The U.K.’s National Institute for Health and Care Excellence (NICE) has recommended that Kymriah (tisagenlecleucel) be offered to patients with difficult-to-treat diffuse large B-cell lymphoma (DLBCL) through the Cancer Drugs Fund.
Specifically, this Novartis-developed therapy will be available for patients whose disease has not responded (refractory) or whose disease has come back (relapsed) after treatment with two or more chemotherapies, according to NICE’s final draft guidance.
The decision represents a change in NICE’s earlier decision not to recommend Kymriah for patients with DLBCL. The negative appraisal of Kymriah issued in September 2018 was based on its cost and the lack of studies supporting its long-term effectiveness, or comparing its benefit to the current treatment, salvage chemotherapy.
Novartis and NICE had further negotiations about the cost of the treatment before a final committee meeting Oct. 23.
Kymriah is a CAR T-cell therapy approved by both the U.S. Food and Drug Administration and the European Medicines Agency for children and adolescents with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL), and for adults with relapsed or refractory DLBCL. Treatment of children and young adults with ALL is funded by England’s National Health Service (NHS).
Treatment with Kymriah requires collecting a patient’s own immune T cells and genetically modifying them to express a chimeric antigen receptor (CAR), which recognizes a cell surface protein called CD19. This then leads to more efficient identification and destruction of cancer B cells upon reintroduction of the engineered T cells into the patient. Kymriah is given as a single intravenous infusion.
“Recommending another revolutionary CAR T-cell therapy for adults with lymphoma represents a step forward for personalized medicine,” Meindert Boysen, director of the Centre for Health Technology Evaluation at NICE, said in a press release. ”We are pleased that patients are set to benefit from such an innovative therapy so rapidly because of joint working between NICE, NHS England and the company.”
Kymriah’s list price in the U.K. is £282,000 (about $365,000). Novartis has agreed to offer the therapy at an undisclosed discounted price. NHS England is working with hospitals to deliver the therapy.
Although noting that CAR T-cell therapies are expensive, Boysen underlined that they are specifically manufactured for each patient “and could be a potential cure for some, although it is early days.”
“Our recommendation for tisagenlecleucel on the Cancer Drugs Fund (a source of funding for cancer medications in England) means people can benefit while more data is collected,” Boysen said.
Ropinder Gill, chief executive at U.K.-based Lymphoma Action, said: “We’re very pleased that this groundbreaking CAR T-cell therapy will now be available on the NHS for some people with diffuse large B-cell lymphoma.” He added that NICE’s recommendation “offers patients and their families faced with a poor prognosis a more hopeful outlook.”
John Stewart, NHS England’s director of specialized commissioning, mentioned another deal between the NHS and Novartis “which means that … more patients will benefit from this game- changing treatment at even more hospitals throughout the country.”
Stewart added that making more CAR T-cell therapies available and providing competitive treatment deals “are just two of the ways that the NHS Long Term Plan will transform cancer care across the country.”
Alasdair Rankin, director of research and patient experience at Bloodwise, a blood cancer charity, said that CAR T-cell therapy is “the most promising breakthrough in blood cancer treatment of the past decade, with the potential to be used much more widely in the future.” Making CAR T-cell therapies available, he added, “gives these patients the real chance of long-term survival when all other treatments have failed.”