Strategic Patient Advocacy vs. Evidence Package: The “Rare” Challenge for Orphan Drug Manufacturers

UPDATE – December 2017: After initially refusing to reimburse EXONDYS 51™, ANTHEM and EMBLEMHEALTH reversed their decisions in late 2017. While EMBLEM opted for a somewhat restrictive policy requiring patients to complete at least 300m in the 6-MWT, ANTHEM decided to cover patients as long as they are ambulatory. In their…

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