NICE has turned down NHS funding in preliminary guidelines for use of Novatis' Mayzent (siponimod) as a treatment for secondary progressive multiple sclerosis (SPMS) with evidence of active disease.
According to the Institute, clinical trial results show that the drug reduces the number of relapses and slows disability progression compared with placebo.
However, it is uncertain how effective Mayzent is compared with interferon beta-1b – the only disease-modifying treatment available for people with active SPMS – because there is no evidence directly comparing them.
'Because of the limited clinical evidence, the cost-effectiveness estimates are uncertain, and none of the analyses reflect the committee’s preferred assumptions. Therefore, siponimod is not recommended', according to the draft guideline.
Novartis said it is disappointed with the decision.
“Currently, treatment options for people diagnosed with SPMS with active disease are extremely limited and we believe siponimod addresses an unmet need in this patient population,” the drug giant said.
According to the company, the diagnosis of SPMS with active disease is often delayed because of uncertainty around when relapsing remitting MS (RRMS) progresses to SPMS.
“The very limited treatment landscape, together with these diagnostic challenges, often leads to delay and reluctance in confirming a diagnosis of SPMS with active disease. As a result, it is likely that many people with SPMS with active disease continue to receive treatment licensed for use in RRMS, which has not been proven effective for treating their disease.
“Whilst the NICE committee acknowledged the clinical effectiveness of siponimod, which has a sustained effect in delaying disability progression, they concluded that RRMS treatments were not relevant comparators for cost-effectiveness analysis.”
Novartis insists that the comparator should reflect NHS practice, and as many patients with SPMS continue to receive treatment for RRMS, RRMS treatments are the more appropriate comparators for this appraisal.
Showing cost-effectiveness versus no treatment (i.e. best supportive care) is challenging, so this approach could risk patients being left unable to access truly innovative medicines such as Mayzent, it argues.
“If the initial decision from NICE remains unchanged patients will be denied access to the first licensed oral therapy for SPMS with active disease, leaving them without an effective, convenient treatment to manage their condition and help them maintain independence for longer”.
NICE's draft guideline on Mayzent is now open for consultation until July 16.