A potentially life-saving treatment for children and young adults with a rare type of blood cancer will be rolled out widely on the NHS in England after receiving the green light from the health service’s spending watchdog.
A charity described the decision by the National Institute for Health and Care Excellence (Nice) as a “ray of hope” for patients with few other options.
Tisagenlecleucel, sold under the brand name Kymriah, is a Car-T therapy, a personalised treatment which reprogrammes a patient’s immune system cells to target the cancer.
The drug has been available on the NHS for patients with B-cell acute lymphoblastic leukaemia (ALL) via the Cancer Drugs Fund (CDF) since 2018.
Nice has now recommended its routine rollout in final draft guidance.
Tisagenlecleucel will be available to ALL patients aged 25 and under whose disease has either not responded to treatment, relapsed after a stem cell transplant, or relapsed for a second time or more.
Helen Knight, director of medicines evaluation at Nice, said: “I am delighted that we have been able to recommend continued use of this innovative treatment, which is already making a huge difference to the lives of children and young adults.”
Nice estimates about 40 children and young people in England will be eligible for the treatment.
According to Cancer Research UK, about 790 people are diagnosed with ALL in the UK each year.
Since 2018, data has been collected on the use of tisagenlecleucel on NHS patients.
Data showed people lived longer without the disease progressing or coming back.
The 24-month overall survival was 72% following treatment, according to Nice.
Clinical trials found the median overall survival was 48 months, compared to 7.5 months for the immunotherapy blinatumomab, and three months for salvage chemotherapy, which are two other standard treatments.
Professor Peter Clark, lead of NHS England’s CDF, added: “It’s great news that this potentially curative drug is now being rolled out for routine use in the NHS for treating children and young people with an aggressive type of leukaemia.
“Tisagenlecleucel is just the latest drug to be recommended for routine use on the NHS and follows its use in certain circumstances for patients as part of our Cancer Drugs Fund since 2018, where it has shown its effectiveness in helping more children and young people live longer, healthier lives.”
Charlotte Crowley, policy and evidence manager at charity Leukaemia Care, said: “These patients have few other options for treatment and Car-T therapies have revolutionised the treatment of these patients already.
“We are really pleased that Nice has been able to approve this treatment for the longer term, giving patients and doctors much greater clarity on what is available if they do find themselves in this awful position.
“The severity of ALL which has not responded to treatment or that has relapsed after treatment was recognised when Nice gave the treatment its highest possible severity modifier.
“This means the NHS is willing to pay than they usually would for each year of health people gain when they access this treatment.”
Leukaemia Care’s advocacy officer Sophie Wheldon was diagnosed with ALL when she was 20 and started Car-T therapy the following year.
Five years on, she said the treatment saved her life.
“I’m very pleased that other ALL patients who receive the devastating news that they have relapsed will have access to this treatment,” Ms Wheldon added.
“It saved my life and allowed me to start supporting other people who are going through such a life-changing experience.
“I had already tried a stem cell transplant and various chemotherapies, which were sadly unsuccessful for me. Without receiving Car-T therapy, I simply would not be here today.
“This treatment represents a ray of hope for others in the same position that I was and I couldn’t be happier with the decision.”
Dr Sam Godfrey, science engagement lead at Cancer Research UK, said: “No child or young person with cancer should ever have to face the reality that there are no treatment options left for them, so there is an urgent need for new treatments to be developed and made available to young cancer patients.
“The news that tisagenlecleucel will now be available routinely on the NHS is very welcome indeed and an important step forward.
“The fact that this effective treatment doesn’t require repeat visits to the hospital for multiple transfusions and causes less severe side effects than the current standard treatment, means it offers more precious time to spend with loved ones and allows children and young people to live longer, healthier lives.”
Yasmin Sheikh, head of policy and public affairs at stem cell charity Anthony Nolan, added said: “We’re thrilled children and young people can continue to receive this potentially curative Car-T therapy, which is associated with fewer side effects than alternative treatments.
“We urge the industry and NHS England to continue to work together to make innovative cell and gene therapies like Kymriah available to patients so they can benefit as soon as possible.”
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