Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what steps NHS England is taking to ensure that NHS Trusts are able to deliver emerging treatments for Duchenne muscular dystrophy when they are needed by patients.

The Department understands the impact that Duchenne muscular dystrophy has on those living with it and their families, and the urgent need for new treatment options. If new therapies for Duchenne muscular dystrophy are approved by the National Institute for Health and Care Excellence (NICE), then appropriate commissioning plans will be put in place to enable equitable access to treatment through Specialised Neurology Services.

The delivery of timely and equitable access to new treatments for Duchenne muscular dystrophy under company-sponsored early access schemes is not the responsibility of NHS England. Individual pharmaceutical 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 National Health Service trust level, and under these programmes, the cost of the drug is free to both patients taking part in it, and to the National Health Service, although NHS trusts must still cover administration costs and provide clinical resources to deliver the EAP.

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, which can create significant pressures on clinical and financial resources. Companies providing a sponsored EAP also reserve the right to limit or to close registration of new patients at any time, meaning that any financial and clinical investment made by trusts to establish an EAP could be undermined by a commercial decision that would most likely happen in the event of the treatment not being recommended following an appraisal by NICE.

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:

https://www.england.nhs.uk/long-read/free-of-charge-foc-medicines-schemes-national-policy-recommendations-for-local-systems/