A U.S. medication cost watchdog, after advocating for a significant discount on Pfizer’s widely used tafamidis for transthyretin amyloid cardiomyopathy (ATTR-CM), has issued a final report that proposes a lesser, but still substantial, price reduction request.
The Institute for Clinical and Economic Review (ICER) determined in an updated ATTR-CM study released Monday that Pfizer’s tafamidis medications, Vyndaqel and Vyndamax, need a minimum reduction of 85% off their yearly list pricing of around $268,000 to satisfy widely recognized cost-effectiveness criteria.
According to ICER, to achieve savings within the range of $100,000 to $150,000 every quality-adjusted life year (QALY), tafamidis and its family of transthyretin-stabilizing agents should be priced between $13,600 and $39,000 annually. This indicates a discount ranging from 85% to 95%.
The pricing figures exceed those presented in a July draft report when ICER established the yearly wholesale purchase cost limitations at $5,200 and $36,000 according to the same cost-effectiveness benchmarks.
In a statement, Pfizer highlighted ongoing concerns about the use of QALY as a measure of cost-effectiveness in rare ailments, noting challenges and limitations, especially against elderly patients with severe illnesses who have shorter life expectancies and limited time to benefit from treatments.
The final analysis comes as the FDA is expected to make a decision on BridgeBio Pharma’s competing transthyretin stabilizing agent, acoramidis, next month, and shortly after Alnylam Pharmaceuticals submitted its RNA silencer, Amvuttra, which may alter the ATTR-CM market dynamics.
The ICER pricing range includes both tafamidis and acoramidis. However, Amvuttra is not included. The Alnylam medication was introduced in 2022, receiving FDA clearance for ATTR-polyneuropathy with a yearly cost of $463,500.
The revised assessment incorporates many modeling changes relative to the draft. To clarify the escalation in price range, ICER’s CMO, Dr. David Rind, attributed it to an alteration in mortality estimation methods, which included a recalibration reflecting that tafamidis’s survival advantage became evident only 18 months post-treatment in the phase 3 ATTR-ACT trial. He added that there was also a miscalculation in the prior assessment of a subjective quality of life metric.
The final report once again omitted a financial evaluation of Amvuttra, despite the recent release of findings from the HELIOS-B study indicating that the small-interfering RNA treatment might decrease the risk of mortality from any cause by 35% compared to placebo over 42 months.
The HELIOS-B data was given an ‘A’ rating by ICER, which shows it has “high certainty that treatment with vutrisiran, compared with no disease-specific therapy or when added to tafamidis, provides a substantial net health benefit.”
