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Officials REJECT a ‘game-changing’ cancer drug for NHS patients

A row over drugs pricing has delayed a revolution in cancer treatment which could provide hope for thousands of patients.

Health bosses last year promised to fast track what they called ‘game-changing’ new gene-targeted cancer drugs.

Simon Stevens, the chief executive of NHS England, in June said the advantages of these new drugs was ‘potentially huge’ – and pledged that NHS patients would be ‘among the first in the world to benefit’.

But despite last summer’s excitement, NHS watchdog NICE has now issued draft guidance rejecting the first of these drugs on grounds of cost.

Officials said larotrectinib – which costs £15,000 a month – could not be recommended for the NHS on its current price.

Drugs giant Bayer offered a confidential discount but officials said the price had not been lowered far enough to pass the watchdog’s strict cost-effectiveness thresholds.

Officials said larotrectinib – which costs £15,000 a month – could not be recommended for the NHS on its current price

Larotrectinib, which has been available in the US for more than a year and in Europe since September, is the first of a range of drugs which are tailored to the genetic make-up of a patient’s tumour – instead of where the cancer is located in the body.

The treatments are known as ‘tumour agnostic’ or ‘histology independent’ drugs because they can treat a wide range of cancers, regardless of where they are in the body.

This differs from conventional treatment that is based on where the tumour originated – such as in the breast, liver or prostate.

It is a revolutionary approach which offers hope to the many people with rare cancers who are often left with no treatment options because drugs have simply never been trialled for their cancer type.

The new drugs could pave the way for a new era of personalised cancer care – and could eventually help a large proportion of the 360,000 people who are diagnosed with cancer in Britain each year.

Because they are simply based on someone’s genes – and have been trialled across a range of tumours – the drugs could work for anyone with particular genetic mutation, no matter how obscure their cancer.

For example larotrectinib, which targets a rare genetic mutation called NTRK-fusion, has been shown to work for thyroid cancer, lung cancer, soft tissue sarcoma and a range of rarer cancers.

In early trials, tumours responded in two-thirds to three-quarters of the cancers tested – and in some cases the tumours disappeared altogether.


Tumour agnostic drugs are part of a broader approach to treatment called personalised care.

Research has expanded here in recent years due to developments in genomic sequencing technologies.

A tumour agnostic drug can treat any type of cancer, regardless of its origins in the body or the tissue.

Most cancer treatments are developed to treat a cancer that has developed in a specific organ or tissue, like breast cancer or lung cancer. 

But tumour agnostic drugs treats any kind of cancer as long as the cancer has the specific molecular alteration targeted by the drug. 

Findings out the molecular features  of a tumour can help doctors decide which treatments may be best for an individual with cancer, regardless of where the cancer is located or how it looks under the microscope. 

These drugs show promise in clinical trials, with tumours responding in two-thirds to three-quarters of the cancers tested, according to NHS England.

Targetted therapies already exist, such as Pembrolizumab (Keytruda). But these don’t have the potential to treat a wide range of cancers like tumour agnostic drugs do.

It gives hope to patients with rare or difficult to treat cancers.  

Dr David Propper, medical oncologist at Barts and London NHS Trust, said: ‘Treatment with larotrectinib… has the potential to deliver clinically meaningful responses in patients with cancers which are otherwise challenging to treat.

‘It is important that patients who are eligible can get access to this treatment option.’

But the huge flexibility for patients also means it is very hard for NHS officials to work out how cost-effective it would be according to their rigid thresholds.

NICE admitted trials suggest larotrectinib works – but they cannot work out how well according to their usual methods.

‘Evidence from clinical trials on larotrectinib suggests that tumours with NTRK gene fusions shrink in response to treatment.

‘However, it is difficult to know how well these drugs work because they have not been compared with other treatments.’ 

Officials stressed the decision is merely a draft – and more meetings will take place with Bayer to try to reach an agreement.

Meanwhile competitor Roche is close to securing a European licence for another tumour-naive drug called entrectinib.

NICE has already assessed this and hinted at good news – with officials saying it ‘could become the first’ tumour-naive drug to be approved for NHS patients.

Meindert Boysen, director of the centre for health technology evaluation at NICE, said: ‘Histology independent medicines are an exciting new development in cancer care.

‘These cutting-edge therapies target rare genetic mutations, so the clinical evidence is usually based on extremely small sample sizes, requiring novel approaches to testing them in clinical trials.

‘We’re hopeful that further data collection, coupled with responsible pricing from the companies, will lead to progressive, new treatments like these being available to patients.’ 

John Stewart, national director of specialised commissioning at the NHS, said: ‘It is disappointing that Bayer has not yet been willing to price larotrectinib at a level which represents value for the NHS and the taxpayer, however, should they reconsider, the NHS’ door remains open for further discussion.

‘In the meantime, preparation continues for the introduction of this next generation of therapies, including constructive conversations with Roche on a commercial deal for entrectinib.’ 

A Bayer spokesman said the company was ‘disappointed’ by the decision, adding: ‘It is vital that all parties continue to work in close collaboration and demonstrate flexibility so that NHS patients with NTRK fusion genes can benefit from access to a new treatment option as soon as possible.

‘Securing access is also central to realising the ambition for the NHS to be a world leader in genomic medicine and fast tracking the introduction of histology independent therapies.’ 

Karen Lightning-Jones, of Roche Products Limited added: ‘After working closely with regulatory authorities and NICE, we are hopeful that eligible UK patients will be able to access this new medicine as soon as it receives its European licence, which is expected before the end of the year.’