Keytruda not proven, Pharmac boss says

Pharmac chief executive Steffan Crausaz says Keytruda hasn’t been proven to help people live longer lives and it isn’t in the top tier of drugs it would like to see funded; about 20 other drugs are a higher priority.

Those 20 drugs include medicines for viral and bacterial infections, vaccines for shingles and chicken pox and other cancer drugs.

Yet “even though the Keytruda drug hasn’t demonstrated benefits in terms of helping those people live longer… we’re actually working with that company and the other company to see if we can resolve the issues," he says.

Pharmac has called on Merck Sharp & Dohme to cut its margin on Keytruda to increase the chance of funding, he says, adding “If the price were lower, we could help resolve some of the issues.”

Another melanoma drug, Opdivo, is likely to be ready “shortly” and Pharmac will start studying it in the next “couple of months," the chief executive says.

RAW DATA: Lisa Owen interviews Pharmac chief executive Steffan Crausaz on The Nation

Lisa Owen: Now we’re turning first today to the debate over Keytruda. For, some it’s a life-saving wonder drug, for others an expensive treatment without proof of long-term benefits. On Tuesday melanoma sufferers presented petitions with 45,000 signatures to Parliament, calling for the medicine to be publicly funded. But what do those with the power to decide say? Well, drug-funding agency Pharmac has so far given Keytruda a low priority. Its chief executive, Steffan Crausaz, is with me now. Good morning.

Steffan Crausaz: Good morning.

Let’s clear up a few things first before we get into this. If you want to fund Keytruda or any new drug, do you have to defund another?

Well, that’s not the case. If I can just explain the context around how we do go about making decisions, essentially there're two things that Pharmac does. Firstly, with its $800 million budget, it’s looking to achieve savings from its existing medicines that we fund. That frees up about 50 million new dollars every year to spend on new investments and new treatments. The other thing that we then do is with any new funding that’s provided for the budget and those savings, what we then look at is how do we best use it? And the process is for 40 to 70 applications a year for funding new medicines is those applications go to senior medics in New Zealand, so in the case of Keytruda, for example, the nine senior cancer doctors help us review that. And then we go through and we do some further analysis on it.

So to get a new drug funded, would another one necessarily have to be defunded?

No, that’s not the case. What Pharmac typically is able to do is to— through its processes that have been well established over more than 20 years, able to reduce the prices of existing treatments by such an extent that we actually free up large sums of money that is then able to be reinvested into new treatments. And obviously the discussion at the moment is about whether that will be Keytruda or whether it would be another drug that would be in a similar class for the treatment of melanoma.

Okay, I do want to ask about that, but also one other thing is that you talked about the committees that consider these drugs, so what is it that they are looking at? The number of people who have the disease, the cost? What factors are the main deciding features?

Yeah, so the main factors for those clinical committees is to look at the clinical evidence that the drug companies put together in their development of the product, and they’re looking to see what is the magnitude of the benefit, what is the nature of the benefit, do people actually live longer and better lives if they receive these treatments, and then to balance that up against, yes, the resource uses, what the cost would be and any other factors like how it will be used in practice in the New Zealand clinical setting, which can be different from elsewhere.

So are there other life-saving drugs that you would like to fund but simply are not because you cannot afford it?

Well, we do get 40 to 70 applications every year for new medicines, so perhaps to put that into a bit of context around Keytruda, the committees have reviewed the evidence, and in their view Keytruda hasn’t been proven to help people live longer lives. It does have some effects on tumour size and tumour progression, and that’s important, but what they really want to see is whether it actually helps people live longer and live better.

So are there actually other drugs as good as Keytruda or better? You know, because the Health Minister has acknowledged that Keytruda could save about 120 lives a year. But are there others ahead of that that are better and there’s more evidence that suggests they’re better value for money?

Yeah, so after that review, they’ve said this isn’t in the top tier of medicines that we would like to see funded. That’s why they said compared to everything else, a low priority. The context is—

But how many? How many—?

About 20 at the moment we’re actively working on would have a high-priority recommendation from those committees.

But they’re not necessarily cancer drugs, are they?

Well, there are some cancer drugs, certainly. There are also medicines for infections, both viral and bacterial, and a whole range of other things like vaccines for things like shingles and chicken pox and other things. So there are a lot of options.

But shingles – because the thing is people will think, ‘Shingles? That’s not a life-threatening disease, not like melanoma.’

Yeah, so there are range of different types of treatments that have different sorts of effects. So absolutely melanoma in the late stage is a life-threatening disease. There are other cancer treatments that we also have that we’re evaluating that, if you like, are in the same sort of situation. They’re life-saving treatments potentially, and the question is – does the evidence that’s put together actually show that that’s the case? And that’s really quite important when what we’re trying to do at Pharmac is fairly evaluate all of the options and really come up with things that are going to make the biggest difference and the best impact for New Zealand patients.

So what you’re saying is actually it’s not all just about the money. In fact, you’ve said that there is a gap between how good the public think the drug is and what the tests showed. What do you mean by that exactly?

Well, I think also it’s not just about Keytruda. So Keytruda is the issue that’s been in the spotlight –the medicine has been in the spotlight. But there is actually another treatment that, if you like, hits the same cellular button, supplied by another company. It’s not quite available yet, but it’s called Opdivo. That’s its brand name. It’s going to be available really quite shortly, I suspect, in New Zealand, and we’re interested in that because that will help us, I think, answer many of the questions about these types of treatments.

So are you more interested in that drug than you are in Keytruda? Do you think it’s a better option?

We’re as interested. It will have a new data set that will help us, I think, unravel some of these questions about – do these treatments actually extend survival?

