FDA launch­es pi­lot pro­gram in sup­port of com­plex tri­al de­signs for drugs and bi­o­log­ics

The FDA an­nounced a new pi­lot pro­gram Wednes­day aimed at en­cour­ag­ing the use of com­plex in­no­v­a­tive tri­al de­signs in the de­vel­op­ment of drugs and bi­o­log­ics.

Spon­sors se­lect­ed by the FDA based on the cri­te­ria for par­tic­i­pa­tion in the “Com­plex In­no­v­a­tive De­signs Pi­lot Meet­ing Pro­gram” will be able to en­gage staff in ear­ly dis­cus­sions on ap­proach­es to com­plex de­signs.

Scott Got­tlieb

The pi­lot will re­main op­er­a­tional through 30 Sep­tem­ber 2022, but spon­sors have un­til 30 June 2022 to sub­mit meet­ing re­quests, ac­cord­ing to Wednes­day’s no­tice. Meet­ings un­der the pi­lot will be con­duct­ed by FDA’s Cen­ter for Drug Eval­u­a­tion and Re­search or its Cen­ter for Bi­o­log­ics Eval­u­a­tion and Re­search.

Tri­als de­signed as part of the pi­lot, which ful­fills a com­mit­ment un­der the sixth it­er­a­tion of the Pre­scrip­tion Drug User Fee Act, will be used by the FDA to de­vel­op case stud­ies. Up to two meet­ing re­quests will be ac­cept­ed on a quar­ter­ly ba­sis each year through­out the pi­lot’s du­ra­tion.

Par­tic­i­pa­tion in­volves an ini­tial and a fol­low-up meet­ing on a de­sign and prod­uct with­in about 120 days, and the se­lect­ed spon­sors will re­ceive a sum­ma­ry “with­in 60 days of each meet­ing,” the no­tice adds.

The no­tice al­so ex­plains the process for sub­mit­ting meet­ing re­quests and the rec­om­mend­ed for­mat­ting of the in­for­ma­tion pack­age se­lect­ed spon­sors are re­quired to sub­mit elec­tron­i­cal­ly to the FDA.

Plans to launch the pi­lot were first dis­cussed dur­ing a pub­lic meet­ing in March, though cer­tain con­cerns were raised by stake­hold­ers such as Genen­tech and Bio­gen re­gard­ing flex­i­bil­i­ty and scope.

“Through in­ter­ac­tion be­tween FDA and pi­lot par­tic­i­pants, this pro­gram will help ac­cel­er­ate the de­vel­op­ment of prod­ucts in ar­eas of un­met need, and en­sure knowl­edge ex­change to help ad­vance the sci­en­tif­ic com­mu­ni­ty’s ap­proach to de­sign­ing clin­i­cal tri­als over­all…,” FDA Com­mis­sion­er Scott Got­tlieb said in a Wednes­day post. He al­so point­ed to oth­er re­cent ef­forts aimed at achiev­ing the pi­lot’s goals.

The re­lat­ed ef­forts in­clude new draft guid­ance doc­u­ments on seam­less clin­i­cal tri­als for ex­pe­dit­ed drug de­vel­op­ment and on the use of place­bo-con­trolled tri­al de­signs, both of which were is­sued this month.


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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