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

A fight over the price of drugs has delayed a revolution in cancer treatment that could offer hope to thousands of patients.

Last year, health employers promised to quickly follow what they called ‘game-changing’ new gene-targeted cancer drugs.

Simon Stevens, the CEO of NHS England, said in June that the benefits of these new drugs were “potentially huge” – and promised that NHS patients would “be among the first in the world.”

But despite the excitement of last summer, NHS watchdog NICE has now issued draft guidelines rejecting the first of these drugs on the basis of costs.

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

Drug giant Bayer offered a confidential discount, but officials said the price was not lowered far enough to exceed the watchdog’s strict cost-effectiveness thresholds.

Officials said that larotrectinib - which costs £ 15,000 per month - could not be recommended for the NHS at the current price

Officials said that larotrectinib – which costs £ 15,000 per month – could not be recommended for the NHS at the current price

Larotrectinib, which has been available in Europe for more than a year since September and in Europe, is the first of a series of drugs tailored to the genetic composition of a patient’s tumor – rather than where the cancer is located in the body.

The treatments are known as “tumor 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 based on where the tumor originated – such as in the breast, liver, or prostate.

It is a revolutionary approach that offers hope to many people with rare forms of cancer who often no longer have treatment options, because drugs simply have never been tested for their type of cancer.

The new drugs can pave the way for a new era of personalized cancer care – and can ultimately help a large proportion of the 360,000 people who get cancer every year in Britain.

Because they are simply based on someone’s genes – and have been tested for different tumors – the drugs could work for anyone with a specific genetic mutation, no matter how obscure their cancer is.

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 some rarer cancers.

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


Tumor-agnostic drugs are part of a broader approach to treatment called personalized care.

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

A tumor-agnostic drug can treat any type of cancer, regardless of its origin in the body or tissue.

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

But tumor-agnostic drugs treat every type of cancer as long as the cancer has the specific molecular change that the drug targets.

By discovering the molecular characteristics of a tumor, doctors can decide which treatments are best for a person with cancer, regardless of where the cancer is located or what it looks like under the microscope.

These drugs show promising results in clinical trials, where tumors respond in two-thirds to three-quarters of the cancers tested, according to NHS England.

Targeted therapies already exist, such as Pembrolizumab (Keytruda). But these do not have the potential to treat a wide range of cancers, such as tumor-agnostic drugs.

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 provide clinically meaningful responses to cancer patients who are otherwise difficult to treat.

“It is important that eligible patients have access to this treatment option.”

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

NICE admitted that tests suggest that larotrectinib works – but they cannot figure out how well according to their usual methods.

“Evidence from clinical trials with larotrectinib suggests that tumors 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 to other treatments.”

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

Meanwhile, competitor Roche is close to getting a European license for another tumor-naive drug called entrectinib.

NICE has already assessed this and hints at good news – with officials saying it could be “the first” tumor-naive drug approved for NHS patients.

Meindert Boysen, director of the Center for Health Technology Evaluation at NICE, said: “Histology-independent drugs are an exciting new development in cancer care.

“These advanced therapies target rare genetic mutations, so the clinical evidence is usually based on extremely small sample sizes, which require new approaches to test them in clinical trials.

“We hope that further data collection, combined with responsible prices from the companies, will lead to progressive new treatments as available to patients.”

John Stewart, National Director of Specialized Commissioning at the NHS, said: “It is disappointing that Bayer has not yet been willing to praise larotrectinib at a level that represents value for the NHS and the taxpayer, if they reconsider the NHS.” door remains open for further discussion.

“In the meantime, preparations are underway for the introduction of this next generation of therapies, including constructive discussions with Roche about a commercial deal for entrectinib.”

A Bayer spokesperson said the company was “disappointed” by the decision and added: “It is vital that all parties continue to work closely together and show flexibility so that NHS patients with NTRK merger genes can benefit from access to a new treatment option, as soon as possible.

“Securing access is also crucial for realizing the NHS’s ambition to be a world leader in genomic medicine and to quickly follow the introduction of histologically independent therapies.”

Karen Lightning-Jones, of Roche Products Limited added: “After working closely with regulatory authorities and NICE, we hope that eligible UK patients will have access to this new drug as soon as it receives its European authorization, which is expected before the end of the year. “