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5 Feb

Kalydeco and other CF drugs – what happens now?


Wednesday 5 February 2020

On Friday 24th January, the New Zealand CF community received the exciting, and long-awaited, news that Vertex and Pharmac had reached a provisional deal to supply Kalydeco.

Now that the dust has settled on the news, what happens next? And what about the other CF modulator drugs still not funded?


First of all, what exactly is Kalydeco?

Ivacaftor, known by its brand name Kalydeco, is the first medication that fixes the underlying cystic fibrosis defect, essentially turning off cystic fibrosis. Kalydeco represents a breakthrough in cystic fibrosis research; current medications only treat the symptoms.

Kalydeco is effective in anyone with at least one of the following nine Class 3 gating mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D, or those with the R117H mutation.

When will Kalydeco become available?

The funding is still subject to a final consultation, which went out last week to health professionals. If the feedback from the consultation is supportive and the proposal is approved, then people could be prescribed Kalydeco from 1 March this year.

It is proposed that the drug will be made available via DHBs and not community pharmacies.

As part of this process both CFNZ and Kalydeco for Kiwis will each be submitting a letter of support, together with a joint submission from CFNZ’s Clinical Advisory Panel, Paediatric Society CF Clinical Network and Starship Cystic Fibrosis Healthcare team. Pharmac indicated that every piece of feedback they receive in relation to a funding proposal must be reviewed and a report filed. If there are a large number of submissions, this can mean a huge body of work and possible delays in the process of funding a medicine.

With that in mind, we ask that if you have something you would like to submit, please send it to either or, and we will add it to our submission. The information on the consultation process is listed on the Pharmac website, and the closing date for submissions is 10 February. We ask that any comments are provided by 6 February latest.

Why did it take so long to get Kalydeco in comparison with other countries?

Drug funding is a complicated issue and each body responsible for assessing and recommending (such as PHARMAC in NZ, NICE in the UK, and PBAC in Australia) each have different rules, criteria and budget thresholds to work with.

With a list price of around $360,000 per patient, per year, Kalydeco is one of the most expensive drugs available. Pharmac declined the initial application for Kalydeco back in 2014 citing high cost and limited clinical data.

In contrast, many other OECD countries approved funding for Kalydeco for ages six and over based on the same evidence and list price between 2013 and 2015. Expanded use for two-to-five-year olds was secured in subsequent years.

Some countries, such as the UK, use a system whereby if you meet a certain threshold of benefit it will fund the drug. A commonly used assessment is QALYs or Quality-Adjusted Life Years, which measures the length and quality of life. In the UK, a medicine is funded if it works out at about £30,000 per QALY gained, making the process of funding formulaic and transparent.

Pharmac has no specific level of health benefit which triggers funding. The Pharmac process looks at four areas, the 'factors of consideration' - the need, the health benefits, the costs and savings and the suitability of the medicine, which also includes the use of QALYs. However, Pharmac itself says the factors "are not weighted or applied rigidly" and that "not every factor is relevant for every funding decision" it makes.

Currently New Zealand spends less on medication per capita, waits longer to buy new drugs, and registers fewer new medicines than almost any other nation in the OECD. The IQVIA International Comparisons of Modern Medicines report, commissioned by Medicines New Zealand, ranked New Zealand last for both access to funded medicines and pharmaceutical investment overall.

All these factors combined has made it extremely difficult and time-consuming to secure funding for Kalydeco.

On a positive note, we are pleased that Pharmac has shown the foresight to fund Kalydeco in New Zealand with no age restriction. In Australia for example, Kalydeco is only funded for 12 months and above. Up until August 2019 it was only funded for ages two and above.


It’s clear there’s a bigger issue with Pharmac – what are you doing to change it?

CFNZ supports the Patient Voice Aotearoa petition to double the Pharmac budget and call for reform of its investment model. A larger budget and funding model more in line with that of other OECD countries is an essential prerequisite for the funding of Orkambi and other CF medications in the short-term future.

We encourage as many people to sign it as possible before it closes on 22 July 2020. The more signatures we can gather, the louder our voices will be and the better chance of being heard.

