NICE issues final draft guidelines for Novartis’ Kymriah

NICE has recommended a pioneering cancer treatment for people under the age of 25 with leukaemia in final draft guidelines.

The decision paves the way for young people with relapsed or refractory B-cell acute lymphoblastic leukaemia (ALL) to access Novartis’ Kymriah (tisagenlecleucel) through the Cancer Drugs Fund (CDF).

NHS England is now preparing to make the CART cell therapy, which involves taking a person’s own immune cells and modifying them to fight their cancer cells, available to patients under 25 years who have not responded to current treatment or who have relapsed after stem cell transplant.

Around 25 to 30 people will be eligible for treatment with Kymriah each year in England, and a specialised NHS service is being developed to manage access to the new therapy.

NHS England reached a commercial agreement for the treatment with Novartis back in September, facilitating NICE’s CDF recommendation.

"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," noted Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE.

"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."

"CART-cell therapy can give children with leukaemia the real possibility of long-term survival if they do not respond to standard treatments," added Dr Alasdair Rankin, director of Research at the blood cancer charity Bloodwise. "We hope that people will be able to access the therapy as soon as possible."

"This result is only possible due to our close collaboration with NHS England and NICE," said Novartis. "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."

Kymriah is also approved in Europe to treat adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), but initial guidance from NICE has not back the therapy in this setting, concluding that it is not cost effective for routine funding or use within the Cancer Drugs Fund (CDF).

A key sticking point is the fact that there is no data comparing Kymriah to salvage chemotherapy, the most common treatment currently used, making it difficult to determine the exact benefits of the therapy, NICE said.

Novartis stressed that it is continuing 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. "

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