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NICE Recommends Novartis' Kymriah for Pediatric Acute Lymphoblastic Leukemia

NEW YORK – The UK's National Institute for Health and Care Excellence (NICE) on Thursday recommended Novartis' autologous CAR T-cell therapy Kymriah (tisagenlecleucel) for children and young adults with previously treated B-cell acute lymphoblastic leukemia (ALL).

In a final draft guidance, NICE recommended that the National Health Service provide Kymriah to patients aged 25 or younger whose cancers have relapsed following a stem cell transplant.

In 2018, NICE had agreed to make the cell therapy available to this patient population under a managed access agreement through the Cancer Drugs Fund, which allowed more time to collect additional data on Kymriah in children. Although Novartis had not conducted a randomized clinical trial of Kymriah in this setting, the additional time allowed for a matching-adjustment indirect comparison study, through which data from single-arm Kymriah studies were compared to adjusted data from other clinical trials of comparator treatments in this setting.

Through these additional data and real-world evidence, NICE found that the two-year overall survival rate for pediatric patients treated with Kymriah in the NHS was 72 percent. Patients lived for a median of four years after receiving Kymriah versus 7.5 months when they received Amgen's bispecific T-cell engager Blincyto (blinatumomab) or 3 months when they received salvage chemotherapy.

Now, the new finalized guidance recommends routinely rolling out Kymriah and removing it from the Cancer Drugs Fund accordingly.

According to NICE, roughly 40 children and young adults in England will be eligible for Kymriah. The treatment's list price is £282,000 ($352,946.97) per infusion, though Novartis has a commercial agreement with NHS through which it will provide the treatment at an undisclosed discount.

In the US, Kymriah has been approved for pediatric ALL patients since 2017.