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An inquiry into possible abuses of the Orphan Drug Act has been opened by Sen Chuck Grassley (R, Iowa), chairman of the Senate Judiciary Committee, following a report that pharmaceutical companies may be manipulating the law to repurpose existing drugs to treat rare diseases at exorbitant prices.
The finding, reported by a Kaiser Health News (KHN) investigative team in January, detailed how the 1983 law, which provides incentives for drug makers to develop medications for diseases affecting fewer than 200 000 people, may have had the unintended consequence of allowing companies to game the system and drive up the prices of widely used drugs.
Lyon J. Senate Questions Orphan Drug Pricing. JAMA. 2017;317(10):1009. doi:10.1001/jama.2017.1429
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