Pfizer Loses Appeal on Copay for Rare Heart Disease Drug

The United States Court of Appeals for the Second Circuit upheld a lower court ruling that Pfizer’s $13,000 co-payment for tafamidis, the company’s medicine for a rare and progressive heart condition, violated the federal anti-kickback statute.

The three-judge panel sided with the U.S. Department of Health and Human Service Office of Inspector General, which had issued an advisory opinion in 2019 that found Pfizer’s Direct Copay Assistance Program for tafamidis violated the law. A district court in September 2021 granted a summary judgement to HHS when Pfizer first brought suit as it rejected the drugmaker’s argument that the anti-kickback statute requires an element of “corrupt” intent.

Transthyretin amyloid cardiomyopathy (ATTR-CM) is caused by destabilization of a transport protein called transthyretin, which is composed of four identical subunits known as tetramers. In ATTR-CM, heart failure occurs when unstable tetramers dissociate, resulting in misfolded proteins that aggregate into amyloid fibrils and deposit in the heart.

Tafamidis (marketed as Vyndaqel)is a small molecule that selectively binds at specific sites on the transthyretin tetramer to prevent destabilization of the transthyretin transport protein and formation of amyloid that causes ATTR-CM. Tafamidis is the only drug approved by the United States Food and Drug Administration to treat ATTR-CM. It’s an expensive drug with an annual cost of $225,000 per year.  

Pfizer worried that patients would not be able to afford the copayment and sought to cover that directly while making patients responsible for just $35 a month.

The anti-kickback statute was established to combat Medicare and Medicaid fraud. It prohibits providing any payment to induce a person to buy a federally reimbursable healthcare product. The court rejected the company’s argument that there must be a corrupt intent and found that both the court and HHS had properly interpreted the plain meaning of the statute.

Author: Rare Daily Staff