Tafamidis, also called Vyndaqel or Vyndamax, is the first approved drug to treat the cardiac form of a disease called transthyretin amyloidosis, which is caused by protein buildup in the body and can lead to trouble breathing, fatigue and fluid accumulation, and potentially fatal heart problems. The drug slows the disease, helping patients live longer and avoid costly hospital stays.
Pfizer said the $651 per day cost of the drug is appropriate because only a small number of patients are treated with it, and transthyretin amyloidosis is a rare disease.
However, the disease is often mistaken for common heart failure, so many more people than anticipated may likely have it, according to Bloomberg. Some physicians have said tafamidis may be given to far more people than anticipated and will be taken by patients for years or decades.
“It is by no means a rare disease,” Ronald Witteles, MD, a physician at the Palo Alto, Calif.-based Stanford Amyloid Center told Bloomberg. Because of that, “the rationale for pricing in the range that this has been priced disappears.”
Pfizer has said if transthyretin turns out not to be a rare disease, it will revisit the drug’s pricing.
According to a cost-effectiveness analysis released Nov. 17 by some of the physicians speaking out against the prices, tafamidis is only cost-effective with a more than 90 percent price reduction, or $16,563.
Pfizer contested the analysis, saying it wasn’t appropriate for rare-disease drugs or medicines that treat elderly populations, according to Bloomberg.
Read the full article here.
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