Researchers have called for an urgent overhaul of New Zealand's processes for funding cancer drugs to ensure breakthrough treatments get to those who need them as soon as possible.
A new study in the New Zealand Medical Journal outlined the clinical information on new melanoma treatments and compared the funding programmes in New Zealand, Australia, England and the United States.
Despite drug-funding agency Pharmac now covering two drugs - Opdivo and Keytruda - the issues encountered with funding new melanoma treatments have not been addressed, the authors argued.
Cancer Society medical director Dr Christopher Jackson, who has given a public talk as part of Queenstown Research Week, spoke with Herald science reporter Jamie Morton.
Q. Why are these new drugs so important?
A. Recent advances in science have completely changed the treatment of melanoma.
What we know is that a third to a half of melanoma is driven by certain mutations inside genes, which can be very well targeted by new drugs, called targeted therapies, and these work very well for a period of time.
Separately, there have been new advances in immunotherapy, which work for a greater proportion of patients - they work for 70 per cent - and they use the body's own immune system to fight the cancer.
Both of these are major advances in science and have led to major improvements in the clinical outcomes of patients with advanced melanoma.
Q. How responsive has the New Zealand research community and the cancer society been to them - and what recommendations have they made to Pharmac?
A. The Cancer Society, as well as the New Zealand scientific and clinical communities are eager to collaborate with Pharmac.
Pharmac has its own evaluations committee which looks at the evidence and they don't have to engage with scientists or clinicians.
That's one of the things the Cancer Society has been advocating for - there should be greater engagement with scientists and clinicians at an earlier stage in the process.
Q. So how long have researchers here themselves been aware of these advances?
A. Preliminary data led to US registration in 2014.
Studies showing improved survival in melanoma with drugs like Keytruda and Opdivo were published in June 2015, and for Ipilimumab the data was published in 2010 but wasn't quite as strong.
Q. Why then has it only been in the last few weeks that we've seen Opdivo become funded, and Pharmac announce Keytruda's funding from next month?
A. Cost is certainly a factor, but Pharmac also said they didn't think the drugs were very good, which scientists, patients and clinicians vigorously disputed.
There is also no limit on the time Pharmac can take to review a drug.
It would have been better for Pharmac to have a process that included patients, clinicians and interested groups that lead to a more robust decision being made.
The Cancer Society also thinks there should be time limits for decisions so there is more certainty for patients.
Q. Do you feel New Zealand has been behind the eight-ball?
A. We have been slower than other countries to get these drugs over the line.
I don't think these drugs still wouldn't be funded without the efforts of the patients affected by melanoma, and by groups like the Cancer Society that supported them.
Q. What do you think needs to change?
A. A number of things need to change.
First of all, there will always be a lag between when the evidence becomes available and funding becomes available, as Pharmac needs to go through its process of complex commercial negotiations.
We've advocated for an early-access to medicines scheme, whereby genuine breakthrough drugs that improve people's life expectancy, or quality of life, by a set and agreed amount, would be able to be funded for a temporary period while Pharmac goes through its processes.
This would mean we would be able to look after those people in that gap between the evidence and full and final Pharmac review.
And after that full review, if the drugs don't reach the cost-effectiveness thresholds, they wouldn't get on the main list and funding would stop.
Secondly, there needs to be a greater engagement of clinicians and scientists at a much earlier point.
Thirdly, there needs to be an appeals process.
So, if Pharmac makes a decision that appears, at face value, to have errors in it, then there needs to be a mechanism for clinicians, patients, the public or patient representative groups like the Cancer Society, to appeal those decisions and ask for them to be looked at again with a greater deal of scrutiny.
We also need to talk about how big the funding pie needs to be.
Is the amount of money we are spending the right amount?
Could it be more, or does it need to be less?
We need to have that discussion as well.
Q. These recommendations were just laid out in the New Zealand Medical Journal. Has there been any response so far?
A. People know that if we do the same thing we will get the same result.
We think that the Pharmac model is good, but not perfect, and could do with a few minor improvements like we've outlined.
Otherwise, we're going to have this debate again.
The Cancer Society's chief executive, Claire Austin, and I, would like to continue to work with Pharmac and help them with these issues so that patients can benefit in the future. We look forward to seeing progress in this area.
Pharmac: "We are moving as quickly as we can"
Pharmac's director of operations, Sarah Fitt, responds:
Pharmac recognises the challenges faced by individual patients, and their understandable desire to access new cancer treatments.
We are moving as quickly as we can, while ensuring our work and processes are robust.
This includes being guided by expert clinical advisers (who themselves are independent of Pharmac) who are all leading doctors in New Zealand with expertise in analysing information from clinical trials.
While we are considering these treatments and the needs of people with cancer, Pharmac also needs to keep in mind other medicines for other conditions, which could benefit different groups of New Zealanders.
So we need to ensure the process we use is fair and reaches the best decision possible for all New Zealanders.
Clinical evidence is a key input into Pharmac's funding decisions.
We engage extensively with independent clinical experts when considering funding decisions for all treatments, including new cancer therapies.
Senior clinicians and experts in cancer therapies sit on our specialist cancer sub-committee, and in addition to their expert advice, we engage with a wide range of national groups including the, the Ministry of Health working groups, regional cancer networks, DHB cancer specialists and staff as well as the Cancer Society.
Pharmac also consults widely about impending decisions, encouraging feedback from a wide cross section of people- including clinicians and patients.
Our decision to fund nivolumab from July 1, 2016, reflected the quality of evidence, confidence in the survival data obtained through those trials, feedback from cancer treatment services and the overall value achieved through commercial negotiations.
Pharmac understands that patients, their families and whanau, and clinicians ideally want the newest medicines in the hope they will provide the best possible health result.
Pharmac, too, wants to invest in new medicines to improve the range and efficacy of medicines available to New Zealanders.
When it comes to "early access" where evidence is still emerging, Pharmac needs to take a careful and robust approach.
This includes being open-minded to new funding arrangements, provided these have been shown to be better for New Zealand's health outcomes over the long term.
When it comes to "early access" schemes, a number of important issues need careful consideration.