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CF case study: How patients can get costly orphans reimbursed

This article was originally published in Scrip

Executive Summary

Companies that want to secure reimbursement for their expensive orphans could learn much from what happened in Ireland with Vertex' cystic fibrosis drug Kalydeco (ivacaftor). When the local health technology assessment recoiled at its €234,000 price tag, patients stepped in to show the National Centre for Pharmacoeconomics the drug's true value.



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