- A government cost agency in England judged Gilead's cancer cell therapy Yescarta too expensive to be included for coverage through the state-funded health system, potentially throwing a roadblock in the biotech's plans to make the CAR-T treatment commercially available in the country.
- In a draft decision published Tuesday, the National Institute for Health and Care Excellence (NICE) found Yescarta to be clinically effective for treating a type of lymphoma, but concluded there wasn't sufficient evidence to assess the size of benefit over salvage chemotherapy.
- The setback follows Monday's authorization from the European Commission permitting Yescarta's use across EU countries. National health authorities like the U.K.'s NICE, however, can then determine whether approved medicines will be reimbursed under individual country health systems.
NICE is finding itself in more and more headlines as it pushes back against drugmakers seeking coverage for expensive new therapies.
Most notably, Vertex Pharmaceuticals has battled with the agency over the price of its cystic fibrosis therapies, going so far as to request intervention from U.K. Prime Minister Theresa May. Other high-profile drugs, such as Biogen's Spinraza (nusinersen) and Sanofi and Regeneron's Dupixent (dupilumab), have also failed to win over NICE.
NICE took a positive view on Yescarta (axicabtagene ciloleucel)'s efficacy, noting the cell therapy delivered "good response rates, overall survival and progression-free survival" in treating diffuse large B-cell lymphoma.
But, the committee that drafted the initial appraisal document wanted more data comparing Yescarta to salvage chemotherapy to better assess the size of treatment benefit.
Furthermore, Yescarta's cost exceeded cost-effectiveness thresholds of £50,000 (or about $65,000) per quality-adjusted life years, which NICE typically uses to determine whether covering a new therapy is a cost-effective use of National Health Service resources. In the U.S., Yescarta carries a list price of $373,000.
Neither did Yescarta merit inclusion in the Cancer Drugs Fund, the committee said, denying Gilead an alternative route toward gaining interim coverage.
"It's disappointing that patients with non-Hodgkin lymphoma who have exhausted all other treatment options will not be able to access CAR T cell therapy," wrote Raj Chopra, head of cancer therapeutics at The Institute of Cancer Research, London, in an emailed statement on the decision.
"The technique is complex and expensive, but it is also a major advance in cancer treatment that has cured some patients who would otherwise have died."
In an emailed statement, Gilead said it would work with NICE to identify appropriate treatment comparators to Yescarta as it seeks to make the treatment available to U.K. patients.
"Our priority is to make axicabtagene ciloleucel available to patients in the UK as soon as possible and as such we believe we will soon be able to reach an agreement," Gilead said.
NICE will collect comments on its decision through Sept. 18 before meeting again to issue a final ruling. The agency is also reviewing Novartis' rival CAR-T Kymriah (tisagenlecleucel) but has not yet issued a decision.
Kymriah on Monday received European Commission approval for a certain kind of leukemia as well as a type of lymphoma.