Motor Neurone Disease: Hampshire
(asked on 27th November 2025) - View Source
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what steps he is taking to ensure that patients with the rare genetic form of Motor Neurone Disease within Hampshire have timely access to Tofersen; and what assessment he has made of regional disparities that prevent this being available across the NHS.
The National Institute for Health and Care Excellence (NICE) is the independent body responsible for developing authoritative, evidence-based recommendations for the National Health Service on whether new medicines represent a clinically and cost-effective use of resources. The NHS in England is legally required to fund medicines recommended by NICE, normally within three months of the publication of final guidance.
NICE has selected tofersen for treating amyotrophic lateral sclerosis caused by SOD1 gene mutations as a topic for guidance development through its Highly Specialised Technology (HST) programme. The HST programme appraises medicines for the treatment of very rare, and often very severe diseases and evaluates whether they can be considered a clinically and cost-effective use of NHS resources. NICE is working with the company to confirm timelines for this evaluation. If NICE is able to recommend tofersen in draft guidance, NHS England is committed to exploring an interim funding agreement with Biogen to use the Innovative Medicines Fund to expedite NHS commissioning of the treatment as soon as a positive recommendation is made by NICE.
No assessment has been made of the extent of regional inequities in the provision of early access programmes for innovative treatments and people living with SOD1 motor neurone disease. Participation in company-led early access schemes is decided at an individual NHS trust level, and under these programmes, the cost of the drug is free to both the patients taking part in it, and to the NHS, although NHS trusts must still cover administration costs and provide clinical resources to deliver the EAP. NHS England has published guidance for integrated care systems (ICS) on free of charge medicines schemes, providing advice on potential financial, resourcing, and clinical risks. ICSs should use the guidance to help determine whether to implement any free of charge scheme including assessing suitability and any risks in the short, medium, and long term. The guidance is available at the following link: