NICE publishes draft guidance on access to Duchenne treatment Translarna

NICE has published draft guidance stating that although Translarna (also known as ataluren) is clinically effective and an innovative treatment, there is doubt as to its cost effectiveness. NICE’s draft recommendation is that people currently receiving Translarna should continue to have access to it until they and their NHS clinician consider it appropriate to stop, but that it shouldn’t be provided for new patients after January 2023.

On 30 September 2022, NICE published its draft guidance on Translarna (also known as ataluren). Translarna is used to treat Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene and is currently available through a Managed Access Agreement (MAA), which ends in January 2023. Duchenne muscular dystrophy affects around 2,500 people in the UK, and it is estimated that approximately 10% of these people carry a nonsense mutation in the dystrophin gene. 

The draft guidance recognises the clinical effectiveness of Translarna and that the treatment is likely to slow the progression of Duchenne muscular dystrophy. It also recognises that Translarna has a positive impact on the lives of people receiving it and on caregivers. However, at this stage NICE is concerned about the cost effectiveness of the treatment.

This has led to the draft guidance recommending that while anyone currently receiving Translarna should continue to do so after the MAA ends in January 2023, the NHS should not provide the treatment to newly diagnosed patients after that date. This draft recommendation is now subject to consultation until 21 October 2022, with a final decision likely by January 2023. 

NICE guidance is usually mirrored in Wales and Northern Ireland. In Scotland, Translarna is available through the ultra-orphan pathway until early 2024.

The uncertainties around the cost-effectiveness of Translarna highlighted in the draft guidance include how quality-of-life differences between people receiving Translarna and other people with Duchenne muscular dystrophy are measured, how caregivers’ quality of life is measured, and the age at which people are likely to start receiving Translarna.

Action Duchenne, MDUK and Duchenne UK will be submitting a joint response to the NICE consultation and will shortly highlight key areas of input needed.

Take part in the NICE consultation by visiting the ataluren section of the NICE website

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