NICE has issued draft guidance for public consultation which does not recommend Novartis’ Mayzent (siponimod) for treating secondary progressive MS.
Siponimod is licensed to treat secondary progressive multiple sclerosis in adults with evidence of active disease (where people have relapses or features of inflammatory activity that show up on imaging).
Interferon beta-1b is currently the only disease-modifying treatment available for people with active secondary progressive MS. However, NICE says that few people take it because it can cause side effects such as flu-like symptoms.
Clinical trial results show that siponimod reduces the number of relapses and slows disability progression compared with placebo. However, NICE explains that because there is no evidence directly comparing siponimod with interferon beta-1b, it is uncertain how effective siponimod is compared with that treatment. Because none of the analyses reflected the committee’s preferences and the clinical evidence was limited, the independent appraisal committee was unable to recommend siponimod for use in the NHS in England.
The committee has requested further analyses to be included in the company’s economic model. These include comparing siponimod with best supportive care and assuming that there is a reduction in its treatment effect over time.
Meindert Boysen, Deputy Chief Executive and Director of the Centre for Health Technology Evaluation at NICE, said: “We know there are currently few, if any treatments available for people with this form of MS, and that siponimod is a promising drug that has the potential to address this unmet clinical need. We are, therefore, committed to working with the company to help them address the issues identified by the committee that are highlighted in this draft guidance.”
Siponimod costs £1,643.72 per pack of 28 tablets at its list price. The company has agreed a confidential commercial arrangement which would have made siponimod available at a discounted price.
Novartis released a statement on the decision saying: “We at Novartis UK are disappointed by the initial decision from the National Institute for Health and Care Excellence (NICE) not to recommend Mayzent® (siponimod) for the treatment of secondary progressive multiple sclerosis (SPMS) with active disease, for routine use on the NHS. 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 diagnosis of SPMS with active disease is often delayed or avoided due to 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. We believe that the comparator should reflect NHS practice and that since many patients with SPMS continue to receive treatment for RRMS, RRMS treatments are the more appropriate comparators for this appraisal. Demonstrating 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 siponimod.
“Treatment with siponimod has also been shown to have a significant benefit on cognitive processing speed, the cognitive function most frequently affected by MS. Cognitive changes, including problems with memory, attention span, decision making, and understanding, can significantly impact quality of life and are a key concern for people living with MS.
“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.
“We are committed to continuing to work closely with NICE to address outstanding questions and data requirements in order to secure access as quickly as possible to siponimod for those patients in England and Wales that could benefit.”