NICE recommended two CAR-T therapies to treat aggressive forms of blood cancer, with hundreds of patients set to benefit.
Some people with aggressive forms of blood cancer are set to benefit after two personalised immunotherapy chimeric antigen receptor (CAR-T) treatments were recommended by the National Institute for Health and Care Excellence (NICE) for the Cancer Drugs Fund.
A spokesperson for NICE said that the two treatments could benefit up to "600 people" in total each year in England.
CAR-T therapy is a treatment in which a person's own immune system cells (T-cells) are altered in a laboratory to attach to and kill cancer cells. Both of the newly recommended treatments are given as a one-off infusion.
"We know the devastating impact lymphoma and leukaemia have on people. These innovative new treatment options will help people live longer and improve their quality of life," said Helen Knight, director of medicines evaluation at NICE.
Around 5500 people are diagnosed with diffuse large B-cell lymphoma (DLBCL) each year in England. An aggressive cancer, in some cases it can be treated with a stem cell transplant.
NICE has recommended in final draft guidance axicabtagene ciloleucel (Yescarta, Kite) as a CAR-T therapy for use within the Cancer Drugs Fund as a treatment option for adults with diffuse large B-cell lymphoma, when an autologous stem cell transplant is suitable, if the disease returns within a year of, or is resistant to, first-line chemo-immunotherapy.
"It is currently available as standard care after two or more systemic therapies," explained a NICE spokesperson, "but the new recommendation makes the CAR-T therapy available after just one therapy, providing quicker access for patients."
NICE said that clinical trial evidence suggested the treatment could increase how long people live compared with standard care, extending life by more than 3 months. However, it stressed that the amount of time the drug extends life is uncertain as the trial is still ongoing.
The regulator added that the treatment meets NICE's criteria to be considered a life-extending treatment at the end of life, and that it estimated that just over 500 people a year in England would be eligible to receive the treatment.
It is recommended only if the conditions in the managed access agreement for axicabtagene ciloleucel are followed, NICE emphasised.
The recommendation was not intended to affect treatment with axicabtagene ciloleucel that was started in the NHS before this guidance was published. NICE reassured that those patients having treatment outside the new recommendation may continue without change to the funding arrangements in place for them before the guidance was published, until they and their NHS clinician consider it appropriate to stop.
The list price of axicabtagene ciloleucel for a single infusion, including shipping, engineering, and generation of CAR-T cells is £280,451. The manufacturer had a commercial arrangement that made axicabtagene ciloleucel available to the NHS with a discount.
NICE also estimated that just under 90 people a year in England with relapsed or refractory B-cell acute lymphoblastic leukaemia at age 26 and over would be eligible to receive Brexucabtagene autoleucel (Tecartus, Kite).
Almost 800 people a year are diagnosed with acute lymphoblastic leukaemia each year in the UK.
The treatment was also recommended to go into the Cancer Drugs Fund in final draft guidance for treating the disease, for which standard treatment includes chemotherapy and immunotherapies.
"Brexucabtagene autoleucel would be offered as an additional treatment," a NICE spokesperson highlighted, and added that clinical evidence suggests that people having the treatment may live longer and have more time before their disease relapses.
The final draft NICE guidance recommends the treatment for use within the Cancer Drugs Fund as an option for treating relapsed or refractory B-cell acute lymphoblastic leukaemia in people 26 years and over.
The regulator underlined that only if the conditions in the managed access agreement for brexucabtagene autoleucel are followed would the recommendation be satisfied.
Again NICE sought to reassure those already receiving the treatment, and said that its recommendation was not intended to affect treatment with brexucabtagene autoleucel that was started in the NHS before the guidance was published. "People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop," it reiterated.
Evidence from a study suggested that people having the treatment may live longer and have more time before their disease relapses, but this remained "uncertain", NICE pointed out. It stressed there is also not enough evidence to tell if the treatment can cure B-cell acute lymphoblastic leukaemia.
Brexucabtagene autoleucel cannot be recommended for routine use, explained the regulator, since the most likely cost-effectiveness estimates are uncertain, and some of them are higher than what NICE considered an acceptable use of NHS resources.
The list price for single infusion is £316,118 (excluding VAT). The company had a commercial arrangement that made brexucabtagene autoleucel available to the NHS with a discount.
Further Information to Be Gathered
Three different CAR-T therapies, for six different indications, were now offered on the NHS in England, "benefitting adults and children with a range of cancers", said a NICE spokesperson. The NHS was also the first health system in Europe to agree a full access deal on breakthrough CAR-T therapy, nearly 5 years ago .
NHS National Director for Cancer, Professor Peter Johnson, said: "The NHS continues to take great strides forward in cancer care and it is fantastic that through the Cancer Drugs Fund, we can make cutting-edge CAR-T therapies available to hundreds more patients with advanced blood cancers, giving them real hope of a longer and better quality of life."
Asked to comment for Medscape News UK, Dallas Pounds, director of services at Lymphoma Action, said the charity was "pleased" the two treatments had been recommended for use within the NHS by NICE. "This decision increases the options available to those affected by diffuse large B cell lymphoma," she said.
NICE explained that both treatments were recommended for use in the Cancer Drugs Fund to allow more data to be collected to address uncertainties on how well the treatments worked over a longer period, and whether the results could be applied more generally to NHS practice.
"We are committed to constantly learning from data and implementation, so patients can benefit from ground-breaking treatments while more information is gathered, which will hopefully lead to them being offered routinely on the NHS in the future," Ms Knight said.
Evidence on the two treatments will be collected over the next 4 years, after which NICE will update its guidance.