Tofersen
(asked on 29th April 2025) - View Source
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what assessment his Department has made of the adequacy of access to Tofersen through the Early Access Programme for people with SOD1 motor neurone disease.
Companies may put in place Early Access Programmes (EAPs) to allow for early access to new medicines that do not yet have a marketing authorisation. Participation in EAPs is decided at an individual National Health Service trust level, and under these programmes the cost of the drug is free, both to 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 does not undertake any initiatives to encourage participation in EAPs, which are the responsibility of individual pharmaceutical companies and subject to decision-making by individual NHS trusts.
There are no common clinical, data, or regulatory standards for company-sponsored EAPs, meaning each one demands a new protocol to be devised and delivered by each participating trust. Doing so can create significant pressures on clinical and financial resources. Companies providing a sponsored EAP also reserve the right to limit or to close the registration of new patients at any time, meaning that any financial and clinical investment made by trusts to establish an EAP is done at their own risk, in advance of decisions by the Medicines and Healthcare products Regulatory Agency and the National Institute for Health and Care Excellence.
NHS England has published guidance for integrated care systems (ICS) on free of charge (FoC) medicine schemes, including EAPs, providing advice on potential financial, resourcing, and clinical risks. ICSs should use the guidance to help determine whether to implement any FoC scheme, including assessing suitability and any risks in the short, medium, and long term. The guidance is available at the following link: