Orphan Drugs in the United States
Providing Context for Use and Cost
Institute Report
Oct 17, 2017

There are approximately 7,000 rare diseases and over 25 million patients affected by them in the United States. Rare disease patients and their families form a distinctive community, with half of rare disease patients being children, and with many caretakers and patients actively participating in groups to raise awareness about rare diseases. Although there has been a high level of interest in rare disease medicines among healthcare stakeholders, there still remain significant gaps in patient support, challenges around timely and accurate diagnosis and no treatment options for many patients.


This report provides a historical perspective on the characteristics of rare diseases and their treatment and the role of the Orphan Drugs Act of 1983 in advancing rare disease medicines. It also describes the characteristics of orphan drug volumes and prices as well as placing orphan drugs in the context of specialty drugs and overall medicine spending levels and growth.


The research for this report was undertaken independently by the IQVIA Institute with the support from the National Organization for Rare Disorders (NORD).

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