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 the availability of givinostat to patients eligible through early access programmes.

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 (EAPs) is not the responsibility of NHS England. Participation in company-led schemes is decided at an individual National Health Service 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 the 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:

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