FDA chief Scott Got­tlieb promis­es to stream­line drug de­vel­op­ment path­way for bio­phar­ma

FDA Com­mis­sion­er Scott Got­tlieb on Tues­day told a House Ap­pro­pri­a­tions sub­com­mit­tee that the agency plans to “sharply in­crease” its re­lease of dis­ease-fo­cused guid­ance doc­u­ments to help drug­mak­ers de­vel­op new treat­ments.

“Among some of the many ar­eas we’re work­ing on right now are new guid­ance to lay out mod­ern cri­te­ria for the de­vel­op­ment of drugs tar­get­ed to ul­cer­a­tive col­i­tis; rare pe­di­atric can­cers; pe­di­atric HIV; and se­ri­ous, life-threat­en­ing and non-can­cer blood dis­or­ders like aplas­tic ane­mia,” ac­cord­ing to Got­tlieb’s writ­ten tes­ti­mo­ny on the 2019 bud­get.

The new guid­ance doc­u­ments will aim to make drug de­vel­op­ment more ef­fi­cient and fo­cus more on de­vel­op­ing drugs tar­get­ed to less-com­mon con­di­tions where there’s a lack of avail­able ther­a­py and de­vel­op­ment path­ways can be chal­leng­ing.

“For ex­am­ple, the guid­ance on blood dis­or­ders will al­low drug de­vel­op­ers to re­duce the use of an­i­mal test­ing and will out­line ways to mea­sure ben­e­fit that may per­mit more ef­fi­cient de­vel­op­ment pro­grams and ear­li­er ap­provals,” his tes­ti­mo­ny said.

He al­so not­ed how clin­i­cal tri­al de­sign “can be par­tic­u­lar­ly hard and guid­ance in this area will re­duce reg­u­la­to­ry un­cer­tain­ty and pro­vide clar­i­ty to drug de­vel­op­ers. The fo­cus of this pol­i­cy work mir­rors the gen­er­al di­rec­tion of drug de­vel­op­ment, where new sci­ence is en­abling us to dis­cov­er nov­el, safe and ef­fec­tive treat­ments for pre­vi­ous­ly in­tractable ill­ness­es.”

In ad­di­tion to the guid­ance, he said FDA is look­ing to set up a pol­i­cy of­fice‎ in­side the Of­fice of New Drugs in CDER that will “dis­till and align reg­u­la­to­ry, clin­i­cal and sci­en­tif­ic rea­son­ing of re­view di­vi­sions to pro­mote pol­i­cy trans­paren­cy and con­sis­ten­cy.”

Dis­cus­sions be­tween rep­re­sen­ta­tives and Got­tlieb in Tues­day’s hear­ing cen­tered around FDA’s work to reg­u­late elec­tron­ic cig­a­rettes, the opi­oid epi­dem­ic, con­tin­u­ous man­u­fac­tur­ing in­no­va­tion and med­ical de­vice safe­ty.


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. 

Im­age: Scott Got­tlieb. AP IM­AGES

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