FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

Usu­al­ly re­served for mak­ing de­ci­sions on drug ap­pli­ca­tions or en­forc­ing what Con­gress stip­u­lates, the FDA is now dip­ping its toe in­to the wild world of con­gres­sion­al pol­i­tics as it at­tempts to fix a ma­jor court de­ci­sion that could have a chill­ing ef­fect on rare dis­ease R&D.

The case in ques­tion from last Oc­to­ber saw a US ap­peals court over­turn a pri­or FDA court win, say­ing that the agency nev­er should’ve ap­proved a rare dis­ease drug be­cause a pre­vi­ous­ly ap­proved but more ex­pen­sive drug with the same ac­tive in­gre­di­ent has or­phan drug ex­clu­siv­i­ty bar­ring such an ap­proval.

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