US orphan products more than doubled between 2000 and 2008, study finds

The number of orphan product designations in the US increased from 208 to 425 from 2000-02 to 2006-08, a study by the Tufts Center for the Study of Drug Development has revealed.

According to the Boston, US research firm, orphan product designations increased from 208 in the 2000-02 period to 425 in 2006-08, reflecting growing interest by pharmaceutical and biotech companies in developing drugs to treat orphan diseases.

Since the Orphan Drug Act of 1983 became law in the US, more than 2,000 products in development have been designated as orphan drugs, while the US Food and Drug Administration has granted market approval to 350 drugs and biologicals, said Tufts CSDD.

"The Orphan Drug Act, without question, can be considered a success," said associate director Christopher-Paul Milne, who conducted the study. "It has been emulated throughout the world, and today Japan and Europe have similar programmes, while Singapore and Australia give special allowance for the importation and marketing approval of orphan products approved in the US."

From 2000 to 2008, orphan products comprised 22% of all new molecular entities (NMEs) and 31% of all significant biologics (SBs) receiving US marketing approval, the study found.

Orphan products receiving priority review status rose from 35% of all orphan NMEs in 2000-02 to 50% in 2006-08 and during the same periods, orphan SBs receiving priority review status increased from 17-67%.

While biotech firms during the 2000s gained, on average, about one-third of all orphan drug approvals, they received just over 50% of orphan drug designations.