A 'no pedestrians' sign

It's a provisional 'no' from NICE

EDIT 16/05/12 – NICE has now approved this drug. More here.

Today the National Institute of Health and Clinical Excellence – NICE, the body that decides which drugs the NHS should pay for – has given a preliminary ‘thumbs down’ to a new prostate cancer drug, abiraterone (Zytiga), after it failed to agree a pricing scheme with its manufacturer.

As regular readers will remember, we played a key role in this drug’s development, from pioneering lab work, through pre-clinical studies, all the way up to early patient trials.

This is a deeply disappointing and frustrating decision. Since it became available last year, abiraterone has become one of the most requested drugs on the NHS Cancer Drugs Fund. Both patients and doctors alike value the extra months it gives men with their families, if their prostate cancer comes back after chemotherapy.

Chiefly, we’re upset that the healthcare authorities and the drug’s manufacturers, Janssen (part of Johnson & Johnson), can’t agree a fair price for the drug.

We also think there are some issues with NICE’s calculations, which we’ll discuss below.

Thankfully, this decision isn’t final, and can be appealed NICE is now asking for comments through its consultation process. But we’re dismayed that this will mean another long wait of many more months before there’s any hope of progress. And while we wait, there will be men in the UK who will be denied a drug that could help them.

Here’s a video of our chief medical officer, Professor Peter Johnson, explaining what he thinks about the decision:

We need to be clear: abiraterone isn’t a cure for cancer.

But for the thousands of men diagnosed every year with prostate cancer that has spread, it has the potential to ease suffering in the late stages of a terrible disease, and give men more time with their families.  The average survival time for these men is just 11 months. Trials have shown that abiraterone could give them an extra four.

Vested interest?

Some will say that we’re only upset because Cancer Research UK stands to earn money from sales of abiraterone. And we do have an interest here: thanks to the way we licensed our initial discoveries to the pharmaceutical industry, we stand to receive royalties from the drug’s sale.

But those funds would be ploughed back into our urgent search for better ways to treat this terrible disease. We receive no government funding for our research – almost all our income comes from the public’s generosity. Things are tight for everyone, and we’re having to seriously prioritise our research funding. Every penny counts.

On top of this, it’s hard to tell our supporters that a drug whose development they helped to fund isn’t going to be available to patients. More recently, to try to prevent this situation arising, we’ve been much more forensic in contracts we sign with the pharma industry, to encourage them to market the resulting drugs at a price suitable for the NHS.

But we signed the original agreement to develop abiraterone in the late 80s, long before NICE was even a twinkle in the Department of Health’s eye.

Our motive is not self-interest. At Cancer Research UK we passionately believe that to help people with the disease, we have to understand what makes cancer tick, and use that knowledge to develop better treatments.

Abiraterone is a shining example of this vision. In the 1990s, we helped fund researchers at The Institute of Cancer Research to identify the molecular machinery that helps prostate cancers use testosterone to grow and spread. They developed chemicals that could interfere with this process. They tested and refined these chemicals into a drug that could make a difference. And they conducted early trials of this drug.

We watched proudly as others at The Institute of Cancer Research, with Janssen’s support, built on that platform, rigorously testing the drug in clinical trials. These trials proved that the drug could improve things for the people who really matter – patients.

So hearing that men are to be denied this drug as a routine choice is a huge let-down, and a real blow to the morale of everyone who shares our dream of conquering cancer through scientific research.

Where did things go wrong?

First, let’s look at NICE’s calculations.

Around 37,000 men are diagnosed with prostate cancer every year in the UK. Of these, about 10,000 are diagnosed with advanced disease.

But abiraterone is only currently intended for men with advanced prostate cancer who have already had chemo, which isn’t suitable for all men. We’ve spoken to a range of experts who agree that the true figure is probably fewer than 7,000 men – and we think this number is low enough for the drug to be assessed under NICE’s end-of-life guidance, which has less-stringent rules over cost.

So we think the overall cost could be more manageable than NICE’s initial calculations appear to have indicated. They disagree with us, and issued a statement to say so. But all the evidence Janssen gave them backs up our point of view; they appear to have dismissed this on the advice of a single expert.

The drug’s high cost is perhaps a more significant factor. We’re well aware that abiraterone is an expensive drug* – it costs about £3,000 for one month’s supply – and we live in straitened times. But the challenges of the current economic climate need to be shared. Janssen have offered the NHS a discount, but clearly not enough. We want them to agree a discount scheme that reflects the current financial constraints on the NHS.

If NICE tweak their sums, and Janssen are willing to compromise, we desperately hope abiraterone will be allowed to enter routine use.

Men in England with advanced prostate cancer can, of course, consider asking their doctor to apply for access to abiraterone through the Cancer Drugs Fund. But this is a finite pot of money that’s only available until 2014. It has to service the needs of patients with many other types of cancer. We mustn’t overload it, or others will suffer.

And crucially, the Fund doesn’t apply to men in Scotland, Wales or Northern Ireland (the Scottish drugs watchdog, the SMC, say they aim to appraise abiraterone by March 12th).

How long will the appeal take?

Appeals need to be submitted NICE want to hear from other experts by February 23rd, and say a final decision should be made by May. And this highlights a wider problem – NICE’s system still appears to be overly reliant on economic, rather than clinical, values. As we discussed in December, the Government is planning to reform drug pricing in the UK, through a proposal called ‘value-based pricing’. We want this new system, however it works, to be faster, more transparent, and focused on delivering positive outcomes for patients.

But that’s for the future. In the here-and-now, we’ll be consulting with our colleagues at other clinical and research organisations. If you have anything you want us to highlight, please leave your thoughts below. As well as people affected by prostate cancer, we’re also keen to hear from the people who look after them – clinicians, nurses, GPs and other carers.

We hope that the appeal is successful, responses to this consultation convince NICE and Janssen that the drug represents a real step forward for men with prostate cancer, so it can be made available on the NHS for those could benefit from it.

Henry

*Edit 03/02 – we’ve added more details about the cost of abiraterone above, in response to a request on Twitter

Edit 06/0 – after discussion with NICE, we have slightly amended the wording of a few points above.