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Medicines for Rare Conditions: The Global Effects of Steep US Prices

Despite their intended use for rare diseases, so-called orphan drugs contribute substantially to prescription drug spending; in fact, it is estimated that in just two years’ time, orphan drugs will constitute more than 20 percent of the market for global pharmaceutical sales. Nowhere is this impact more outsized than in the US, where orphan drugs are routinely priced at hundreds of thousands of dollars annually. But while prices may be especially steep in the US, the effects of US pricing can adversely affect orphan drug pricing elsewhere, too. It’s time for US policymakers to more closely scrutinize orphan drug pricing dynamics.