NEW YORK – In a reversal from its previous negative recommendation issued in January, the UK's National Institute for Health and Care Excellence (NICE) today said that it would recommend larotrectinib (Bayer's Vitrakvi) for inclusion on the Cancer Drugs Fund.
The positive recommendation will make larotrectinib available as a treatment of NTRK fusion-positive solid tumors in adults or children with locally advanced, metastatic, or non-surgically resectable cancer, who lack appropriate treatment alternatives.
NICE's original recommendation against including larotrectinib on the Cancer Drug Fund was based on the drug's high price tag. "Larotrectinib does not have the potential to be a cost-effective use of NHS resources at its current price," read the January decision. Another factor NICE noted in that preliminary decision was the lack of clinical evidence demonstrating larotrectinib's efficacy in comparison with other treatments.
Now that Bayer has submitted a new price for the drug, however, NICE has reversed its recommendation and said that patients can access larotrectinib through the Cancer Drugs Fund while further data is collected to address the gaps in evidence. NICE did not disclose the reduced price, but said that it "makes larotrectinib available to the NHS with a discount." Previously, the drug was priced at an estimated monthly cost of £15,000 ($19,610) before applying the UK's value-added tax.
Because NTRK gene fusions are so rare, occurring in fewer than 1 percent of solid tumors, clinical evidence for larotrectinib to date has been based on extremely small patient cohorts. Meindert Boysen, NICE's CEO and director of the Centre for Health Technology Evaluation, said in a statement that novel approaches to evaluating the drug's potential value in NHS practice would be required.
In coming to the new decision, the appraisal committee grappled with several issues that the histology-independent drug raises, including the fact that the diagnostic pathway for gauging NTRK fusions is uncertain until NHS establishes a national service for genomic testing of all advanced solid tumors. According to NICE's report, the NHS is currently working to standardize genomic testing within this national service and replace all local testing.
Until the national laboratory hubs are established, however, next-generation sequencing will be done only after a patient has tried all available NHS commissioned treatment options. In the future, once the national service for genomic testing is up and running, the statement said, "next-generation sequencing to identify gene alterations, including NTRK gene fusions, will be done when locally advanced or metastatic solid tumors are first diagnosed."