On May 3, 2019, the US Food and Drug Administration (FDA) announced approval of the first treatments for cardiomyopathy caused by transthyretin amyloidosis (ATTR-CM). The FDA called the approvals of tafamidis meglumine and tafamidis “an important advancement” in the treatment of ATTR-CM, a “rare, debilitating, and often fatal disease.”1 Both of the approved formulations are lifelong treatments. The FDA granted tafamidis meglumine fast track, priority review, and breakthrough therapy designations and tafamidis meglumine and tafamidis received orphan drug designations. The approval of these novel therapies, their potential applicability to a patient population larger than recognized by the FDA, and their very high price present important challenges associated with access and affordability that will affect patients, clinicians, payers, and policymakers. This Viewpoint discusses these issues and presents recommendations that have heightened relevance as novel, very expensive therapies continue to emerge for previously untreatable conditions.
Gurwitz JH, Maurer MS. Tafamidis—A Pricey Therapy for a Not-So-Rare Condition. JAMA Cardiol. 2020;5(3):247–248. doi:10.1001/jamacardio.2019.5233
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