An independent body that offers guidance to improve health and social care in England recommended against the use of axicabtagene ciloleucel, one of two chimeric antigen receptor T-cell therapies already approved in the United States and European Union.
The National Institute for Health and Care Excellence (NICE) issued draft guidance today that said axicabtagene ciloleucel (Yescarta; Kite Pharma, Gilead) — which received FDA approval last year for treatment of certain B-cell lymphomas — is too expensive to justify its use in England’s National Health Service (NHS).
The list price for axicabtagene ciloleucel in the United States is $373,000. However, the price in the United Kingdom remains confidential.
“[CAR T-cell therapy] is an exciting innovation in very difficult-to-treat cancers, with a promise of cure for some patients,” Meindert Boysen, director of the center for health technology evaluation at NICE, said in a press release.
“We have been working with the companies involved, and with NHS England, with the aim of ensuring that patients in England are among the first to have access to these new treatments in Europe,” Boysen added. “Although promising, there is still much more we need to know about [CAR T-cell therapy]. ... Unfortunately, in this case, we are not able to recommend axicabtagene ciloleucel for use in the NHS in England at the cost per patient set by Kite Pharma.”
NICE did extended a call for “comments and further evidence” that could lead to axicabtagene ciloleucel being made available through NHS. This feedback will be accepted until Sept. 18.
Officials with Gilead Sciences issued the following statement to HemOnc Today and Healio.com: "[Axicabtagene ciloleucel] is a new generation of innovative cell therapy that is bringing a new option to patients who, in many cases, have run out of treatment options and literally have months to live. Our priority is to make axicabtagene ciloleucel available to patients in the UK as soon as possible and, as such, we believe we will soon be able to reach an agreement. Cell therapy is a new and advanced technology and, as NICE has identified, there is little available data about the salvage treatment of patients with aggressive forms of non-Hodgkin lymphoma in England. We’re therefore in ongoing discussions with NICE to identify appropriate treatment comparators which can clarify how cell therapy may be made available to patients in the UK."
The FDA approved axicabtagene ciloleucel — a CD19-directed genetically modified autologous T-cell immunotherapy — in October for treatment of adults with relapsed or refractory large B-cell lymphoma who received two or more lines of systemic therapy.
The approval applies to patients with diffuse large B-cell lymphoma not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma and DLBCL arising from follicular lymphoma.
The FDA based its decision in part on data from the ZUMA-1 trial, which included 101 adults with relapsed or refractory large B-cell lymphoma.
Overall, 72% of patients treated with a single infusion responded to therapy, including 51% (95% CI, 41-62) of patients who achieved complete response. At median follow-up of 7.9 months, median duration of response had not been reached (95% CI, 8.1-not estimable) among patients who achieved complete remission.
NICE members noted there are no data to directly compare the regimen with salvage chemotherapy, the standard treatment for patients in the United Kingdom whose disease recurred or did not improve after at least two courses of chemotherapy.
The panel determined cost-effectiveness estimates for axicabtagene ciloleucel compared with salvage chemotherapy exceeded 50,000 English pounds per quality-adjusted life year gained. That is “the upper limit of the specially extended range of cost-effectiveness for cancer treatments,” according to NICE’s draft guidance.
Axicabtagene ciloleucel was the second CAR T-cell therapy to receive FDA approval.
The other — tisagenlecleucel T-suspension (Kymriah, Novartis) — is indicated for the treatment of children and young adults with B-cell acute lymphoblastic leukemia. The approval applies to patients aged up to 25 years with refractory B-cell precursor ALL, as well as those whose disease is in second or later relapse.
Tisagenlecleucel T-suspension is priced at $475,000.
Both axicabtagene ciloleucel and tisagenlecleucel T-suspension received approval from European Union regulators on Monday.