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.

Scott Got­tlieb

“The re­vised draft guid­ance is­sued to­day dis­cuss­es se­lect is­sues com­mon­ly en­coun­tered in rare dis­ease drug de­vel­op­ment. While sim­i­lar is­sues of­ten al­so come up for com­mon dis­eases, they’re fre­quent­ly more dif­fi­cult to ad­dress in the con­text of a rare dis­ease for which there’s of­ten lim­it­ed med­ical and sci­en­tif­ic knowl­edge,” FDA Com­mis­sion­er Scott Got­tlieb said.

Many of the up­dates are in di­rect re­sponse to feed­back that the FDA re­ceived on the first draft, Got­tlieb said. For ex­am­ple, there’s an up­dat­ed sec­tion de­scrib­ing the use of in­for­ma­tion about nat­ur­al his­to­ry as clin­i­cal com­para­tors and ad­di­tion­al in­for­ma­tion on the use of his­tor­i­cal con­trols and ear­ly ran­dom­iza­tion.

In ad­di­tion to the changes, the 10 sec­tions of the guid­ance deal with nat­ur­al his­to­ry stud­ies; dis­ease patho­phys­i­ol­o­gy, clin­i­cal man­i­fes­ta­tions and iden­ti­fi­ca­tion and use of bio­mark­ers; non­clin­i­cal stud­ies; ef­fi­ca­cy end­points; ev­i­dence of ef­fec­tive­ness and safe­ty; phar­ma­ceu­ti­cal qual­i­ty con­sid­er­a­tions; ad­di­tion­al con­sid­er­a­tions (such as ex­pe­dit­ed and pe­di­atric con­sid­er­a­tions); and in­ter­ac­tions with the FDA.

The agency al­so plans to hold a pub­lic meet­ing to ob­tain new pa­tient and care­giv­er per­spec­tives on the im­pacts of rare dis­eases on dai­ly life. The meet­ing will as­sess com­mon ex­pe­ri­ences that may help the FDA and med­ical prod­uct de­vel­op­ers fur­ther un­der­stand and ad­vance the de­vel­op­ment of treat­ments for rare dis­eases, with the po­ten­tial for en­abling nov­el end­points or tri­al de­signs that fo­cus on com­mon­al­i­ties across a va­ri­ety of rare dis­eases.

First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion.

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FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

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