NHS England announced Wednesday that certain children and young adults with leukaemia will be able to receive Novartis' CAR-T cell therapy Kymriah (tisagenlecleucel), "the first in what is expected to be a rapidly expanding class of personalised cancer therapies available on the NHS." Kymriah, which costs around 282 000 pounds ($363 000) per patient at its full list price, was approved by the European Commission late last month to treat patients up to 25 years old with B cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse.
According to NHS England, the decision to make Kymriah available follows a commercial deal it negotiated with Novartis, although both parties declined to disclose the price. Simon Stevens, chief executive of NHS England, described CAR-T therapy as a "true game changer, and NHS cancer patients are now going to be amongst the first in the world to benefit." Meanwhile, Mari Scheiffele, Novartis Oncology general manager for the UK and Ireland, noted that the decision to make Kymriah "available so soon after being licensed is the result of our close collaboration with NHS England and [the National Institute for Health and Care Excellence], with flexibility shown by all parties."
The first three NHS hospitals to go through the international accreditation process for the provision of CAR-T therapy for children are in London, Manchester and Newcastle, the NHS said, adding that if requirements are met, the first treatments could start "in a matter of weeks." Approximately 30 children a year are expected to be administered the CAR-T therapy, which will be funded via the Cancer Drugs Fund.
The European approval of Kymriah, formerly known as CTL019, also included treatment of adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy, but NICE has yet to decide whether the NHS can afford it. "We trust that the NHS will continue this collaboration and flexibility in granting [Kymriah] access to adult patients with DLBCL," Scheiffele remarked.
Kymriah's price was set at $475 000 for a one-time single administration in the US, where it was approved last year to treat ALL in certain children and young adults, marking the first authorisation of a gene therapy in the country. The product was later authorised by the FDA to treat adults with DLBCL, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma, after two or more lines of systemic therapy.
In a statement to FirstWord, Novartis indicated that based on its experience in the US, it plans "a gradual approach" to its European launch, with an initial focus on the ALL indication as it ramps up capacity. The company added that it "will launch the DLBCL indication progressively." With regards pricing in Europe, Novartis said that it will "vary from country-to-country, based on local regulations, as well as reimbursement negotiations," noting that "a one-size-fits-all approach is not possible."
The latest news follows the EU's approval last week of Gilead Sciences' CAR-T cell therapy Yescarta (axicabtagene ciloleucel) for adults with relapsed or refractory DLBCL and primary mediastinal large B-cell lymphoma, after two or more lines of systemic therapy. However, NICE has already issued draft guidance recommending against routine NHS funding of Yescarta in its two approved indications, noting that while the therapy has demonstrated good response rates, overall survival and progression-free survival, there are no direct data comparing it to salvage chemotherapy. The ptoduct costs $373 000 in the US, where it was cleared last October for certain patients with large B-cell lymphoma.
For related analysis, see ViewPoints: NICE sets up first EU reimbursement hurdles for CAR-Ts.
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