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Tafamidis Received NICE Recommendation for Treating Transthyretin Amyloidosis with Cardiomyopathy | NICE | UK | iPharmaCenter

Tafamidis has been recommended for treating wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in adults, within its marketing authorization. This recommendation is contingent upon the company providing the drug under a specific commercial arrangement.

Tafamidis vs. Best Supportive Care (BSC)

The company suggests that best supportive care (BSC), which involves managing symptoms and providing supportive treatments like diuretics, is the most appropriate comparator for tafamidis in treating ATTR-CM. Some patients with ATTR-CM also experience polyneuropathy with mixed clinical features, which were included in the evaluation scope for comparison.

Clinical Trial Data

Recent clinical data from the ATTR-ACT LTE study, with 84 months of follow-up, provided updated information on the effectiveness and safety of tafamidis. This data includes overall survival (OS) and time-to-treatment discontinuation (TTD) for tafamidis, but specific figures are kept confidential. The study also presented all-cause mortality data and adverse events, focusing on different New York Heart Association (NYHA) classes.

Economic Model and Cost-Effectiveness

The company's economic model for tafamidis employs a cohort-level Markov state-transition approach, based on NYHA classification to reflect the natural progression of ATTR-CM. This model includes five health states: four NYHA classes (1, 2, 3, and 4) and death. Patients can transition to more severe or less severe health states over time.

Utility Values

In the company's submission, utility values for patients in NYHA class 1 and 2 were higher than the UK general population's age-matched average. The committee decided it was appropriate to cap these utility values to the age-matched average, as it was unrealistic to assume patients with ATTR-CM would have a better quality of life than the general population.

Acceptable Incremental Cost-Effectiveness Ratio (ICER)

The National Institute for Health and Care Excellence (NICE) guidelines suggest that an acceptable ICER should fall between £20,000 and £30,000 per quality-adjusted life year (QALY) gained. The committee acknowledged the substantial unmet need for ATTR-CM treatments in England and accepted the company's updated approach to modeling tafamidis's cost-effectiveness.

The committee concluded that tafamidis is a cost-effective use of NHS resources, given the substantial unmet need and the company's reasonable updated approach. Therefore, tafamidis is recommended for treating wild-type or hereditary ATTR-CM, providing a new therapeutic option for patients with this life-limiting condition.

About Transthyretin Amyloidosis (ATTR)

ATTR is a condition caused by abnormal transthyretin proteins produced by the liver, which accumulate as amyloid deposits in various tissues of the body. When these deposits primarily accumulate in the heart, the condition is known as ATTR-CM. There are two types of ATTR-CM:

  • Wild-type ATTR-CM: More common and typically affects older men.

  • Hereditary ATTR-CM: Also known as familial amyloid cardiomyopathy, this type is linked to inherited mutations in the transthyretin gene.

Symptoms and Impact

Clinical experts describe ATTR-CM as a progressive and debilitating disease with a significant impact on life expectancy. Symptoms for wild-type ATTR-CM usually begin after age 70, while hereditary ATTR-CM symptoms often start in individuals over 60. The disease can cause shortness of breath, irregular heartbeats, ankle swelling, fatigue, fainting, and chest pain. Most deaths in ATTR-CM patients result from sudden cardiac events or progressive heart failure.


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