Today is Rare Disease Day, which serves as a time to recognize the more than 30 million Americans living with some type of rare disease. These diseases can be acute or chronic in nature and are generally defined as affecting fewer than 200,000 people in the United States. They include certain types of cancers, autoimmune disorders, digestive disorders, infectious diseases, neurological disorders and many other types of illness.
Although biopharmaceutical researchers have made tremendous progress in advancing innovation for rare diseases, 95 percent of rare diseases still do not have any treatment options, representing a significant unmet need for patients. America’s biopharmaceutical companies are committed to developing new medicines for patients with rare diseases, and because of their dedication, the pipeline of new medicines has never been more promising. The following numbers help paint a picture of this challenging but hopeful research landscape.