Blood cancer patients could be denied life-saving CAR-T therapy

The CAR-T therapy Yescarta is being assessed by the National Institute for Health and Care Excellence (NICE) for the treatment of patients with two types of blood cancer - diffuse large B-cell lymphoma (DLBCL) and primary mediastinal B-cell lymphoma (PMBCL) - who relapse after two or more different types of treatment.

These patients currently have a very poor chance of survival when treated with intensive chemotherapy - current standard treatment in the UK.

In a consultation document published today, the NICE committee recognised that Yescarta was effective and could lead to long-lasting remissions in many patients. But given the high cost, they said that they had not been provided with enough evidence by the pharmaceutical company, Gilead, of how much more effective than chemotherapy it is.   

CAR-T cell therapies work by taking samples of immune cells from the patient and genetically modifying them in a laboratory to recognise, seek out and kill cancer cells, before putting them back into the patient’s blood system. CAR-T therapy is designed to be a one-off treatment and is thought to have the potential to give patients a life-long ‘immunity’ to their cancer.

Clinical trial results presented to NICE showed that 49 per cent of patients treated with Yescarta were alive after six months without any disease progression and 41 per cent of patients were alive with no sign of disease progression after 15 months.

One of the main sticking points for NICE was that a clinical trial has not been carried out to directly compare survival rates between patients treated with Yescarta and patients treated with intensive chemotherapy.

DLBCL is the most common form of blood cancer, diagnosed in around 5,000 people each year in the UK. PMBCL is much rarer, with up to 300 people affected a year.  It is estimated that around 200 patients with these lymphomas could benefit from Yescarta each year if it were to be approved.

The blood cancer research charity Bloodwise is one of the patient organisations that has be consulted in NICE’s appraisal process for Yescarta.

Dr Alasdair Rankin, Bloodwise Director of Research and Patient Experience, 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.

“While CAR-T therapy is still a new technology and evidence of its long-term effectiveness will not be available, we know that there are people alive today who would not be here if they had not taken part in a clinical trial for a CAR-T therapy.”

NICE has asked Gilead to find more UK data on survival rates for chemotherapy in the same group of patients who could benefit from Yescarta, so that the results can be compared. Further evidence and feedback on the consultation document has been invited from both Gilead and patient groups. A final decision is expected within six weeks.

NICE is due to publish its recommendation in the next couple of weeks on another CAR-T therapy, Kymriah, for the treatment of childhood acute lymphoblastic leukaemia and DLBCL.

Bloodwise provides information and support for anyone affected by blood cancer. Our Support Line is open from Monday to Friday between 10am and 4pm.