FDA's Got­tlieb out­lines new goals in bud­get plan, look­ing to spur drug re­views

Scott Got­tlieb

Pres­i­dent Don­ald Trump’s bud­get sought a boost in fund­ing for the FDA and now FDA Com­mis­sion­er Scott Got­tlieb is of­fer­ing his own take on the ini­tia­tives and in­vest­ments the agency plans to pur­sue.

Plans out­lined by Got­tlieb in­clude the cre­ation of two new cen­ters re­lat­ed to drug com­pound­ing and dig­i­tal health.

Pro­mote Do­mes­tic Drug and Bi­o­log­ic Man­u­fac­tur­ing

As phar­ma­ceu­ti­cal and biotech com­pa­nies move to­ward ad­vanced man­u­fac­tur­ing tech­nolo­gies, such as con­tin­u­ous man­u­fac­tur­ing, Got­tlieb said vac­cine sup­plies can be “more eas­i­ly ramped up on short no­tice, and vac­cines them­selves adapt­ed over a short­er time pe­ri­od to ad­dress in­fec­tious dis­eases, such as the flu. The ap­pli­ca­tion of this kind of en­abling tech­nol­o­gy to vac­cine pro­duc­tion has long been a strate­gic pri­or­i­ty for the US.

“By de­vel­op­ing a sci­ence-based frame­work that in­cludes the reg­u­la­to­ry tools and guid­ance for how prod­ucts de­vel­oped in these sys­tems will be eval­u­at­ed, and by fund­ing re­search, de­vel­op­ment and test­ing of the en­abling tech­nolo­gies, the agency can help re­duce the cost and un­cer­tain­ty of adopt­ing these new man­u­fac­tur­ing plat­forms,” he added.

These “small-foot­print, high-tech­nol­o­gy man­u­fac­tur­ing plat­forms” could “re­turn prod­uct man­u­fac­tur­ing to do­mes­tic sites, help­ing to fos­ter job cre­ation,” Got­tlieb said.


FDA is propos­ing to cre­ate, as it did for on­col­o­gy, a “Cen­ter of Ex­cel­lence on Com­pound­ing for Out­sourc­ing Fa­cil­i­ties,” with ex­pand­ed FDA en­gage­ment with out­sourc­ing fa­cil­i­ties and states to help the phar­ma­cy out­sourc­ing in­dus­try grow to meet its in­tend­ed func­tion and ad­here to high­er qual­i­ty stan­dards to pro­tect pa­tient health.

“The Cen­ter of Ex­cel­lence would iden­ti­fy and pro­pose so­lu­tions to mar­ket bar­ri­ers to low­er the cost for phar­ma­cies to be­come out­sourc­ing fa­cil­i­ties. The Cen­ter would pro­vide much-need­ed ed­u­ca­tion and train­ing to im­prove prod­uct qual­i­ty, safe­ty and pur­chas­er con­fi­dence, and help the FDA ad­just its reg­u­la­to­ry over­sight to bet­ter match the scope of pro­duc­tion of an in­di­vid­ual com­pound­ing phar­ma­cy,” Got­tlieb said.

Ad­vance Med­ical De­vice Man­u­fac­tur­ing and Qual­i­ty

FDA is look­ing to es­tab­lish a vol­un­tary pro­gram for de­vice man­u­fac­tur­ers to re­ceive cer­ti­fi­ca­tion for meet­ing ob­jec­tive man­u­fac­tur­ing and prod­uct qual­i­ty cri­te­ria.

“This more mod­ern and nim­ble frame­work would make it more ef­fi­cient for de­vice de­vel­op­ers to in­no­vate man­u­fac­tur­ing process­es in ways that can al­low de­vices to bet­ter meet the needs of pa­tients and the ex­pec­ta­tions of providers – such as through in­tel­li­gent, au­to­mat­ed process­es that mon­i­tor and record man­u­fac­tur­ing qual­i­ty met­rics, in­cor­po­rat­ing fea­tures and tech­no­log­i­cal char­ac­ter­is­tics that can con­tribute to bet­ter op­tions and high­er qual­i­ty that achieves their clin­i­cal pur­pose,” Got­tlieb said.

FDA al­ready is work­ing through the Med­ical De­vice In­no­va­tion Con­sor­tium to de­vel­op the pa­ra­me­ters of the pro­gram.

As part of this ap­proach, the FDA would rec­og­nize third-par­ty cer­ti­fiers and of­fer reg­u­la­to­ry in­cen­tives for those man­u­fac­tur­ers who re­ceive cer­ti­fi­ca­tion demon­strat­ing their qual­i­ty ca­pa­bil­i­ty.

Ad­vance the Use of Re­al-World Ev­i­dence

Got­tlieb said the FDA will ad­vance the use of re­al-world ex­pe­ri­ence “to bet­ter in­form pa­tient care and pro­vide more ef­fi­cient, ro­bust and po­ten­tial­ly low­er-cost ways to de­vel­op clin­i­cal da­ta that can in­form prod­uct re­view and pro­mote in­no­va­tion.”

As part of such ef­forts, he said the FDA will es­tab­lish the abil­i­ty to con­duct near-re­al-time ev­i­dence eval­u­a­tion down to the lev­el of in­di­vid­ual elec­tron­ic health records for at least 10 mil­lion peo­ple in a broad range of US health­care set­tings.

“To­ward these ends, an ex­pand­ed use of nat­ur­al lan­guage pro­cess­ing for the as­sess­ment of in­for­ma­tion sub­mit­ted to the agency would be de­vel­oped in an ef­fort to marked­ly speed recog­ni­tion and re­me­di­a­tion of emerg­ing safe­ty con­cerns,” he added.

