Cancer charities have reacted with dismay after a “new generation” therapy that has proven to save lives has been rejected for widespread NHS use by health officials on cost grounds.

The National Institute for Health and Care Excellence (Nice) published draft guidance stating that axicabtagene ciloleucel, also known as Yescarta, is not recommended for NHS use for the 4,800 patients in the UK with aggressive sub-types of non-Hodgkin lymphoma.

A Nice committee said the cost of the drug was “above £50,000 per year of quality adjusted life (QALY) gained”, the upper limit of the specially extended range of cost effectives for cancer treatments meaning it is “too high for it to be considered a cost-effective use of NHS resources”.

The treatment, created by Kite Pharma – a subsidiary of Gilead Sciences – is a personalised cell therapy which re-engineers a patient’s own immune cells to fight cancer. It is the first of a new line of chimeric antigen receptor T-Cell (CAR-T) therapies which are specifically manufactured for each individual patient.

What the drug does

Axicabtagene ciloleucel is made using a patient’s T cells – a type of immune system cell. A gene for a special receptor called chimeric antigen receptor (CAR) is added to the T cells in the laboratory. These changed T cells called CAR T cells are grown in large numbers in the laboratory and given to the patient by infusion. Axicabtagene ciloleucel binds to a protein called CD19, which is found on most B cell lymphoma cells. This helps the body’s immune system kill cancer cells.

The treatment involves taking some of the patient’s own white blood cells which are then re-engineered in a laboratory so they can recognise and attack cancer cells before being infused back into the patient. It is for patients with aggressive forms of non-Hodgkin lymphoma, including relapsed or refractory diffuse large B-Cell lymphoma and primary mediastinal large B-cell lymphoma, after they have already had two or more different types of treatment.

The Institute of Cancer Research, London (ICR), and the blood cancer research charity Bloodwise said the news was “disappointing” for patients. The ICR said the treatment was a “major advance in cancer treatment” which has cured some patients who would otherwise have died.

Bloodwise said that these patients currently have a very poor chance of survival when treated with intensive chemotherapy – the current standard treatment in the UK. Kite Pharma said that in clinical trials, 72 per cent of the patients responded to therapy and 51 per cent went into complete remission.

But Nice said that there is no direct data to compare it with the current standard treatment of salvage chemotherapy, which means the exact size of the benefit the therapy would offer patients is unknown. The regulator said that cost of axicabtagene ciloleucel was also too high for it to be considered a cost-effective use of NHS resources. The health body also considered whether the treatment should be made available to patients through the Cancer Drugs Fund, but concluded that the therapy would not be cost-effective under the scheme.

Meindert Boysen, director of the centre for health technology evaluation at Nice, said: “CAR-T is an exciting innovation in very difficult-to-treat cancers, with a promise of cure for some patients.

“We have been working with the companies involved, and with NHS England, with the aim of ensuring that patients in England are among the first to have access to these new treatments in Europe. Although promising, there is still much more we need to know about CAR-T, and unfortunately, in this case, we are not able to recommend axicabtagene ciloleucel for use in the NHS in England at the cost per patient set by Kite Pharma.”

Agreement

Hilary Hutton-Squire, general manager at Gilead Sciences UK and Ireland, said: “Yescarta is a new generation of innovative cell therapy that is bringing a new option to patients who, in many cases, have run out of treatment options and literally have months to live. Our priority is to make axicabtagene ciloleucel available to patients in the UK as soon as possible, and as such we believe we will soon be able to reach an agreement.”

Professor Raj Chopra, head of cancer therapeutics at the ICR, said: “The technique is complex and expensive, but it is also a major advance in cancer treatment that has cured some patients who would otherwise have died.”

Dr Alasdair Rankin, director of research and patient experience at Bloodwise, said: “It’s extremely disappointing that people with advanced and highly aggressive blood cancers might not be able to access this breakthrough treatment that offers the genuine chance of a cure.

“CAR-T therapies are an entirely new type of treatment and are expensive, so it understandable that there will be a number of uncertainties and questions that need to be answered. We hope that over the coming weeks the NHS and the pharmaceutical company can work to address these issues and agree on a price so that patients can benefit.”

The Yescarta decision is the latest in a series of cancer drugs rows involving health officials. Earlier this month, an “arbitrary” restriction the NHS had set denying cancer patients a life-extending drug was lifted by the health service giving a potential lifeline to hundreds of leukaemia patients.  NHS England had been accused of defying a legal duty to make approved drugs available to hundreds of patients in an attempt to cut costs. Experts feared it could have set an unacceptable precedent.

The row centred on ibrutinib, a medicine to treat chronic lymphocytic leukaemia (CLL), a blood cancer that affects 3,700 people each year and kills 1,000. Despite the drug being declared a highly effective, value-for-money medication by Nice, NHS England curtailed its use saying it was going to pay for ibrutinib only to treat patients who have relapsed within three years. It reversed its decision after receiving further evidence on its use.