NHS prepares to fast-track ‘game changing’ cancer drugs that target genetic mutations

The NHS is preparing to fast-track the introduction of ‘game changing’ new cancer drugs that target tumours according to their genetic make-up rather than where they originate in the body, Simon Stevens will announce today.

The revolutionary treatments – known as ‘tumour agnostic’ drugs – can be used against a wide range of cancers and could offer hope to patients with rare forms of the disease that may previously have been untreatable.

Extensive preparation by the NHS meant that patients in England were among the first in the world to benefit from genetically modified CAR-T cancer therapy.

Speaking to health leaders in Manchester, NHS England chief executive Simon Stevens will say that the health service must be ready to fast-track tumour agnostic drugs in the same way.

Detecting cancer early when it is easier to beat and providing the latest treatments is a central part of the NHS Long Term Plan.

The first of the new cancer drugs, which target genetic mutations that accelerate the growth of many types of tumours and have particular benefits for children, are set to come on to the market within months.

With such treatments, testing the tumour’s genes or other molecular features assists in deciding which treatments may be best for an individual with cancer, regardless of where the cancer is located or how it looks under the microscope.

Molecular testing therefore becomes a first and essential element of treatment planning.

Around 850 patients a year could benefit from the frontrunners while many thousands a year are eventually expected to benefit from other tumour agnostic treatments on the horizon.

Patients in England are ideally placed to benefit thanks to the NHS’s world-leading national genomic medicine and testing service which means they can be identified and tested, allowing faster access to targeted treatment, often when no other options are available.

NHS England will now work with the industry to step up preparations and –

  • Bring together different cancer specialists to ensure all patients who could benefit from tumour agnostic drugs are identified
  • Embed the tests for these genomic mutations within existing cancer pathways
  • Through NICE ensure that the unique characteristics of these treatments will be valued appropriately and efficiently.

Speaking at the Confed19 event Simon Stevens is expected to say: “This exciting new breakthrough in cancer treatment is the latest example of how the NHS can lead the way in the new era of personalised cancer care.

“The benefits for patients, in particular children, of being able to treat many different types of cancers with one drug is potentially huge, helping them to lead longer, healthier lives.

“NHS patients in England were at the forefront of the CAR-T revolution and I want to make sure that they are also among the first in the world to benefit from these treatments.

“Preparations are underway to make sure the NHS can adopt these next generation of treatments, but manufacturers need to set fair and affordable prices so treatments can be made available to those who need them.”

The Accelerated Access Collaborative – established by the Government as part of the Life Sciences Industrial Strategy to speed up the adoption and uptake of innovative new treatments – will meet next week to discuss how they can support implementation.

Lord Darzi, Chair of the Accelerated Access Collaborative, said: “Today’s announcement is further evidence that the NHS is open for business and is both willing and able to embrace innovative new treatments at pace. The advent of tumour agnostic drugs marks an important next step in the development of personalised, genomic driven medicine and these plans will be warmly welcomed by patients and the clinical community.”

Today’s announcement follows NHS England’s landmark commercial deal in September last year to bring the pioneering CAR-T therapy to cancer patients in the UK.

Existing cancer drugs need to be approved by NICE for each individual type of cancer they treat such as breast or colon cancer. However, when approved, these cutting edge new tumour agnostic drugs would be available to treat all types of tumour without individual approval.

Two of the first candidate drugs are expected to be licenced later this year, and if priced responsibly, could be approved by NICE soon after.

The drugs target tumours with a genetic variation which accelerates growth and children are expected to be among the first to benefit.

Both drugs target tumours with the genetic variation known as neurotropic tyrosine receptor kinase, or NTRK, which accelerates growth.

NTRK is more commonly found in rare cancers affecting children and adults (e.g. salivary tumours, secretory breast cancer, infantile fibrosarcoma, congenital mesoblastic nephroma) but also in low levels in more common cancers. Both drugs are due to receive their EU market authorisations within the next 12 months.

The drugs work by blocking the NTRK enzyme, effectively shrinking the tumour. Early clinical trials showed the tumour responded in two thirds to three quarters of the cancers tested.

For some cancers, NTRK testing will be performed using testing as identified in the National Genomic Test Directory.

The introduction of a new NHS Genomic Medicine Service is expected to provide the vital infrastructure for ensuring access to cutting-edge genomic technologies to enable patients who will benefit most from these targeted and personalised medicines to be identified, and treated, quicker.

Dr Alasdair Rankin, Director of Research, Policy and Support at Bloodwise said: “Tumour agnostic treatments hold real potential to deliver kinder targeted treatments to people with rare blood cancers, including children, where evidence of the effectiveness of drugs can be built up more quickly in trials of people with different types of cancer.

“We welcome this further example of NHS innovation and look  forward to working with NHS England to ensure that these and other advances, as well as whole genome sequencing, translate into better diagnosis and better outcomes for everyone with blood cancer in the future.”

Ian Walker, Cancer Research UK’s director of clinical research, said: “These new cancer drugs absolutely represent the kind of exciting innovation the NHS should be looking to fast-track. And it’s great that the NHS recognises the huge potential of these emerging approaches.

“The concept of focusing on specific mutations that drive a person’s disease, versus the cancer type, is a fascinating one.

“Whilst this approach may not work in all cases, where the weight of clinical evidence supports the approach, this could open up opportunities for cancer treatment that we wouldn’t have had 20 years ago.

“It’s essential that patients are offered routine genetic analysis of their tumours, no matter where they live, to match them to appropriate treatments, such as these drugs.

“If approved by NICE, tumour agnostic drugs could give hope to children and young people with cancer, patients with rare cancer types, and their families.”

Jane Lyons, Cancer 52 said: “This is an exciting development for people with rare and less common cancers. We believe the NHS Long Term Plan will improve the treatment and care for people with rare and less common cancers, specifically the early diagnosis target of three in four of all cancers diagnosed early by 2028.

“As with all initiatives and specially those which are working with smaller numbers and rarer conditions collaboration across industry, the NHS and NICE is critical and we welcome the commitment to just that approach.”