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Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (review of HST3)

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1 Recommendations

1.1 Ataluren is not recommended, within its marketing authorisation, for treating Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene in people 2 years and over who can walk.

1.2 This recommendation is not intended to affect treatment with ataluren that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue until they and their NHS clinician consider it appropriate to stop. This decision should be made jointly by the clinician, the child or young person, and their parents or carers.

Why the committee made these recommendations

This guidance reviews the evidence for ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene. It includes new real-world evidence (evidence collected outside clinical trials) on ataluren and evidence collected as part of the managed access agreement for NICE highly specialised technologies guidance 3.

Duchenne muscular dystrophy, with a nonsense mutation in the dystrophic gene, is a rare and progressive condition. Over time it causes muscle weakness resulting in the loss of the ability to walk and reductions in respiratory ability, and it significantly reduces life expectancy. Current treatment options are limited.

The company used an indirect treatment comparison of ataluren compared with best supportive care based on 2 real-world evidence studies to estimate treatment benefits. The company did not use data from the managed access agreement in its economic model because it believed it did not provide the most appropriate outcome measures. The evidence provided, along with feedback from clinicians and patients, suggests that ataluren is likely to slow down disease progression and delay the time at which the ability to walk is lost.

Evidence for improvements in later stages of the disease and improved survival with ataluren is limited and highly uncertain but ataluren may also improve outcomes once the ability to walk has been lost.

The cost-effectiveness estimates are uncertain because of how treatment benefits were estimated and the limitations of the clinical effectiveness data. There is uncertainty around the estimated costs of ataluren in the company's model. The way that caregivers' quality of life was included in the model was not realistic so this was considered qualitatively. If the committee's preferred assumptions are used, the cost-effectiveness estimates for ataluren are substantially above the range that NICE considers acceptable for highly specialised technologies. Therefore, ataluren is not recommended.