Givinostat

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.

The National Institute for Health and Care Excellence (NICE) makes recommendations for the National Health Service on whether new licensed medicines should be routinely funded by the NHS independently based on an assessment of their costs and benefits. The NHS in England is legally required to fund medicines recommended by NICE, normally within three months of the publication of final guidance. NICE is currently evaluating givinostat for the treatment of Duchenne muscular dystrophy and its independent Appraisal Committee will meet to consider the evidence in July 2025.

Ahead of NICE’s evaluation, ITF Pharma UK, the United Kingdom’s marketing authorisation holder for givinostat, is providing access to givinostat through a type of compassionate use scheme called an Early Access Programme (EAP). The delivery of timely and equitable access to new treatments for Duchenne muscular dystrophy under company-sponsored EAPs is not the responsibility of NHS England. 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 the 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 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/