Why didn’t you lobby for Orkambi, Symdeko and Trikafta at the same time, or just skip to Trikafta which could help more people?

There are a few different reasons we campaigned for Kalydeco exclusively in the first instance.

Kalydeco has over a decade of strong, irrefutable data on its benefits for people with CF and presented a solid argument for funding. Given that Kalydeco was declined on lack of evidence in 2014, this indicated that Pharmac was unlikely to consider Orkambi, Symdeko, or Trikafta seriously with its limited years of clinical data.

In addition to this, Vertex will not look at any of these other medications until it has reached an agreement on Kalydeco.

This means that getting Kalydeco funded is a critical first step to accessing these other medications. Once we have had the final green light for Kalydeco, we will encourage Vertex to submit an application to Pharmac for Orkambi and begin a concerted effort to secure this drug.

Many countries have also negotiated pipeline or ‘portfolio’ deals for access to multiple Vertex drugs over a period of years. These deals provide a suite of existing and future drugs that are likely to be developed over the lifetime of the deal. In the Republic of Ireland this pipeline deal has secured them access to Trikafta once the drug receives approval from the European Medicines Agency (estimated to be in 4-5 months’ time).

A pipeline deal for multiple drugs would certainly provide an ideal solution for us in New Zealand. However, our understanding is the current Pharmac funding model and year-by-year budget does not allow for these types of deals. This is another excellent reason to sign the petition calling for reform of the Pharmac drug-buying model.

Can I lodge my own application to get Orkambi / Symdeko / Trikafta funded?

Anyone can lodge an application with Pharmac for drug funding. However, for the application to be considered it needs to be fully supported by the manufacturer with the full documentation on clinical and cost evidence.

Once we have the final greenlight for Kalydeco, we will encourage Vertex to submit an application to Pharmac for Orkambi and begin a concerted effort alongside the CF community to secure this drug, as we did with Kalydeco.

How can I get involved with the efforts to secure funding for Orkambi / Symdeko / Trikafta?

Our current focus is to support the final consultation process and ensure that Kalydeco is made available on March 1 with no further delay. It is incredibly important that we do not jeopardise or complicate this step.

Once Kalydeco is in the hands of those eligible and that stage is firmly complete, we will develop thenext stage of our advocacy campaign for Orkambi, Symdeko and Trikafta and campaign details will be made available at that point.

We’ve also set up a Facebook group – Access for Aotearoa – which we encourage you to join so that we have an army prepared when the fight for the other CF drugs kicks-off.

You can join here:

Lastly, the campaign for Kalydeco was incredibly compelling because it featured Eddie and Emma fighting for their son Otis. We’re on the lookout for families who would benefit from any of these unfunded precision medications who would be comfortable sharing their story. This may be in the form of TV, radio and newspaper interviews, providing written evidence on day-to-day life with CF or supplying photos and/or quotes for us to use. Please email if you might be interested.

What else is happening to move advocacy for CF medicines forward?

Our CF research strategy is encouraging more clinical trials to take place in New Zealand. Several Pharmaceuticals have expressed interest in using New Zealand as a trial site.

The New Zealand CF data registry, Port CF, is in excellent shape and has proven to be a valuable advocacy tool. This anonymous data will allow us to give accurate information on possible trial sites.

Late in 2019 we gave Deloitte New Zealand the green light to begin collecting information for the Social and Economic Cost of CF report. This report will look at health sector and treatment costs, as well as productivity losses and the cost of caring for people with CF, both formally and informally. It will also consider more qualitative data such as loss of wellbeing and the impact of premature mortality on families.

While we recently closed the survey for feedback from the CF community, Deloitte has suggested that more data would assist in the robustness of the report. We are holding the survey open for an additional two weeks and encourage anyone who hasn’t completed it to please do so.

Learn more and take the survey here:

To date there has been no economic or social cost data to provide to Pharmac or the Government, so this will be an invaluable tool to improve care in New Zealand, as well as in securing Orkambi, Symdeko and Trikafta.

Have you got any other questions?

We’re happy to answer them! Contact Chief Executive Jane Bollard by emailing or by calling the office on 09 308 9161.