Tofersen
(asked on 10th October 2025) - View Source
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, for what reason some people with motor neurone disease are receiving tofersen during its evaluation by the National Institute for Health and Care Excellence; and if he will take steps to make it available to all patients who would benefit from it.
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 the superoxide dismutase – 1 (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 has not yet been able to start the evaluation of tofersen as it is unable to issue guidance on the use of the technology without receiving an evidence submission about the technology’s clinical and cost-effectiveness from the marketing authorisation holder. Therefore, NICE is ready to review tofersen via its HST programme, as soon as Biogen indicates that it is ready to start the NICE evaluation.
I am aware that the marketing authorisation holder has established early access programmes (EAPs) through which some patients are currently accessing tofersen. Participation in company-led schemes is decided at an individual NHS trust level and under these programmes, the cost of the drug is free to both patients taking part in it, and to the NHS, but NHS trusts must still cover the administration costs and must provide clinical resources to deliver the EAP. No assessment has been made of regional variation in access to tofersen through the programme.
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 of these schemes, including assessing suitability and any risks in the short, medium, and long term. The guidance is available at the following link: