Abstract
In 1983, the US Congress enacted the Orphan Drug Act to incentivize pharmaceutical companies to develop drugs, vaccines, and diagnostic agents for rare diseases.1 The Act offers 7 years of marketing exclusivity, a 25% tax credit (50% before 2017) on qualified research and development costs, and research subsidies for orphan diseases, defined as conditions that affect less than 200,000 people in the United States. Since its passage, the number of orphan drugs has risen dramatically. In 2018, 58% of all new drug approvals had a rare disease indication.
Highlights
I n 1983, the US Congress enacted the Orphan Drug Act to incentivize pharmaceutical companies to develop drugs, vaccines, and diagnostic agents for rare diseases.[1]
TRANSTHYRETIN AMYLOID CARDIOMYOPATHY Consider the case of transthyretin amyloidosis (ATTR) cardiomyopathy (CM), a rare condition in which amyloid deposits build up in the heart, increasing wall thickness and impairing function, often leading to heart failure and arrhythmias.[2]
In May 2019, tafamidis was approved for ATTR-CM, the first drug to receive approval by the US Food and Drug Administration (FDA) for the condition.[3]
Summary
I n 1983, the US Congress enacted the Orphan Drug Act to incentivize pharmaceutical companies to develop drugs, vaccines, and diagnostic agents for rare diseases.[1]. If diseases are diagnosed more frequently after approval, pivotal trial data may misestimate therapeutic efficacy.
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