NEW YORK – The UK's National Institute for Health and Care Excellence (NICE) on Thursday recommended Johnson & Johnson's FGFR inhibitor Balversa (erdafitinib) as an option for National Health Service (NHS) patients with metastatic or unresectable FGFR3-altered urothelial cancer who have previously received at least one line of therapy containing a PD-1 or PD-L1 inhibitor.
In a final draft guidance, NICE said it recommended the drug based on results from the Phase III THOR trial which compared Balversa to vinflunine or docetaxel chemotherapy in patients with FGFR2- or FGFR3-altered advanced urothelial cancer who had received prior immunotherapy. In the trial, patients on Balversa lived about one year compared to 7.8 months on chemo. The treatment also improved the median progression-free survival, which was 5.5 months on Balversa and 2.7 months on chemo.
In December 2024, NICE had initially said in a draft guidance that it wouldn't recommend Balversa in this setting due to uncertainties in J&J's clinical comparisons and cost-effectiveness models. The NICE committee noted that there was no direct comparison for Balversa to the standard treatment for metastatic urothelial cancer, paclitaxel with or without carboplatin, and to best supportive care. NICE further said the population of the trial supporting Balversa did not reflect people treated within the NHS.
Since then, J&J conducted a retrospective real-world study using data from the National Cancer Registration and Analysis Service (NCRAS) and assessed the use of different chemotherapies in metastatic urothelial cancer. They also used the NCRAS data to conduct an indirect comparison of Balversa to the preferred standard treatment at NHS, paclitaxel with or without carboplatin. That indirect comparison showed Balversa had improved the time to next treatment and overall survival compared to paclitaxel with or without carboplatin.
"There are limited treatment options for this devastating and debilitating disease, which in many cases responds poorly to immunotherapies, so I'm sure today's guidance will be welcomed by patients," Helen Knight, director of medicines evaluation at NICE, said in a statement. "One of the benefits of this drug is it can be taken in the comfort of the patients' own home rather than having to travel to hospital to receive the treatment."
The list price of Balversa is £12,750 ($16,345) for 28 days of tablets. NICE said the drug was made available to the NHS at a confidential discount. Evaluating a patient for FGFR3 alterations would mean adding this gene to next-generation sequencing panels, which would cost £37.33, since routine testing for this biomarker is currently not standard practice in the NHS. J&J divided that cost by the expected prevalence of FGFR3 alterations (16.6 percent) to estimate that it would cost £224.85 to identify a single person with FGFR3 mutation-positive unresectable or metastatic urothelial cancer.
Based on this analysis, NICE concluded that Balversa was a cost-effective use of NHS resources, though it noted it was toward the upper end of the range. NICE estimated that about 423 NHS patients each year would benefit from the treatment.
Balversa was also approved in this setting in the US in January 2024 and in Europe in August 2024. The US accelerated approval indication from 2019 initially included both FGFR2- and FGFR3-altered metastatic urothelial cancer patients, but it was narrowed to only FGFR3 patients in 2024.