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. 

John Hood [file photo]

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It will be sold as Rinvoq — at a wholesale price of $59,000 a year — and will likely soon face competition from a drug that AbbVie once controlled, and spurned. Reuters reports that a 4-week supply of Humira, by comparison, is $5,174, adding up to about $67,000 a year.

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ICER blasts FDA, PTC and Sarep­ta for high prices on DMD drugs Em­flaza, Ex­ondys 51

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The final report — which cements the conclusions of a draft issued in May — incorporates the opinion of a panel of 17 experts ICER convened in a public meeting last month. It also based its analysis of Emflaza (deflazacort) and Exondys 51 (eteplirsen) on updated annual costs of $81,400 and over $1 million, respectively, after citing “incorrect” lower numbers in the initial calculations.

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On June 18, 2019, we initiated a confidential arbitration proceeding against Dr. Asa Abeliovich, our former consulting co-founder, related to alleged breaches of his consulting agreement and the improper use of our confidential information that he learned during the course of rendering services to us as our consulting Chief Scientific Officer/Chief Innovation Officer. We are in the early stage of this arbitration proceeding and are unable to assess or provide any assurances regarding its possible outcome.

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Abeliovich and Prevail, though, aren’t taking this one lying down.

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