Four Doctors Who Worked on the Development of Tafamidis Criticize its Price


A recent article in Bloomberg’s SFGate focused on the price of drugs. The average patient cannot afford the price of many drugs now on the market. Insurance companies have denied coverage on some drugs and many retired people are forced to make a choice between buying food or paying for treatment.

These discussions are very common. However, it is highly unusual for a lead author and his associates to be critical of a company while being directly involved with a study that led to a breakthrough therapy.

Dr. Matthew Maurer spoke to a recent gathering of cardiologists in Philadelphia about the benefit of recently approved Tafamidis (trade name Vyndaquel) to treat cardiac transthyretin amyloidosis (ATTR-CM).

Dr. Maurer explained his objections to the cost of the drug. As Dr. Maurer is still involved with the Pfizer trial, he joked that next year he may be out of a job. Information about ATTR-CM can be found here.

About Tafamidis

Tafamidis (Vyndaquel) is the first medication approved by the FDA to treat the early stages of the cardiac form of ATTR-CM.

The disease is a result of abnormal deposits of protein that build up in the body’s tissues and organs. The symptoms are fatigue, problems breathing, and accumulation of fluid. Any or all of these symptoms may lead to an early death.

The heart is affected by the cardiac form of the disease. This form of ATTR-CM leads to arrhythmia (abnormal heartbeat), orthostatic hypertension, and an enlarged heart.

Tafamidis functions by slowing the disease, avoiding hospital stays and prolonging the life of the patient.

 Pfizer’s Position on Cost

Pfizer’s spokesman explained to Bloomberg that the cost of Tafamidis is based on its expectations of a low number of sales. The company added that if the drug is used more widely than anticipated they would reduce the price.

Tafamidis was developed to treat cardiac transthyretin amyloidosis. However, there is an ongoing debate as to whether cardiac transthyretin amyloidosis is a rare disease when it has been found to closely resemble common heart failure.

The position that Pfizer has taken, which a price of $255,000 per year, takes into consideration the benefit to patients, the prevalence of the disease and an estimated 150,000 patients with cardiac transthyretin amyloidosis. This number puts the disease in the rare disease category.

Putting the company’s estimates aside, Pfizer has admitted that there is a possibility that Tafamidis has the potential to be a “blockbuster”. This statement is based on the drug bringing in approximately $160 million in 2019’s third quarter which topped Wall Street’s estimate.

Pfizer’s Acknowledgement

 A spokesman for the company said that it recognizes the uncertainty about the actual number of people who have the disease. He still referred to the condition as rare but added that it is significantly under-diagnosed. Pfizer is currently conducting two studies for an in-depth understanding of the ATTR-CM.

It is noteworthy that initially, to diagnosis cardiac transthyretin amyloidosis, an invasive heart biopsy was necessary. This procedure called for a catheter. It appears that because of the previous lack of corrective therapy, doctors had little incentive to perform these tests.

Further Consideration

A follow-up email was sent by Pfizer stating that hospital costs will be reduced to about $34,000 per year. This information was provided by a study of Medicare patients and funded by the pharmaceutical industry.

If, however, it is determined that cardiac transthyretin amyloidosis is not a rare disease, he said that the price will be adjusted accordingly. There was no indication about when such a decision would be made.

Tafamidis and other oral medications are fairly inexpensive to produce.  However, it can cost millions of dollars to develop and take the product to market. Manufacturers say that in order to be encouraged to develop new therapies, they should be able to recoup their investment.

The Opposition

Dr. Mauer and his associates expect Tafamidis to be prescribed to many more patients and for many more years than originally anticipated by Pfizer. Another objection is that the drug is being sold at a price comparable to drugs that cure diseases or at prices similar to a medication that treats extremely rare diseases.

Dr. Mauer said that the drug’s cost of $651 per day is what most people on a fixed income pay for a month’s groceries.

Pfizer’s Financial Assistance to Patients

Pfizer’s co-pay assistance program provides assistance to people who have commercial insurance. Pfizer also provides free drugs to those patients who do not have insurance or are underinsured, including those on Medicare.

Unfortunately, not all patients meet the program’s criteria. Pfizer’s spokesman explained that due to legal restrictions, Pfizer is not able to assist Medicare patients in the same way that it gives assistance to people with commercial insurance.

 The Controversy

ATTR-CM’s similarity to common heart disease is the basis for the current controversy. Until more definitive numbers are established, the company and the opposing doctors can only express their opinion.

The U.S. Government gives a special status to drug manufacturers who develop treatments for rare diseases, including market exclusivity and tax credits that are intended to provide incentives.

New genetic and biological discoveries have enabled drug manufacturers to target smaller groups of rare disease patients who were previously untreated. But working with rare diseases often results in a price tag of $100,000 or more.

Dr. Maurer’s Estimate

Dr. Maurer and his associates prepared and released an analysis that shows Tafamidis would be cost-effective if its price was cut by ninety percent to $16,563.

According to their figures, treating 120,000 people in the United States with the drug would have the effect of increasing health-care costs an estimated $32.3 billion annually.

Pfizer challenged these figures by saying that a new cost benchmark must be created when a new drug is established in an area where one had previously not existed. The company maintains that the analysis should not apply to rare disease drugs that treat the elderly.


Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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