FDA Com­mis­sion­er Mar­ty Makary is em­brac­ing the Trump ad­min­is­tra­tion's "Amer­i­ca First" mantra, seek­ing po­ten­tial­ly high­er user fees for com­pa­nies op­er­at­ing out­side the US.

"If your Phase 1 tri­al is not in the Unit­ed States, maybe you should pay a high­er user fee," Makary said at a sup­ply chain re­silien­cy meet­ing at the Na­tion­al Acad­e­mies of Sci­ences, En­gi­neer­ing, and Med­i­cine late last month.  The FDA and in­dus­try are about to kick off ne­go­ti­a­tions for the next round of user fees un­der PDU­FA VI­II, which will run from 2028 through 2032.

Asked whether the FDA will call for high­er ex-US user fees in the ne­go­ti­a­tions over the next year, an FDA spokesper­son told End­points News on Tues­day that the ad­min­is­tra­tion "is pri­or­i­tiz­ing an Amer­i­ca first ap­proach to all ac­tiv­i­ties, in­clud­ing drug de­vel­op­ment and re­search. FDA is con­sid­er­ing a wide range of op­tions to sup­port Amer­i­can in­no­va­tion and en­sure the in­tegri­ty and ac­cu­ra­cy of clin­i­cal tri­als for prod­ucts that Amer­i­cans de­pend on."

FDA Com­mis­sion­er Mar­ty Makary spent hours over the week­end at Rick Paz­dur's house try­ing to con­vince the agen­cy's long­time can­cer chief to step up to the role of top drug reg­u­la­tor, a source fa­mil­iar with the mat­ter told End­points News.

Part of that dis­cus­sion in­volved Makary as­sur­ing Paz­dur that as di­rec­tor of the Cen­ter for Drug Eval­u­a­tion and Re­search, he would have au­ton­o­my and in­de­pen­dence from bi­o­log­ics di­rec­tor Vinay Prasad, ac­cord­ing to the per­son, who spoke on con­di­tion of anonymi­ty. Prasad has feud­ed with past FDA lead­ers, over­ruled CBER re­view staff, and specif­i­cal­ly tar­get­ed Paz­dur on­line be­fore Prasad's time at the FDA.

The FDA on Fri­day re­vised the la­bel for Sarep­ta’s Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py Ele­v­idys to add a boxed safe­ty warn­ing and re­strict the ther­a­py’s use to am­bu­la­to­ry pa­tients, re­flect­ing changes that the agency and com­pa­ny pre­vi­ous­ly sug­gest­ed.

The FDA al­so said that it will re­quire Sarep­ta to con­duct an ob­ser­va­tion­al study “to fur­ther as­sess the risk of se­ri­ous liv­er in­jury.” The post­mar­ket­ing study will in­clude around 200 pa­tients — a con­sid­er­able num­ber — and will pe­ri­od­i­cal­ly as­sess their liv­er func­tion for at least a year.

Over the life of the treat­ment, Sarep­ta has on­ly dosed a to­tal of over 1,100 peo­ple with Ele­v­idys across clin­i­cal tri­als and through com­mer­cial chan­nels, the com­pa­ny said in a press re­lease.

Top FDA of­fi­cials on Wednes­day sketched out a new reg­u­la­to­ry ap­proach that aims to keep pace with sci­en­tif­ic ad­vances in per­son­al­ized med­i­cine. It builds off the cel­e­brat­ed case of Ba­by KJ, who was treat­ed with a cus­tom gene ther­a­py ear­li­er this year.

In a pa­per pub­lished in the New Eng­land Jour­nal of Med­i­cine, FDA Com­mis­sion­er Mar­ty Makary and CBER Di­rec­tor Vinay Prasad laid out how drug­mak­ers can reuse tech­nol­o­gy and ear­li­er da­ta from past ef­forts to stream­line fu­ture reg­u­la­to­ry re­views.

This new “plau­si­ble mech­a­nism path­way” al­so opens the door to be­spoke med­i­cines be­ing paid for by gov­ern­ments and in­sur­ers. That could be a po­ten­tial boon for biotechs and aca­d­e­mics who have strug­gled to fund ther­a­pies for rare con­di­tions.

Coun­sel for Eli Lil­ly, No­var­tis and Bris­tol My­ers Squibb told a pan­el of judges that the gov­ern­ment’s re­bate pi­lot pro­gram is too lim­it­ed in scope to ad­dress drug­mak­ers’ broad­er con­cerns with the 340B sys­tem.

HH­S' Health Re­sources and Ser­vices Ad­min­is­tra­tion re­cent­ly ap­proved ap­pli­ca­tions from nine drug­mak­ers to par­tic­i­pate in a pi­lot that would sig­nif­i­cant­ly change the way the 340B dis­count pro­gram op­er­ates. For now, it on­ly ap­plies to the 10 drugs that were sub­ject to the first round of Medicare ne­go­ti­a­tions un­der the In­fla­tion Re­duc­tion Act.

“The prob­lem with the pi­lot pro­gram, of course, is it on­ly ap­plies to man­u­fac­tur­ers whose drugs have been cho­sen for drug price ne­go­ti­a­tion and on­ly to those drugs,” Cather­ine Stet­son, a Hogan Lovells at­tor­ney rep­re­sent­ing the three drug­mak­ers, said dur­ing a Mon­day oral ar­gu­ment be­fore the US Court of Ap­peals for the DC Cir­cuit.