Dig­i­tal Health Tech­nol­o­gy

The FDA is work­ing col­lab­o­ra­tive­ly with in­dus­try, pa­tients and providers to es­tab­lish a new par­a­digm for dig­i­tal health tech­nolo­gies un­der which a com­pa­ny could mar­ket low­er-risk prod­ucts with­out FDA pre­mar­ket re­view and mar­ket high­er-risk prod­ucts fol­low­ing a stream­lined FDA pre­mar­ket re­view if the com­pa­ny re­ceives a pri­or third-par­ty cer­ti­fi­ca­tion for en­gag­ing in high-qual­i­ty soft­ware de­sign and test­ing (val­i­da­tion) and on­go­ing main­te­nance.

This reg­u­la­to­ry mod­el would be an ex­pan­sion of the cur­rent pre-cer­ti­fi­ca­tion pi­lot for dig­i­tal health, Got­tlieb said.

In ad­di­tion, the agency aims to cre­ate a “Cen­ter of Ex­cel­lence on Dig­i­tal Health” to es­tab­lish the reg­u­la­to­ry par­a­digm, build new ca­pac­i­ty to eval­u­ate and rec­og­nize third-par­ty cer­ti­fiers, and sup­port a cy­ber­se­cu­ri­ty unit to com­ple­ment the ad­vances in soft­ware-based de­vices.

“To mod­ern­ize and ex­pand its ex­ist­ing ca­pa­bil­i­ties to en­sure the cy­ber­se­cu­ri­ty of med­ical de­vices, the FDA would en­hance its abil­i­ty to co­or­di­nate de­vice-spe­cif­ic re­spons­es to cy­ber­se­cu­ri­ty vul­ner­a­bil­i­ties and in­ci­dents,” he added.

Val­i­dat­ing New Drug De­vel­op­ment

As part of ef­forts to ad­vance the sci­ence of drug de­vel­op­ment, Got­tlieb said the FDA would “build a knowl­edge man­age­ment sys­tem and por­tal to ex­ist­ing and de­vel­op­ing in­for­ma­tion on drug de­vel­op­ment and pre­vi­ous reg­u­la­to­ry de­ci­sions.”

This con­tent man­age­ment plat­form will en­able the FDA to build on evolv­ing in­for­ma­tion and de­ci­sions and iden­ti­fy gaps in reg­u­la­to­ry poli­cies and path­ways en­abling rapid, con­sis­tent re­spons­es to reg­u­la­to­ry ques­tions and pre­vent­ing de­lays in re­sponse to in­no­va­tions in drug de­vel­op­ment.

“As part of this plat­form, the agency al­so would ex­pand its ca­pa­bil­i­ty to quick­ly eval­u­ate new reg­u­la­to­ry ques­tions, us­ing lab­o­ra­to­ry re­search or oth­er ap­pro­pri­ate meth­ods. With the ad­di­tion­al fund­ing, the On­col­o­gy Cen­ter of Ex­cel­lence al­so would stand up a new mod­el for team-based prod­uct re­view that fos­ters col­lab­o­ra­tion across our med­ical prod­uct cen­ters, im­proves re­view ef­fi­cien­cy and ex­pe­dites the de­vel­op­ment of nov­el sci­ence that can im­prove the lives of pa­tients with can­cer,” he said.

Med­ical Prod­ucts Tar­get­ed to Rare Dis­eases

Got­tlieb said the FDA al­so is look­ing to de­vel­op clin­i­cal tri­al net­works to cre­ate an un­der­stand­ing of the nat­ur­al his­to­ry and clin­i­cal out­comes of rare dis­eases.

“The ini­tial fo­cus would be on rare and ul­tra-rare dis­eases, where prod­uct de­vel­op­ment can be chal­leng­ing be­cause of the dif­fi­cul­ty of re­cruit­ing clin­i­cal tri­als,” he said. “FDA has al­ready in­vest­ed, on oc­ca­sion, in the de­vel­op­ment of nat­ur­al his­to­ry mod­els for a small sub­set of rare dis­eases and has ex­per­tise in this area. The FDA would stim­u­late med­ical prod­uct de­vel­op­ment for rare dis­eases by ex­pand­ing and en­hanc­ing the un­der­stand­ing of rare dis­eases and the re­search and drug de­vel­op­ment process­es in this space.”

Mod­ern­ize Gener­ic Drug De­vel­op­ment

On the gener­ic drug front, mean­while, the FDA will cre­ate a new re­view plat­form that would mod­ern­ize gener­ic drug re­view “from a text-based to a da­ta-based as­sess­ment with struc­tured sub­mis­sions and FDA as­sess­ments,” Got­tlieb said.

“This more au­to­mat­ed sys­tem would help to sup­port time­ly de­vel­op­ment and re­view of gener­ic drugs by im­prov­ing clar­i­ty for gener­ic spon­sors, mak­ing ini­tial re­views more ef­fi­cient and de­creas­ing the risk of refuse-to-file let­ters, in­creas­ing the rate of first-cy­cle ap­provals and great­ly in­creas­ing over­all ef­fi­cien­cy.

“This in­vest­ment would al­so sup­port ef­forts to up­date gener­ic drug la­bel­ing, with an ini­tial fo­cus on on­col­o­gy prod­ucts, as part of the agency’s ef­forts to en­sure that pa­tients and their providers have ac­cess to up-to-date in­for­ma­tion to in­form clin­i­cal de­ci­sions,” he added.

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.


Zachary Brennan

managing editor, RAPS

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