Translarna Fast Track - access to treatments

Translarna (also known as ataluren) is a treatment for people with Duchenne muscular dystrophy with a nonsense genetic mutation, manufactured by pharmaceutical company PTC Therapeutics. It is currently available to eligible children aged 2 and over with Duchenne muscular dystrophy through a Managed Access Agreement (MAA) in England (which also covers Wales and Northern Ireland) and through the ultra-orphan pathway in Scotland.

In May 2021, NICE announced that the MAA had been extended from June 2021 to January 2023, by which point they will have made a full assessment of whether it should be provided on the NHS in the longer-term. In Scotland, access through the ultra-orphan pathway is available until early 2024.

MDUK is a member of the Managed Access Oversight Committee (MAOC) for Translarna and will be working with the Duchenne community to fully engage in both the NICE and SMC processes.

2022 NICE Appraisal

On 30 September 2022, NICE published its draft guidance on Translarna (also known as ataluren)

The draft guidance recognises the clinical effectiveness of Translarna and that the treatment is likely to slow the progression of Duchenne muscular dystrophy.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This has led to the draft guidance recommending that while anyone currently receiving Translarna should continue to do so after the MAA ends in January 2023, the NHS should not provide the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.

The uncertainties around the cost-effectiveness of Translarna highlighted in the draft guidance include how quality-of-life differences between people receiving Translarna and other people with Duchenne muscular dystrophy are measured, how caregivers’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

Muscular Dystrophy UK (MDUK) has been working alongside individuals, families and other patient organisations throughout the MAA and the appraisal process. We welcome the recognition of the clinical effectiveness of Translarna and the news that people currently receiving it will be able to continue to do so until they and their NHS clinician jointly consider it appropriate to stop.

We are, however, very disappointed that at this stage it is not being recommended for people diagnosed after January 2023. It is not unusual for a treatment to receive this type of decision at this stage and for it to then be recommended for use after the consultation stage. MDUK will continue to work in partnership with individuals, families and other patient groups to engage in the consultation process. We urge NICE and PTC Therapeutics, the company that manufactures Translarna, to work together without delay to address the concerns around cost-effectiveness that have been raised at this stage, and for all parties to show flexibility to ensure that people diagnosed after January 2023 can have access to the treatment.

On Wednesday 5 October we held a community virtual briefing to explain the draft decision and outline next steps. You can view the recording here.