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CAR T-Cell Therapy in Europe: Differing Decisions on Approval of Two Agents

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The European Commission (EC) recently approved the chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel (Kymriah) in the European Union (EU) for the treatment of pediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse posttransplant, or in second or later relapse, as well as for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy. 

Tisagenlecleucel is a one-time treatment that uses a patient's own T cells to fight cancer. It is the only CAR T-cell therapy to receive regulatory approval in the EU for these two distinct B-cell malignancies. In the EU, the treatment is expected to launch initially in the pediatric acute lymphoblastic leukemia indication. Tisangenlecleucel was initially approved by the U.S. Food and Drug Administration (FDA) for certain pediatric and young adult patients with ALL in August 2017.

UK Announcement

Just days after the EC decision on tisagenlecleucel, The National Institute for Health and Care Excellence (NICE) of the United Kingdom announced a draft decision not to recommend CAR T-cell therapy with axicabtagene ciloleucel (Yescarta) for UK patients with two types of aggressive non-Hodgkin lymphoma for whom other treatments had failed. Although NICE agreed that patients had good response rates and there was an unmet need, it said it hadn’t been given enough evidence of benefit compared with other types of treatment—and the initial cost presented to them was much too high. As this is a draft decision, the manufacturer will get another chance at a later date to present data to NICE on clinical and cost effectiveness.

Commenting on the NICE decision, Raj Chopra, MD, PhD, Head of Cancer Therapeutics at The Institute of Cancer Research, London, said:

“It’s disappointing that patients with non-Hodgkin lymphoma who have exhausted all other treatment options will not be able to access CAR T-cell therapy, a brand-new type of treatment for blood cancers. CAR T-cell therapy uses a patient’s own genetically modified immune cells to target their cancer. The technique is complex and expensive, but it is also a major advance in cancer treatment that has cured some patients who would otherwise have died. If we’re going to see CAR T-[cell] therapy widely available on the National Health Service [NHS], we need to find ways to reduce the costs. I hope NICE and the manufacturer will be able to work together both to bring the costs of treatment down and continue to build a stronger evidence base for it, so it can be made available to NHS patients as quickly as possible.”

Axicabtagene ciloleucel was approved by the FDA in October 2017, for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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