So are you about to study that or look into that in more detail?

Yes. That’s going to happen over the next short while – a couple of months.

A couple of months? Because some people, obviously… Time is of the essence for a lot of people here, so they want decisions now, don’t they?

Well, we can absolutely understand the folks with late-stage melanoma in a pressing clinical situation. That’s why even though the Keytruda drug hasn’t demonstrated benefits in terms of helping those people live longer, even though that’s not the case, we’ve been quite clear that we’re actually working with that company and the other company to see if we can resolve the issues. So what really needs to happen is…

Yeah, but the problem with that is people at home will look and say – if you had funded this at the first opportunity, you could’ve saved, I think it was, 100, 120 lives a year. Since your decision in September that’s 50 people who have died, isn’t it? It’s saving lives in Australia and in America and in Britain. Are you condemning these people?

I think to agree with that statement, we would have to look at whether this drug actually does save people’s lives – whether it cures people, whether it actually extends folks’ lives in that situation – and the evidence…

So you don’t necessarily think that there would be 50 people still alive as a result of Keytruda?

No, but the data doesn’t show that at all. There’s nothing in that data set that’s come from the company that would lead us to believe that that would be the case. Now, that’s not to say that it ultimately isn’t. All we can say is based on the studies, based on the advice from the cancer doctors, that that isn’t happening.

But the people on the steps of Parliament this week, they are convinced that they need that drug and that it is life-sustaining, so where are they getting that impression from?

Well, I mean, anyone in that situation would want to receive access to a new treatment. I think that would be accepted. When we look at what the data actually shows, it shows responses in tumours, so the tumours shrink or they disappear entirely – maybe they’re actually still there but just can’t be seen – so these are called partial, incomplete responses in the clinical trial terminology. It’s not certain that that necessarily translates, in the end, into people living longer and better. And so this has actually been looked at very recently, end of last year, in the United States – approvals through the FDA for cancer drugs based on tumour responses and other early data; how many of them actually ended up, in the end, showing benefits in terms of overall survival – and out of 36, there was only five that actually translated all the way through. So I think in the melanoma situation, what we’ve got is a really hard-to-treat tumour type, and there’s a lot of interest, actually and including from Pharmac, about the possibility, because we can see activity in terms of the tumours – there’s a lot of interest in that – but what we really need to see, given that we are weighing up a lot of options, many of them also demonstrated to extend and improve life, that’s what Pharmac’s weighing up.

So the people who’ve signed the petition, the people who desperately want this, they’re misinformed?

Well, I think that they’ve read the promising studies and they believe that this is a chance for them, whatever, you know, in the end, the clinical trials ultimately play out… I think that’s… anyone in that position would do the same. Pharmac’s role, though, is… Given all the range of options, we have to do and obtain the best possible results from all the medicines that are available to us with the funding that’s provided. That’s Pharmac’s role.

Okay, well, the nub of this is if you had the money right now, would you fund Keytruda?

More money will always help Pharmac make more decisions. That’s absolutely the case. The issues with Keytruda are also deeper than that, because we need to address the question about the ethicacy, whether this translates into something, in terms of survival, that has to be resolved.

So what piece of proof would you definitively need to say, ‘Okay, the money’s going on this now’?

Well, the studies are ongoing. I think that could happen in two ways – one is either, yes, we get the evidence out of the existing trials as they continue to report, and that is under immediate and constant review. Actually, a lot of these issues that are in there are resolvable commercially as well, and so that’s why Pharmac as said we’ve actually been talking with both companies, setting out what we think would be necessary in order to progress these treatments.

So to be clear, when you say they could be resolved commercially, what you mean is that there is a really high price tag on Keytruda, isn’t there? So it would help if the company lowered its expectations in terms of the money it wants to get?

Yeah, well, I mean, absolutely. A simple lower price absolutely helps, but there are other mechanisms that we have around things like targeting towards people who benefit most and demonstrate those sorts of things. So…

So you are urging Merck Sharp & Dohme to cut their margin, aren’t you?

Well, what we’re saying is at least we could come to a view that perhaps could be pushing Keytruda or any of those other treatments higher up the list, putting into a bracket where we could get funding available for it if we can address those issues, and actually, we only need…

You’re asking them to cut their price, aren’t you?

We’re actually asking them to work with us to resolve those issues, and whether it’s Keytruda or whether it’s another treatment…

Yes or no? Are you asking them to cut…?

Yeah, absolutely.

So are you saying that you’d be more prepared to take a calculated risk on this drug if it was cheaper?

Well, that’s actually the nature of what Pharmac deals with day in, day out – is balancing up what the evidence tells you with what you’re able to achieve commercially, because, you know, we know that if we accept that we can’t fund everything in every situation at any price that the companies asked, we can get lower prices, we can actually fund more things. So, yes, absolutely, if the price was lower, we could help resolve some of the issues. It’s not the only issue, though.

We’re running out of time, and I just want to get through these two. There have been suggestions that patients have been used for PR. Are you comfortable with the company’s role in this, the drug company’s role in that?

Well, I think the companies will always do what they’re to do, which is to maximise the returns to their shareholders, and I would leave it to other folks to have a view on how they think of the company’s behaviour, whether it’s appropriate or not.

What about the politicians wading in, then? Because politicians weighed in over Herceptin; now it’s Keytruda. How do you feel about that?

Well, Pharmac’s role, basically, is to take the funding that is available, maximise the benefits for patients in New Zealand, so we don’t really mind what the drug companies do. There’s obviously debate, and it’s good and legitimate debate, because these are really serious issues that Pharmac deals with.

All right. Thanks for joining us this morning. Appreciate your time.