Rare Daily Staff

Orphan drugs accounted for only 7.9 percent of the $460 billion in drug sales in the United States in 2016, according to a new study by QuintilesIMS Institute that the National Organization for Rare Disorders commissioned.

The report comes as there is increasing scrutiny over drug prices in general, and high-priced drugs specifically and new questions raised about potential industry abuses of the Orphan Drug Act, which provides economic incentives to companies that develop drugs for patients populations less than 200,000 in the United States.

“As we look at ways to reduce the burden of healthcare costs in the U.S., some people have looked upon orphan drugs as a possible contributing factor,” said NORD president and CEO Peter Saltonstall. “The QuintilesIMS study provides important new perspective that helps to reinforce the basic value of the Orphan Drug Act and to quantify orphan drug spending within the broader context of total drug sales.”

The report calls the Orphan Drug Act has been an important driver for the development of orphan drugs with nearly 600 orphan drugs approved since its passage in 1983. However, it argues that the act has not been a significant driver of healthcare spending in the United States because the small patient populations for these drugs creates relatively low demand for them.

“As our healthcare system evolves and we look at ways to control costs,” Saltonstall said, “it’s important for these conversations to be data-driven and to reflect the realities of our healthcare spending.”

Nord releases a whitepaper arguing against needs to revise the Orphan Drug Act along with the  QuintilesIMS Institute report. Both can be found on the NORD website.

October 17, 2017

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