NHS patients with an aggressive form of blood cancer are to benefit from a life-extending daily tablet after the health service became Europe’s first to routinely commission the treatment.
The new targeted treatment, quizartinib, can now be prescribed to newly-diagnosed patients with a specific type of leukaemia, boosting their chance of remission and long-term survival.
The treatment will be routinely commissioned and immediately available thanks to NHS England’s Cancer Drugs Fund, which fast-tracks new innovative cancer treatments into standard care.
Professor Peter Johnson, NHS National Clinical Director for Cancer, said: “It’s very good news that patients with this type of leukaemia now have an option that helps their chemotherapy to work better, boosting their chance of remission and long-term survival and offering them precious extra time with their families and friends.
“People who have acute myeloid leukaemia and the specific FLT-3 ITD abnormality will be able to take this oral treatment, quizartinib, which the NHS has fast-tracked.
“The NHS in England has enviable track record of making innovative treatments available and this is the latest in a long list of new cancer drugs to help people to live with and beyond cancer, making life-changing differences to people affected by cancer and their families across the country”.
Acute myeloid leukaemia (AML) aggressively interferes with blood production, leaving the body open to often fatal infections but, with effective treatment, patients can go into remission and in some cases be cured.
Around 3,100 people are diagnosed with this type of cancer in the UK annually, with just over a quarter (27%) of those having the FLT3-ITD genetic mutation and therefore able to receive the new treatment.
The once-a-day tablet will be offered to adults as part of initial treatment alongside chemotherapy, and then for a further 3 years as a maintenance treatment to reduce the likelihood of cancer relapses.
If a patient’s condition has improved enough because of quizartinib and chemotherapy, eligible patients can then be offered stem cell transplants, which can increase the chance of being cured.
For patients not deemed suitable for stem cell transplants, the drug can still be life-extending, enabling them to have more time with their families and loved ones.
The UK’s medicines regulator, the Medicines and Healthcare products Regulatory Agency, determined the safety and effectiveness of the drug earlier this year, noting that patients receiving quizartinib had a median overall survival of 31.9 months compared to 15.1 months for those receiving the placebo. The median survival rate marks the point at which half of patients had survived in trials.
AML with the FLT3-ITD mutation develops due to uncontrollable cell growth within the bone marrow. Quizartinib is an inhibitor drug which specifically targets and restricts the enzyme responsible for this.
The positive decision follows the manufacturer, Daiichi Sankyo, offering a fair price for the treatment that the National Institute for Health and Care Excellence (NICE) determined to be cost-effective and could therefore recommend its use on the NHS in England.
Helen Knight, Director of Medicines Evaluation at NICE, said: “This is a rare and aggressive form of blood cancer with few treatment options. Patients tell us that existing treatments can be very gruelling and that more options would be welcome.
“Clinical evidence found patients taking quizartinib had an average overall survival of almost 32 months compared to just over 15 months for those receiving the placebo, and our independent committee found it to be a cost-effective treatment.
“Our focus is to the ensure the best care for people with this aggressive form of cancer while offering value for money to the taxpayer.
“I am therefore delighted we can recommend it for use on the NHS as a further treatment option that could give people more precious time with their loved ones”.
