NICE Explains Its UK Funding Recommendation For Kymriah
Executive Summary
The final appraisal document published today by the health technology assessment body NICE provides more insight into the health technology assessment body’s recent recommendation on
Health technology assessment body NICE today published the reasons why it decided in September that Novartis’s CAR T-cell therapy Kymriah (tisagenlecleucel) should not be routinely used by England’s National Health Service for young people with leukemia but should instead be provided via the Cancer Drugs Fund.
Uncertainties in the evidence for Kymriah, including long-term survival data and the cost of subsequent stem cell treatments, raised questions about the product’s cost effectiveness for routine NHS funding, according to NICE’s final appraisal document on the matter, published Nov. 16.
However, NICE recognized that tisagenlecleucel was a highly innovative treatment and felt that further evidence was likely to resolve these uncertainties, the appraisal document reveals. As such, the HTA body recommended that Kymriah should be made available via the CDF, which provides interim funding for drugs via managed access arrangements while more evidence is collected.
“Collecting more data on overall survival, subsequent stem cell transplant rates, and immunoglobulin usage will reduce the uncertainty in the clinical- and cost-effectiveness evidence,” the document says.
NHS England announced on Sept. 5 that NICE had “green-lighted” Kymriah’s use via the CDF for treating relapsed or refractory B-cell acute lymphoblastic leukemia in people aged up to 25 years. It said at the time that the agreement marked Europe’s first full access deal on a CAR-T therapy.
The appraisal document now provides the public with insight into the reasons for NICE’s recommendation.
It also clarifies that the data collection period for the drug is expected to end in June 2023 when the follow-up of the final patient in Novartis’s ELIANA study will be completed. “At this point the process for exiting the Cancer Drugs Fund will begin and the review of the NICE guidance will start.”
Kymriah is expected to be made available to patients with leukemia in the coming weeks, NICE said in a statement today. NHS England in September had also said that the drug would be made available within weeks. It told the Pink Sheet on Nov. 15 that the process of producing such a treatment was immensely complex and that preparations were now in their final stages. The first NHS hospitals are going through the international accreditation process for the provision of CAR-T therapy for children.
Kymriah, given as a single intravenous infusion, has a list price of £282,000, but Novartis has agreed a confidential discount.
NICE’s Reasons
NICE’s appraisal document, some 33 pages in length, covers a range of topics including Novartis’s positioning of tisagenlecleucel in the treatment pathway for the given indication, clinical evidence, adverse events, the company’s economic model, survival modeling and costs in the economic model, and discount rate.
It notes that “clinical trial evidence suggests that compared with current treatment, people having tisagenlecleucel may live for longer, or have more time before their disease relapses.” But it says that “the evidence is uncertain and it is not known whether tisagenlecleucel can cure acute lymphoblastic leukaemia.”
The document adds that there is also not enough evidence to determine the costs of treating side-effects and whether people will need a subsequent stem cell transplant.
It says that “the most plausible cost-effectiveness estimates for tisagenlecleucel are higher than what NICE normally considers acceptable. In addition, “the life expectancy of people with relapsed or refractory acute lymphoblastic leukaemia is uncertain.” Tisagenlecleucel “does not meet both of NICE’s criteria to be a life-extending treatment at the end of life” and, as such, “cannot be recommended for routine use in the NHS,” the document says.
However, NICE believes that “further data on overall survival would be a valuable addition to the clinical evidence base and would likely resolve uncertainties around length of overall survival and if tisagenlecleucel could be considered a curative treatment.” Therefore, “tisagenlecleucel is recommended for use in the Cancer Drugs Fund,” the document says. “Using tisagenlecleucel in the NHS would allow data to be collected which more accurately reflect the costs and benefits of its use in clinical practice.”
CAR T-Cell Funding In Europe
In August this year Kymriah and Gilead Sciences’ Yescarta (axicabtagene ciloleucel) became the first two CAR T-cell therapies to be approved for sale in the EU. NICE is currently reviewing the therapies for the treatment of diffuse large B cell lymphoma in adults. (Also see "CAR-T Funding: It's (Nearly) All About The Price In The UK" - Pink Sheet, 11 Oct, 2018.) Other countries in Europe are also dealing with the CAR T-cell therapy reimbursement. (Also see "CAR-T Therapies Should Impress EU Reimbursement Bodies" - Pink Sheet, 22 Oct, 2018.)
From the editors of Scrip Regulatory Affairs.