A Kaiser Health News investigation claims that the Orphan Drug Act is being manipulated by drugmakers to maximize profits and to protect niche markets for medicines already being taken by millions.
Passed into law over 30 years ago, an approval of a medicine for an orphan disease grants drugmakers seven years of exclusive rights to the marketplace, as an incentive for companies to develop new drugs for people whose rare diseases had been ignored. But KHN’s investigation found that about a third of orphan approvals by the FDA since the program began have been either for repurposed mass market drugs or drugs that received multiple orphan approvals.
According to the report, "The companies aren’t breaking the law but they are using the Orphan Drug Act to their advantage in ways that its architects say they didn’t foresee or intend."
The extent to which companies have been winning approval for drugs that aren’t what advocates call “true orphans” hadn’t been documented until the investigation, says Kaiser Health News.