GAO Highlights 2022: Rare Diseases

By definition, few people have any specific rare disease. But there are many different rare diseases—about 7,000—and as a result, an estimated 30 million people in the United States have one or more of them. About half of those with a rare disease are children. Often genetic, many rare diseases are chronic, progressive (they worsen over time), and life-threatening. Relatively little is known about the costs of rare diseases, either individually or collectively.