Young people with relapsed or refractory B-cell acute lymphoblastic leukaemia (ALL) will now have access to tisagenlecleucel, also known as Kymriah made by Novartis, through the Cancer Drugs Fund (CDF).
Children and young people at this stage of their disease have usually undergone repeated cycles of treatment, which can have significant psychological and physical effects and s poor outcomes.
Tisagenlecleucel will be offered to people under the age of 25 who have not responded to current treatment or who have relapsed after stem cell transplant.
The therapy, which involves taking a person’s own immune cells and modifying them to fight their cancer cells, has the potential to be a cure.
Around 25 to 30 people will be eligible for tisagenlecleucel each year in England and a specialised NHS service is being developed to manage access to the new therapy.
NHS England are preparing to make tisagenlecleucel available in the coming weeks.
Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE said: “NICE’s recommendation of tisagenlecleucel marks a new generation of personalised medicine that has the potential to transform the care of patients with cancer worldwide.
“CART-cell therapy is expensive and complex. We have worked in partnership with our stakeholders, NHS England and the company to make the therapy available to patients quickly. Novartis have agreed to offer tisagenlecleucel at a lower price so that people using the NHS can be among the first in the world to access this exciting new treatment.”
Dr Alasdair Rankin, Director of Research at the blood cancer charity Bloodwise, said: “CART-cell therapy can give children with leukaemia the real possibility of long-term survival if they do not respond to standard treatments. Today’s announcement will come as a huge relief for a number of worried families. We hope that people will be able to access the therapy as soon as possible.”
Cancer Drugs Fund
NICE has recommended tisagenlecleucel for the CDF due to some uncertainties in the evidence.
NICE recognises tisagenlecleucel as a highly innovative treatment and believes further evidence is likely to resolve these uncertainties. The CDF approval will allow young people to have access to the therapy as more data is gathered.
The therapy, given as a single intravenous infusion, has a list price of £282,000, but the company have agreed a confidential discount.
John Stewart, NHS England’s director of specialised commissioning, said: “NHS cancer patients will now be amongst the first in the world to benefit from this game changing therapy.”