NICE turns down "life saving" drug for liver cancer.

[MrJustAbout]

The National Institute of Health and Clinical Excellence (NICE) is getting a kicking - again - for saying no to a new cancer drug called nexavar.

news.bbc.co.uk/1/hi/health/8367614.stm

The drug looks like it costs about £35,000 per year. At one level, this doesn't look like much to "save" a life, although; a) it doesn't work for everyone and b) it doesn't "save" anyone's life but just gives them an extra 6 months or so.

I can understand that people might want to give people a few more months of life. NICE already puts a lot more money into doing this, and they'll pay about twice as much, per month as they normally would to give people a little more life when they're close to death.

In end of life, NICE also requires much worse evidence of benefit in order to pay for a drug. Importantly, you don't need to show, for instance, that drugs acutally give you a reasonable quality of life - just a pulse is enough. Given this, there's no way we can talk about it providing for a good death - indeed, it gives absolutely no value to a drug rid that's associated with better quality of life in those it treats.

If we're going to treat people who are dying differently than everyone else, I would have thought it would be more important to give them a decent life in the time that they've got left rather than any life, just for the sake of living. Is this fair?

So, NICE already pays over twice as much for these sorts of last-chance therapies and requires worse evience but nexavar still doesn't get funded. To me, this says something about just unreasonable the drug company is being in demanding £35,000 per year.

No one really wants to put a price on keeping someone alive at all costs. To fund nexavar means that other people have to have treatment taken away. This means that other people would probably have to die - and more people than we'd "save" for a few months on using nexavar. If two people with inoperable brain cancer die so that one person with liver cancer lives then I really don't see that this would be fair.

Cancer isn't fair - it's nasty and kills those we love. NICE seems to be doing all it possibly can to accommodate cancer treating drugs. I just can't see how NICE can be held responsible for a company demanding a lot more than the NHS can pay. We hear from NICE, we hear from patients, but we never hear from the companies justifying the massive fees that they ask for.

In order for it not to be funded at 50p, it'd generally have really poor evidence - basically, to the degree that it just doesn't work or that you'd need to buy it for thousands of people in order to get it to work for one. Unfortunately, that was pretty much the issue with the Alzheimers drugs ... and I think everyone wishes that they did work.

For 6 months more in even pretty poor health, you're normally talking upwards of £5k minimum before anyone even thinks about questioning it.

In terms of quality of life - NICE tries to keep those sorts of judgements away from doctors because, as you say, they're not the best people to comment. Normally, it's a mixture of patient's saying how bad a condition is and information from the public saying how much weight to give them.

I agree Longtall and especially about the thing of making their money on one or two blockbuster drugs.

I still don't understand why they need to aim for, say, a 15% return on a product for a few people. Why not 2% on that and 18% on the blockbuster? There's nothing at all to suggest that the profits should be the same on all treatments ...

I'm willing to admit I might be wrong and they might just be trying to break even on that product. Ultimately, either what it costs to produce is above what NICE is able to pay or it isn't? If it costs more than NICE can pay, it's probably efficient that it's not funded - and if it costs less than what NICE can pay, then the problem seems to come down to the company asking a lot more than it costs to produce. Even if they couldn't break even on that product, I would have thought that making back 50% of your R&D costs is better than making back none of them.

There was a very good series on r4 called Inside the Ethics Committee (I think still available on listen again) and it dealt very well with the tensions involved in medical ethics. It made me see that a good death is actually more important than prolonging life. Much as we might instinctively cling to the latter. Worth a listen.

Justabout - I think you're underestimating the number of blue rinses!

Doctors are involved but it's probably safer to assume that patients know what it's like to be a patient more than their doctor does. Especially when that doctor is a mister, and likes cutting things out/off :-)

2 issues fro me here.

1. I get itchy at the idea of Drs being the ones best place to judge quality of life.

That represents a total devotion to science, and the science of medicine. It places a huge trust in the institution, in the training of medics, and the ethics of medics. I am not convinced I have seen enough evidence that these assumptions are safe. I am not saying I believe that this not the case, but that I would like evidence it is.

I think lay people, spiritual groups, expert witnesses, patients, society in all it's forms, may have something to contribute on this.

I have a vauge feeling that those who reach the stage of making decisions in NICE will have in some way proved they do not simply believe 'patient ill, illness bad, cure illness or patient may as well be dead'. I really hope so at any rate.

2. If a drug at 50p is worth 6 months of life, why is it not worth it at £30, £300, £30k.....

If it isn't worth it at £30k, then we have to be very honest and say plainly that we cannot afford a life prolonging drug. It is really important to be truthfull, and not dress it up in quality of life type lipstick. It is what it is, a financial decision. Let us be plain. If that is the truth then lets say it. How society feels about this truth is a debate that needs to happen.

