The Institute for Clinical and Economic Review (ICER), a US-based non-profit organization specializing in drug cost-effectiveness analysis, has released a pricing analysis for treatments of the rare heart disease transthyretin amyloid cardiomyopathy (ATTR-CM). The report particularly focuses on Pfizer’s Vyndamax/Vyndaqel (tafamidis) capsules, which were approved for ATTR-CM in the US in 2019. ICER’s assessment indicates that the current annual cost of tafamidis—USD 268,000 at wholesale or approximately USD 194,000 after discounts—is significantly higher than the accepted cost-effectiveness thresholds. The study authors suggest that the annual treatment cost of tafamidis would need to be reduced by at least 96% to align with the commonly accepted cost-effectiveness thresholds for QALY (quality-adjusted life year) gained, ranging between USD 5,200 to USD 10,400.
ATTR-CM is a rare condition characterized by the deposition of misfolded transthyretin (TTR) protein in the heart valves, leading to heart failure symptoms and a shortened lifespan. It affects approximately 50,000 patients in the United States. Tafamidis, a TTR stabilizer, has been shown in a key Phase III trial to reduce the risk of death by 30% compared to placebo and to decrease cardiovascular-related hospitalizations while slowing functional decline and improving quality of life.
Since its market introduction in 2019, tafamidis has facilitated earlier detection of ATTR-CM, enabling treatment of healthier patients in subsequent trials. The drug generated USD 3.3 billion in sales for Pfizer in 2023, marking a 36% year-on-year growth.
The ICER report also evaluates Alnylam Pharmaceutical’s RNAi drug Amvuttra (vutrisiran), approved in 2022 for a different form of ATTR, and BridgeBio Pharma’s acoramidis, a next-generation TTR stabilizer in late-stage development for ATTR-CM. The report is currently in draft form, open for public feedback, with ICER planning to release an updated evidence report on September 5 and a final report on October 21, 2024.- Flcube.com
