Posted by Michael Wonder on 23 Mar 2017
An FDA program incentivising rare disease drugs will be investigated for abuses
23 March 2017 - In late 1982, Congress overwhelmingly passed the Orphan Drug Act, which was then signed into law by President Ronald Reagan on January 4, 1983 - a long overdue New Year's present for the collective of Americans with devastating rare diseases and their families, who had, until then, gotten the short end of the stick when it came to biopharmaceutical innovation and investment.
The landmark law signalled a paradigm shift in treatments for rare disorders which afflict 200,000 or fewer people. Since its inception, hundreds of drugs to treat genetic diseases, scourges stemming from rare protein deficiencies, and a collection of other maladies have made their way onto the market and provided hope to countless families.
But as with many incentive systems - the Orphan Drug Act gives firms access to beefed-up patent and market exclusivity rights and imposes less stringent clinical trial requirements for treatments with an FDA orphan drug designation - there's the potential for abuse. And after a growing chorus of complaints that some pharmaceutical companies are gaming the system to rake in profits, the Government Accountability Office has now agreed to officially investigate the issue.
