Doctors involved in Pfizer-funded clinical trials for tafamidis have since become critics of the drug's $225,000-a-year price after seeing patients' early financial struggles since it was approved in May, reported Bloomberg.
"We can agree to disagree," said researcher Mathew Maurer, who is still involved in an ongoing trial of the drug, "but I had told them I thought $25,000 a year might have been a reasonable cost." Tafamidis, also called Vyndaqel or Vyndamax, is the first-ever medication approved by the FDA for the cardiac form of transthyretin amyloidosis.
Pfizer called the price of the drug appropriate, and said it set the cost based on what it anticipates will be a small number of patients getting it, although if it ends up being more widely used, the company said it may cut the price.
The company estimates that 100,000 to 150,000 individuals in the US have cardiac transthyretin amyloidosis, meeting the criteria for a rare disease, and that today just 4% or 5% of patients know they have it, according to Nolan Townsend, regional president of Pfizer’s North America rare disease unit.
Maurer and collaborators released a cost-effectiveness analysis last week at the American Heart Association’s (AHA) Scientific Sessions meeting, concluding that tafamidis is only cost-effective with a more than 90% price reduction, or a price of $16,563. At current prices, treating an estimated 120,000 patients in the US with tafamidis would "increase annual healthcare spending by $32.3 billion," they noted.
However, Pfizer contested the findings, saying that the analysis is not appropriate for rare-disease drugs or medicines that treat elderly populations, which have less time to benefit.
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