FDA re­vis­es draft guid­ance on rare dis­ease drug R&D

The FDA has up­dat­ed a 2015 draft guid­ance on rare dis­eases, with new in­for­ma­tion gleaned since the orig­i­nal draft was re­leased.

Among the re­vi­sions to the 24-page draft guid­ance, ac­cord­ing to the FDA, are up­dates to the nat­ur­al his­to­ry stud­ies sec­tion, the in­clu­sion of is­sues for eval­u­at­ing and val­i­dat­ing bio­mark­ers as sur­ro­gate end­points, a dis­cus­sion of ad­di­tion­al flex­i­bil­i­ty on the non-clin­i­cal as­pects of the eval­u­a­tion of nov­el drug com­pounds, the ad­di­tion of a sec­tion de­scrib­ing the eval­u­a­tion of safe­ty ques­tions and added in­for­ma­tion on changes to drug sub­stance or drug prod­uct man­u­fac­tur­ing process with clar­i­fi­ca­tion on ar­eas of ad­di­tion­al flex­i­bil­i­ty.

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