their families. For example, Pharmac’s economic analysis manual states:
“Direct patient health care costs do not include:
“Examples of indirect patient costs that should be excluded from costs are:
People with cystic fibrosis, their families and whānau bear significant financial costs in these excluded cost categories, for example lost wages for those with cystic fibrosis and their care givers and lost opportunities to work. Costs such as the hours spent doing daily treatments, going to and from hospital appointments, frequent hospitalisations, and a parent or caregiver having to give up work to care for a sick family member are all excluded.
The uncounted costs are not considered in the cost-utility analysis undertaken by Pharmac when assessing a medicine for funding but are borne by those living with cystic fibrosis and their families.
Pharmac advises that it takes such impacts into account in its Factors for Consideration, but this is qualitative only and can in no way adequately represent the major financial and economic burden endured by those with cystic fibrosis and their families. As a result, Pharmac’s analysis significantly underestimates the true benefit of funding a medicine like Trikafta.
Are Pharmac’s negotiations with Vertex to fund Trikafta underway?
The true status of commercial negotiations between Pharmac and Vertex over Trikafta remains unclear.
Vertex says, “it stands ready to negotiate and explore creative and innovative funding solutions,” while Pharmac says “discussions to get the drug funded are underway”.
However, Pharmac’s own process requires that a medicine is prioritised, then identified as one on the list Pharmac wants to take forward, before they can begin price negotiations with a supplier. 
Cystic Fibrosis NZ understands that Trikafta has currently been prioritised for funding and included in the Options for Investment list, but that the subsequent steps in Pharmac’s process - Identify and Negotiate – have yet to commence.
Following the presentation of her petition to Parliament, Carmen Shanks has called for an urgent hearing by the Health Select Committee into the funding of Trikafta.
Cystic Fibrosis NZ endorses Carmen’s call and has submitted a supporting request to the Committee requesting the Government hear first-hand from those living with cystic fibrosis about the urgent need for public funding of Trikafta.
Read Rare Disorders NZ media release here
 Listing of Ivacaftor(Kalydeco) in the Pharmaceutical Schedule for people with cystic fibrosis and certain Class 111 gating mutation. 9 February 2021.
 Record of the Respiratory Subcommittee of PTAC - Meeting held 26 August 2021.