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New HTA Decisions in England
May 2021
Drug name
VYNDAQEL® (tafamidis)
Decision date
Therapeutic area
Cardiovascular conditions
Therapeutic sub area
Structural heart defects
Not recommended
Tafamidis is not recommended, within its marketing authorisation, for treating wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR‑CM) in adults.
Decision Detail
The clinical evidence for tafamidis came from a phase 3 double-blind randomised controlled trial (ATTR-ACT (pivotal)) and an open-label extension of ATTR-ACT. Evidence shows that it reduces deaths and hospitalisation from conditions affecting the heart and blood vessels compared with placebo. But clinical benefit varies across different types and stages of ATTR-CM. Also, the measure used to assess how severe ATTR-CM is has limitations. This makes it difficult to clearly identify who benefits from tafamidis and whether they should continue treatment. The cost-effectiveness estimates are higher than what NICE normally considers an acceptable use of NHS resources. This is because there is not enough evidence that recommending tafamidis would reduce diagnosis delays and uncertainty about how long the treatment works after it is stopped. So, tafamidis is not recommended.