NICE U-turn opens up new treatment option for some adults with leukaemia

In collaboration with the Press Association

Patients living with a specific type of leukaemia will have access to a new immunotherapy treatment on the NHS in England.

Blinatumomab (Blincyto) – a drug used to treat adults with acute lymphoblastic leukaemia  – has been recommended by the National Institute for Health and Care Excellence (NICE) for some patients, after being initially rejected for use in a larger group of patients in March 2019 due to a lack of evidence. 

It will be available for patients in England who may still have a small number of cancer cells in their body after treatment, but not enough to be picked up in standard tests.

Patients in Wales and Northern Ireland should also now be able to access the drugs.

Currently these people are treated with continued chemotherapy followed by a stem cell transplant, if possible, or a stem cell transplant without chemotherapy.

Rose Gray, Cancer Research UK’s policy manager, said the announcement was “very welcome news”.

“It’s great that NICE, NHS England and the manufacturer have been able to work together to overcome the challenges that meant the treatment was initially not recommended back in March.”

Why the U-turn?

NICE based their decisions on the evidence from two clinical trials.

In its provisional decision, NICE said the patients who took part in the trials did not accurately reflect those who would be receiving the drug on the NHS, so it couldn’t verify the benefits of blinatumomab.

The NICE committee said the cost effectiveness of the drug could not be measured.

Since this original decision, the manufacturer submitted new information on the studies’ overall survival results and safety. It also updated its estimates of how much it would cost to treat patients with blinatumomab rather than other treatments. This reduced uncertainty over the treatment’s benefits and value for money.

Harnessing the power of the immune system

Blinatumomab has been recommended for treating a specific type of leukaemia called Philadelphia negative leukaemia. It works by bringing leukaemia cells into close contact with immune cells, allowing the immune system to more effectively kill them.

“Clinicians and patients told NICE current treatments can have serious side effects,” said Gray. “So this is a positive step because this treatment could offer a better quality of life, as well as reducing the chance their cancer comes back.” 

In one trial involving 405 patients, blinatumomab significantly increased survival compared to standard chemotherapy. Patients using blinatumomab lived for 7.7 months on average after treatment, compared to four months for patients given chemotherapy. 

Severe side effects were reported in 9 in 10 patients in both groups, with the most common being low white blood cell count and infection.

The other, smaller trial only included 20 people taking blinatumomab. After following patients for 6 years, around half the patients were still alive and their disease had not come back.

References

H, Kantarjian (2017) Blinatumomab versus Chemotherapy for Advanced Acute Lymphoblastic Leukemia. NEJM. DOI: 10.1056/NEJMoa1609783