Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what steps his Department is taking to ensure that variations in local NHS (a) capacity and (b) resourcing of (i) Early Access Programmes and (ii) the availability of tofersen support the reduction of health inequalities.

The Medicines and Healthcare products Regulatory Agency has received the application for tofersen and is currently reviewing it rapidly for quality, safety, and efficacy, for use in the United Kingdom.

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 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.

Companies may put in place Early Access Programmes (EAPs) to allow early access to new medicines that do not yet have a marketing authorisation. Participation in EAPs 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 the administration costs and provide clinical resources to deliver the EAP.