NICE has recommended a pioneering blood cancer therapy tisagenlecleucel, this is a CART-cell therapy for young people with blood cancer.
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).
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.
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. Around 25 to 30 people will be eligible for the blood cancer therapy tisagenlecleucel each year in England and a specialised NHS service is being developed to manage access to the new therapy.
Young people with relapsed or refractory B-cell ALL have repeated cycles of treatment, which can have substantial psychological and physical effects and some have poor outcomes. The current treatment for this stage of the disease is blinatumomab or chemotherapy.
NHS England is preparing to make tisagenlecleucel available in the coming weeks.
In September 2018, NHS England reached a commercial agreement with Kymriah’s manufacturer Novartis, paving the way for the NICE CDF recommendation.
Cancer Drugs Fund
Uncertainties in the evidence, including long-term survival and the cost of subsequent stem cell treatments, raised questions about the cost-effectiveness for routine NHS funding at this time.
However, NICE recognises blood cancer therapy 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 has agreed a confidential discount.
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.”
Mari Scheiffele, Novartis Oncology General Manager, UK & Ireland, said: “Today represents a milestone for children and young adults with acute lymphoblastic leukaemia (ALL) who have not responded to other treatments and whose prognosis is poor. This one time therapy, specifically custom-made for each individual patient using their own immune cells, has the potential to extend their survival and improve their quality of life.
“This result is only possible due to our close collaboration with NHS England and NICE. In parallel with these discussions, we have been working alongside the selected specialist hospitals to ensure they are now trained and ready to deliver this life-saving therapy to eligible patients.
“We are proud to be playing our part to ensure UK patients benefit from this groundbreaking therapy. We continue to work closely with NICE and the NHS to provide tisagenlecleucel access to adult patients with diffuse large B cell lymphoma (DLBCL) – the most common non-Hodgkin’s lymphoma, where there is also a high unmet need for patients who have relapsed and have no other treatment options. In addition, Scottish Medicines Consortium (SMC) is currently considering our submission to the NHS in Scotland, for both indications, so that every appropriate patient in the UK has access to Kymriah.”
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.”
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. Constructive engagement with NICE and fast-track negotiations with NHS England shows how responsible and flexible life science companies can succeed – in partnership with the NHS – to make revolutionary treatments available to patients”