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    Has all of the relevant evidence been taken into account?

  • Question on Document

    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

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    Are the recommendations sound and a suitable basis for guidance to the NHS?

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Draft guidance consultation

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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 is a progressive condition that can lead to heart failure. Current treatment options are limited to managing symptoms and best supportive care. Tafamidis is the first treatment for ATTR-CM that aims to treat the condition.

Evidence from the main clinical trial shows that tafamidis reduces deaths and hospitalisations from conditions affecting the heart and blood vessels compared with placebo. There is longer-term follow-up evidence from this trial on how long people live when taking tafamidis and how long they take tafamidis for.

There are uncertainties in the economic model for tafamidis, including how long the effect of treatment lasts after it is stopped. The most likely cost-effectiveness estimates are higher than what NICE normally considers an acceptable use of NHS resources. So, tafamidis is not recommended.