Does this breakfast club do pancakes?

treedelivery:
"If it isn't worth it at £30k, then we have to be very honest and say plainly that we cannot afford a life prolonging drug."

Well that's essentially what NICE does.

It weighs cost against benefit. And sometimes the benefit outweighs the cost, and sometimes it's the other way around.

I don't think they are trying to obscure that conclusion.

And the quality of life argument IS important. If a drug gives you 6 months of relatively normal health to spend at home with your family and then a fast deterioration, that's worth a lot more to a patient than giving them 6 months half-dead and unconscious on a ventilator.

That's not lipstick - it's a very important part of the argument from the point of view of the patient as well as the NHS.

And NICE committees aren't only made up of doctors - they include patients and lay-people, as well as professionals from other disciplines.

I'm not sure how the different committees work on the different parts of the NICE remit, but I know that patients and lay-people are involved in coming up with the NICE treatment guidelines, for eg.

btw am I too late for a bacon sarnie? Brown sauce please, and I like the bacon crispy.

theyoungvisitor - I am not saying that we are in the situation of applying lipstick - sorry that wasn't clear. Just that we should be gaurded we don't go there in the very difficult years to come. I would be disgusted if economic decisions were dressed up as mercy on the suffering victims. By, say....ooo..the media and the party in power at the time.

I agree Nice do come out and say it is down to cost. I actually like that about them - they do seem honest. I feel they do a good job in that they fulfil their brief.

I am unsure how society feels about the brief though, and if this is what modern medicine should be about. On a very base level, I rise against anything that takes away choice. We are actively involved in removing choice from patients, in this instance on the grounds of cost. I understand that, I really do, but I find my hackles raise all the same.

"I rise against anything that takes away choice. We are actively involved in removing choice from patients, in this instance on the grounds of cost."

But choice in this case comes down to not just what the patient can choose, but what society chooses for the illness as a whole.

In a health service with limited funds, one patient's right to choose an exorbitant drug may mean another patient dies because there are not enough nurses, or a premature baby is denied a SCUBU place because funding isn't there.

You shouldn't have a right to choose if your choices harm other people.

Greatest good for the greatest number and all that.

I have issues with QALYs. Far from a perfect measure. But the only thing we've got - how on earth do you measure something as nebulous and subjective as quality of life? It's not really something that is reducible to figures, tbh.

Also hope the quality of debate has improved considerably since I sat on the DH committee that recommended drugs for NICE appraisal (as a lay person). I was astonished to see how rubbish it was. Basically at the level of someone down the pub. You do need to take into account what the ordinary man or woman in the street would think about a decision, of course, but it really was crap.

There is also an issue about PCTs using the fact a drug has or hasn't been referred to NICE as reason to deny funding - while a drug is in the queue or because it's not in the queue at all yet (like any other organisation NICE can only do X amount of work in Y time).

Then there's drug company pricing. Very mysterious. They keep arguing that developing a new molecule costs lots of money, which is true, but drug companies spend more on marketing than they do on R&D.

In the end, the fact we have NICE probably means more people benefit than would lose out if we didn't have it. Because most information about effectiveness and side effects comes at the post-marketing stage. Other countries introduce new drugs much faster than us, so we get to see all their data about how many patients are harmed and which groups of patients benefit.

Doesn't mean NICE decisions in each case are perfect, though.

Edam - in terms of QALYs there are definitely issues there, and some of the health economists that came up with them are their fiercest critics.

Having said that it is as you've said - they are the only things we've got and quality of life is nebulous. The only real arguments in favour is that at least they reflect the sorts of judgmeents that people say that they'd make for themselves. For instance, 50% quality of life might correspond (using one method) to a state where you'd be willing to flip a coin - heads you're cured, tails you're dead.

I'd be very interested to hear about the committee recommending stuff for NICE. How much of the time did you discuss new things (possible investment), and how much of the time did you discuss existing treatments (possible disinvestment)?

I don't think PCTs can refuse treamtent because NICE hasn't looked at it yet anymore - I think this stopped with Herceptin. It's a shame though because I can't see that the PCTs can easily make these judgements - they are hard enough for NICE and there just aren't enough "experts" to go around.

I definitely agree that NICE is imperfect ... but they don't deserve all the kickings they get.

"drug companies spend more on marketing than they do on R&D"

Which sounds dreadful - until you consider that after making a discovery, if no-one knows to prescribe it, to make a fuss for it to be made available on the state, to ask their private healthcare insurance for it - it's never going to make its money back.

