The National Institute for Health and Care Excellence issued draft guidance Friday recommending against NHS reimbursement of Bayer's Vitrakvi (larotrectinib) for treating advanced NTRK fusion-positive solid tumours, in adults and children who have no satisfactory treatment options. The agency said that at its current price of £15,000 ($19,575) for a 30-day supply, the oral TRK inhibitor "doesn't have the potential to be cost-effective."
NICE's preliminary decision came shortly after the German Institute for Quality and Efficiency in Health Care (IQWiG) published its own review of Vitrakvi, concluding that "data are not yet sufficient for derivation of an added benefit." The drug gained European approval in September as the first treatment in the EU to receive a tumour-agnostic indication.
Meindert Boysen, director of the centre for health technology evaluation at NICE, noted that as the drug targets a rare genetic mutation, "the clinical evidence is usually based on extremely small sample sizes, requiring novel approaches to testing them in clinical trials. Therefore, further data collection is likely to be needed, for example through the Cancer Drugs Fund." European approval was based on pooled data from 102 patients, with results showing that in the primary analysis population, the overall response rate (ORR) was 72%, including 16% complete responses (CR) and 55% partial responses (PR). Meanwhile, in a further analysis including patients with primary central nervous system tumours, the ORR was 67%, including 15% CR and 51% PR.
John Stewart, NHS national director of specialised commissioning, said "it is disappointing that Bayer has not yet been willing to price [Vitrakvi] at a level which represents value for the NHS…however, should they reconsider, the NHS' door remains open for further discussion." The draft guidance is open for consultation until February 6.
NICE is also currently assessing Roche's entrectinib, which the agency noted could become the first histology-independent treatment to be available to patients. The drug is currently approved in Japan and the US under the name Rozlytrek for the treatment of adult and paediatric patients aged 12 years and older with solid tumours that have an NTRK gene fusion and for whom there are no effective treatments.
"We're hopeful that further data collection, coupled with responsible pricing from the companies, will lead to progressive, new treatments like these being available to patients," Boysen remarked. He noted that the two drugs "offer a major change in the treatment of NTRK fusion-positive solid tumours," adding "the introduction of a new NHS Genomic Medicine Service is expected to support the uptake of these progressive therapies, testing tens of thousands of solid tumours per year once it is fully established."
Meanwhile, in its review, IGWiQ highlighted the fact that none of the three Phase I and II studies of Vitrakvi had a comparator arm, making it "very difficult to conduct an early benefit assessment." In response, a Bayer spokeswoman said the NTRK gene fusion, which occurs in only about 0.5% to 1% of patients with solid tumours, is too rare to allow for a control group.
"There are many signs that the drug may have great potential, but even for an 'early benefit assessment' it can sometimes be too early – this is the case when the data presented do not allow conducting meaningful comparisons," commented Stefan Lange, deputy director of IQWiG. "In future we will need robust comparator data for benefit assessments of histology-independent treatments," Lange said, adding "like the Canadians, who have recently denied regular reimbursement of [Vitrakvi]…we see the lack of suitable data as the main obstacle to making adequate assessments."
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