Around 130,000 people across the UK live with multiple sclerosis, and for those with the progressive form of this challenging neurological condition, treatment options remain frustratingly limited. Now, a provisional decision by the National Institute for Health and Care Excellence (NICE) threatens to keep another potential therapy out of reach on the NHS.
NICE has issued draft guidance recommending against the use of Mayzent (siponimod) for treating active secondary progressive multiple sclerosis (SPMS) in NHS England. If this decision stands, patients will not have routine access to this Novartis-developed disease-modifying therapy that has shown promise in clinical trials.
To understand what this means, it's helpful to know that SPMS typically develops after an initial period of relapsing-remitting MS. In this stage, patients experience a gradual worsening of neurological function over time, often with fewer distinct relapses. When doctors describe SPMS as 'active', they mean there's evidence of ongoing disease activity – either through relapses or new lesions visible on MRI scans.
Mayzent is an oral medication designed to slow disability progression in adults with active SPMS. Clinical trials have demonstrated its effectiveness in this specific patient group, offering hope to those facing limited treatment options. However, NICE's role extends beyond clinical effectiveness to include cost-effectiveness assessments. Whilst the full reasoning behind this provisional decision hasn't been detailed, such rejections typically arise from concerns about a drug's cost relative to its benefits, or uncertainties about real-world effectiveness within the NHS.
Novartis has expressed disappointment with the draft guidance, highlighting the significant unmet need for effective SPMS treatments. The company has committed to working with NICE during the consultation process to explore options for reconsideration. Patient advocacy groups are also expected to voice concerns, particularly given the limited therapeutic arsenal currently available for this stage of MS.
Importantly, this is a provisional decision. The draft guidance now enters a consultation period where stakeholders – including patient organisations, healthcare professionals, and the manufacturer – can provide additional evidence and challenge the recommendation. This thorough review process means the final decision could still change.
For the MS community, this represents a concerning setback. Access to effective treatments remains crucial for managing SPMS progression and maintaining quality of life. The coming consultation period will prove pivotal in determining whether Mayzent can still find its way onto the NHS for those who might benefit from it.