I was reading the other day that drugs companies are preparing to meet the government halfway on some drugs though. That in my opinion is what should be happening - the NHS is a huge customer, and rather than just saying "yes" or "no" - should say, well we wouldn't pay £30k a month for it, but we think £15k is reasonable. Or whatever.

"Then there's drug company pricing. Very mysterious. They keep arguing that developing a new molecule costs lots of money, which is true, but drug companies spend more on marketing than they do on R&D."

I'm not a big fan of drug companies but I have to stick up for them here and say that that's comparing apples and bananas.

Their profits have to cover their R&D costs (including the costs of those drugs that never make it to market). Marketing increases sales to a level where that profitability is viable. The marketing is done only up to a point where the amount spent is more than reflected in increased sales, after that it's pointless.

If they didn't spend so much on marketing they wouldn't have more to spend on R&D, they'd have less, because there would be less profits and therefore less research.

Arguably, therefore marketing increases the R&D budget, so it's meaningless to set one off against the other as if they are clear either/or alternatives.

Comparing the amount paid out to shareholders vs the amount reinvested in the business would be interesting though.

NICE isn't allow to do that though - and only very recently has the NHS been able to try to negotiate.

Yes, this is barmy.

The problem is that with the target returns that they demand, the drug industry often declines to be funded at all. Or, as here, they gee up lobby groups to make a fuss when they aren't given what they want.

Justabout - typically, no-one and everyone. It's nobody's specific job and there's massive danger that the voice of those who lose out gets missed. Generally, the health economists try and the chairs of the committees are completely aware of it.

In practical terms you can't have everybody there within the meeting, but it is important to always think about who bears the opportunity cost.

It's also worth noting now that the meetings are public but you can't do anything more than just observe. A lot of the meeting is available to see and it's also now streamed on the internet too I think. Unfortunately, some of the information is commercially sensitive and so nothing can be said to refer to specifics where the drug company doesn't want anyone (read either their competitors or the public, depending on your cynicism) to know just how good, bad, or expensive the drug is. Sometimes this means that the public documents that get produced after the analysis on behalf of NICE end up with pages and pages of redacted material as commercial in confidence.

(Interestingly, the companies are much less keen for NICE's own analysis to be in confidence ... make of that what you will.)

Agree to a point youngvisiter - how much marketing should they really have to do in the UK? Anything NICE says yes to has to be provided up within three months in the NHS (in fact, it's normally immediate) and doctors are pretty good at knowing what the current guidance is. Do "we" need advertising?

I'd also be interested in seeing how much of the marketing budget is spent in countries with a regulator like NICE, and comparing that with the money spent in somewhere like the US where they can advertise to patients. After all, those ads during the superbowl get expensive :-)

A breakdown of what money is spent on too is also important (although I recall someone saying that the rules have got a tighter on what GPs etc can now accept in terms of marketing from industry).

MrJ - no, the public here don't need marketing for prescribable drugs. But they do spend money making health professionals aware of their products and how to prescribe them.

It's also worth noting that many drug companies make the bulk of their profits from over the counter remedies which of course do require advertising as they are consumer led.

And many (like Johnson & Johnson for eg) are financed by toothpaste, baby wipes, cosmetics... nothing to do with pharmaceuticals.

I don't know how far the marketing/R&D budgets are broken down in edam's example - but nobody would object to Johnson & Johnson spending more money marketing baby oil than in researching new varieties of baby oil.

MrJustAbout - I know, some of my best friends are health economists. Boy, do we have entertaining parties! (Actually the ones I know are jolly sociable as it happens.)

The DH committee I sat on was the one that decides which drugs go to Nice for consideration. However, this was a looong time ago - think I left in 2005. So hopefully much more on the ball now. At that time, it seemed to depend whether any docs from that specific clinical area were there to speak up for X group of patients. And there was a huge issue about drugs that would only benefit very small groups would be referred - think that has now been dealt with to some extent.

As for R&D and marketing, yes, I'm aware of the arguments that one depends on the other, but really is it justifiable to spend more on marketing? Especially when you consider that involves setting up 'front' patient groups that are funded by the drug company to press for their drugs? And when drug companies argue that it costs them sooooo much money to bring a new molecule to market?

Maybe we need to get those guys behind Freakonomics to take a look at it. Would be entertaining if nothing else.

btw, drug companies are always pressing to be allowed to advertise drugs direct to consumers - they were trying again recently having been turned down last time. Not sure if any decision has been made yet by the EU.

Has anyone asked yet if MrJustabout and Justabouttoturn35 are related?