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Tafamidis for treating transthyretin amyloidosis with cardiomyopathy

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1 Recommendations

1.1 Tafamidis is not recommended, within its marketing authorisation, for treating wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in adults.

1.2 This recommendation is not intended to affect treatment with tafamidis that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.

Why the committee made these recommendations

ATTR-CM can lead to heart failure, but treatment options are limited to managing symptoms and best supportive care. Accurately diagnosing ATTR-CM is challenging and can take a long time.

Tafamidis is the first treatment for ATTR-CM that aims to treat the disease. Evidence from clinical trials shows that it reduces deaths and hospitalisation from conditions affecting the heart and blood vessels compared with placebo. But inconsistent results on how effective tafamidis is for different types and stages of ATTR-CM mean that the evidence is uncertain. Also, the measure used to assess how severe ATTR‑CM is has limitations, so it is difficult to identify who benefits from treatment and decide who should stop.

Because of this, and uncertainty about the assumptions related to early diagnosis and how long the treatment works after it is stopped, the cost-effectiveness estimates are very uncertain. They are higher than what NICE normally considers an acceptable use of NHS resources so tafamidis is not recommended.