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Novartis and the National Health Service in England have struck a deal on Kymriah (tisagenlecleucel) for use in children and young people with a certain form of leukemia, marking the first time a European country has agreed to fund access to a CAR-T therapy.

Announced today, the agreement comes less than 10 days after Novartis became one of the first two companies to receive EU marketing authorization for a breakthrough CAR-T therapy. It also represents one of the fastest funding approvals in the 70-year history of the NHS.

Kymriah was given the green-light to enter the NHS Cancer Drugs Fund by health technology assessment body NICE, NHS England said in a Sept. 5 statement. Under the deal, the one-time therapy may be used to treat patients up to 25 years old with B cell acute lymphoblastic leukemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse. The Cancer Drugs Fund provides interim funding for promising new treatments while further evidence is collected to help NICE decide whether they should be routinely funded.

Novartis told the Pink Sheet it was “proud to be playing our part to ensure UK patients benefit from this ground-breaking therapy.” It noted that the UK is the first country in Europe, post approval, to grant reimbursement for a CAR-T therapy based on a health technology assessment.

Treatments Imminent

Patients could be set to receive the treatment within weeks, according to NHS England.

While preparing CAR-T therapies involve an immensely complex process, preparations for Kymriah are now in their final stages. The first three NHS hospitals going through the international accreditation process for the provision of CAR-T therapy for children are in London, Manchester and Newcastle.

Novartis told thePink Sheetit was working closely with NHS England “to ensure select specialist hospitals are appropriately trained, supported and ready to deliver this therapy.”

The company could not comment on how much Kymriah would cost under the NHS England deal as this information is commercially sensitive. The full list price for the treatment is around £282,000 per patient.

Only a small number of patients with leukemia in England are expected to be eligible for the treatment every year; reports estimate up to around 30. The company is also seeking to strike a UK funding deal for the other indication for which Kymriah received EU approval – i.e., for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), the most common subtype of non-Hodgkin lymphoma.

“We are working closely and collaboratively with NICE and NHS to make sure that we can get this important new therapy to adult DLBCL patients whose life expectancy is currently very short,” the company said.

In the rest of the EU, Novartis said that, as in the UK, it is targeting the pediatric ALL market first. However, it said that “no additional information can be provided at this time” regarding negotiations with the national health and reimbursement authorities in Europe.

The timing for Kymriah’s availability in the rest of Europe “will depend on multiple factors, including the completion of national reimbursement procedures in complex national pricing reimbursement landscapes, and treatment center on-boarding processes,” Novartis said.

The company added that it was “already working to establish a network of select treatment centers that will offer Kymriah in the EU. Because of the sophisticated and individualized nature of Kymriah and our deep commitment to patient safety, the centers are required to undergo training to adhere to quality standards for cell supply and manufacturing in order to ensure patient safety.”

First In A Wave For England

NHS England chief executive Simon Stevens said Kymriah was the “first in a wave” of treatments in a new era of personalized medicine and part of the NHS’s long term plan to upgrade cancer services.

Commenting on the deal with NHS England, Stevens said: “This constructive fast-track negotiation also shows how responsible and flexible life sciences companies can succeed – in partnership with the NHS – to make revolutionary treatments available to patients.”

Charles Swanton, Cancer Research UK’s chief clinician, said: “We applaud NHS England, NICE and the company for working together to make this immensely complex treatment available to patients quickly, through the Cancer Drugs Fund.”

According to the UK BioIndustry Association CEO Steve Bates: “The ability for NICE and NHS England to say yes for English patients to receive this new therapy within weeks of a drug regulatory approval will be a significant and pleasant surprise to the global industry. “

The decision “shows that the UK is well set to be the best country in the world in which to invent, trial, develop, manufacture and market next generation CAR-T cell cancer therapies,” Bates said. 

“Away from the headlines, industry, government and the NHS has laid the groundwork for adopting this type of innovation for a number of years so it’s fantastic to see UK patients starting to see the benefit of years of research and development. This step should enable the UK to be the first country in Europe where patients benefit from full access to this breakthrough treatment.”

Gilead/Kite’s Yescarta

As for the other company to have received an EU marketing authorization for a CAR-T therapy, Gilead Sciences/Kite Pharma learned last month that NICE had recommended against funding its product, Yescarta (axicabtagene ciloleucel). Draft guidance from NICE said that the cost of the treatment – for adult patients with diffuse large B-cell lymphoma and primary mediastinal B-cell lymphoma after two or more systemic therapies – was too high. (Also see "Gilead And Novartis Unveil EU Marketing Plans For CAR-T Therapies, But Hurdles Remain" - Scrip, 28 Aug, 2018.)

Commenting on its negotiations for routine market access in Europe, Gilead told the Pink Sheet that it had “communicated our openness to considering solutions that improve patient access in Europe and are already engaged in a dialogue with a number of governments and reimbursement bodies across the region.”

“As CAR T therapy is a new, personalized way of treating certain types of cancer, regulatory authorities are reviewing appropriate solutions to ensure access for eligible patients in urgent need of new treatment options,” Gilead added.

Unlike Other Treatments

Unlike other forms of treatment, CAR-T therapies are specifically developed for each individual patient and involves reprogramming the patient’s own immune system cells which are then used to target their cancer. They have been shown in trials to cure some patients, even those with advanced cancers where other treatments have failed.

Kymriah and Yescarta both received approval for sale in the US last year. In the US, Yescarta had sales of $68m in the second quarter of 2018, compared with around $16m in sales for Kymriah. 

This article was updated on Sept. 6, 2018 to clarify that the Cancer Drugs Fund provides interim funding before NICE decides whether a product should be routinely funded.

From the editors of Scrip Regulatory Affairs.

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