This morning, the UK’s drug guidance body, NICE, announced its preliminary decision on whether the NHS should use four new cancer drugs – bevacizumab, sorafenib, sunitinib and temsirolimus – to treat people with kidney cancer that has spread (‘metastatic renal cell carcinoma’).

Despite clinical evidence that these drugs can actually help, NICE has decided that they’re too expensive. In essence, NICE doesn’t think that these four drugs are value-for-money for the NHS.

We’re very disappointed with this decision, and we feel it raises fundamental questions about how NICE evaluates cancer drugs, particularly for cancers that only affect a relatively small number of people.

NICE have stressed that this is a preliminary decision, and are undergoing a public consultation. Cancer Research UK will be making representations to NICE on the matter.

That’s why we’d like to hear what you have to say – if you have a strong opinion on this, please leave your comments below.

Clinical trials for rare diseases

The gold-standard method of testing whether a treatment works and is safe is the clinical trial – a careful look at how a new treatment compares against the treatments currently in use.

The more people enrolled on a clinical trial, and the longer it lasts, the more sure researchers can be about its results.

But only about a couple of thousand people every year are diagnosed with metastatic renal cell cancer*. And only one in ten people diagnosed with this stage of the disease is alive five years later.

So, for relatively rare diseases like this, it can take a long time to run trials large enough to gather watertight evidence about how well new treatments work.

No other treatment options

Currently, the only available treatment for metastatic renal cell cancer is immunotherapy. This halts the disease’s progress for just four months on average. But if people are unsuitable for immunotherapy, or it doesn’t work, that’s it. There’s no other treatment option.

So doctors urgently need new treatments for this disease. And the four drugs NICE has rejected have shown considerable promise in clinical trials.

New generation therapies

These four drugs are part of a new generation of cancer drugs, developed after years of painstaking research. They target key processes within the body that get hijacked when cancer develops.

Trials looking at whether these drugs can help people with metastatic renal cell cancer to live longer have had extremely encouraging results. NICE’s assessment contains details of several such trials.

In fact, several of the trials were stopped early, to allow those people not receiving the new treatment to have it. Other trials showed that some of these drugs could stop the cancer from growing for several months more than immunotherapy alone. That doesn’t seem much, but when you’re trying to beat cancer, those extra months can mean a lot.

NICE agreed that patients tended to live longer when they were given these drugs. But they felt that the evidence wasn’t sufficiently robust. And when they put the data from the trials into their computer models, they found that the drugs cost a lot (£20,000 – £35,000 per patient per year) compared to the benefit they brought patients – too much for them to recommend that the NHS prescribes these drugs.

Our concerns

These computer models were developed to look at giving drugs to large numbers of people. We question whether they’re valid for looking at relatively uncommon diseases like kidney cancer that has spread.

We’re also worried that NICE is setting the bar too high with regard to the strength of evidence they require to approve these drugs. Doctors don’t have a lot to offer people with advanced kidney cancer. If these drugs can help them – and the clinical trials show that they do – shouldn’t they be made available?

Share your views

We’re keen to work with NICE to improve their drug assessment mechanism for ‘orphan’ diseases – diseases that affect relatively few people. But we want to hear from you. If you’d like to share your views on this decision, please email [email protected] or post your comments below.

Worried?

If you have questions about cancer or its treatment, including kidney cancer, please visit our patient information website, CancerHelp – or contact our cancer information nurses. There’s also more information on NICE’s decision on advanced kidney cancer on CancerHelp UK

Henry

*A note on the stats:

According to the figures used from the NICE report, for 2006, 17 per cent of renal cell cancer cases were stage 4 disease, i.e. metastatic. If we were to apply this figure to the 2005 cancer incidence figures that we have published for the UK then this would mean that there are fewer than 1,100 cases of metastatic renal cell carcinomas in the UK each year.

However, data from the South East of England in 2005 showed that a quarter of kidney cancers were diagnosed with distant metastases, with a third of cases having unknown stage. If the South East of England was representative of the UK then we could assume that there are least 1,500 cases of metastatic renal cell carcinomas. But if the unstaged tumours followed the same distribution as those that are staged then this could rise to as many as 2,250 cases per year.

However, assumptions have been made with all these figures, which produce very different results. This underlines the necessity to have excellent quality staging data to assess just how many people are affected by distant metastases, for all cancer sites – not just renal cell carcinomas.

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Cancer in the news Treatment Health service policy Kidney cancer Policy