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. 

Paul McKenzie took over as CEO of Australian pharma giant CSL this month, following in the footsteps of long-time CSL vet Paul Perreault.

With an eye on mRNA, and quickly commercializing its new, $3.5 million-per-shot gene therapy for hemophilia B, McKenzie and chief medical officer Bill Mezzanotte answered some questions from Endpoints News this afternoon about where McKenzie is going to take the company and what advances may be coming to market from CSL’s pipeline. Below is a lightly edited transcript.

Boehringer Ingelheim reported human pharma sales of €18.5 billion on Wednesday, led by type 2 diabetes and heart failure drug Jardiance and pulmonary fibrosis med Ofev. Jardiance sales reached €5.8 billion, growing 39% year over year, while Ofev took in €3.2 billion, notching its own 20.6% annual jump.

However, Boehringer is also looking ahead with its pipeline, estimating “In the next seven years the company expects about 20 regulatory approvals in human pharma.”

The FDA today approved Emergent BioSolutions’ Narcan brand naloxone nasal spray for over-the-counter sales. The nod was expected and comes on the heels of a unanimous 19-0 advisory committee vote in favor of approval last month.

The move to OTC means the opioid overdose reversal agent will now be available on grocery, convenience and gas stations shelves, as well as potentially for purchase online.