Rare diseases are receiving increased interest and investment from many stakeholders in healthcare, including biopharma companies, regulators, physicians, payers, and even Congress.

In the past few years, significant progress has been made in rare diseases; in 2014, orphan drugs constituted 17 of the 41 new molecular entities approved by the Food and Drug Administration. An average of 140 drugs have been designated as orphan drugs by the FDA each year over the past decade, compared with 64 in the previous 10 years, according to the Pharmaceutical Research and Manufacturers of America.

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