June 2, 2016
(STAT News) – The first gene therapies could hit the US market next year, but with their arrival comes a painstaking conundrum: how to pay for single-dose medicines that promise a quick cure, but have sky-high price tags. Consider a drug called Glybera. While approved in Europe to treat an ultrarare enzyme disorder, its $1 million cost precluded coverage in most European countries. The manufacturer subsequently ended plans to win regulatory approval in the United States and sold off its European marketing rights.
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