Got­tlieb tack­les spec­u­la­tors, FDA trans­paren­cy and the R&D gold stan­dard in a last round of queries ahead of con­fir­ma­tion vote

Field­ing a fi­nal bar­rage of writ­ten ques­tions from a group of skep­ti­cal De­mo­c­ra­t­ic US Sen­a­tors ahead of to­day’s com­mit­tee vote on Scott Got­tlieb’s nom­i­na­tion as head of the FDA, the na­tion’s like­ly next Com­mis­sion­er of Food and Drugs vowed to square off against fi­nan­cial spec­u­la­tors who are gam­ing the sys­tem to charge high prices, reaf­firmed his un­wa­ver­ing sup­port for the coun­try’s gold stan­dard on drug de­vel­op­ment and re­views and called for pub­lish­ing most of what the agency’s CRLs say about reg­u­la­tors’ rea­sons for re­ject­ing a mar­ket­ing ap­pli­ca­tion.

This in­ter­change be­tween the prospec­tive head of the FDA and law­mak­ers helps il­lus­trate key points Got­tlieb is like­ly to raise in the com­ing years with the world’s drug de­vel­op­ers. And they mark a clear line in the sand that il­lus­trates where the agency is like­ly to be more — and less — ac­com­mo­dat­ing for com­pa­nies charged with han­dling the world’s drug pipeline.

De­mo­c­ra­t­ic Sen­a­tors Robert Casey (D-PA) and Al Franken (D-MI) both pressed him on drug pric­ing and the role he could play at the FDA in bring­ing costs down. Franken in par­tic­u­lar cit­ed hun­dreds of gener­ic drugs with no cur­rent com­pe­ti­tion, leav­ing them wide open to play­ers like Mar­tin Shkre­li, the for­mer CEO at Tur­ing, who hiked the price of gener­ic Dara­prim by more than 5000%, trig­ger­ing a lin­ger­ing con­tro­ver­sy.

Here is Got­tlieb’s an­swer, which he re­peat­ed in oth­er re­spons­es to the ar­ray of ques­tions he faced (em­pha­sis added):

While drug pric­ing does not fall di­rect­ly with­in FDA’s purview, I be­lieve the agency can play an im­por­tant role on this im­por­tant is­sue by tak­ing steps to im­prove prod­uct com­pe­ti­tion. If con­firmed, I will work to en­sure FDA has the ap­pro­pri­ate poli­cies and process­es in place to ef­fec­tive­ly fa­cil­i­tate gener­ic mar­ket en­try and com­pe­ti­tion, es­pe­cial­ly for com­plex drugs that some­times don’t face ef­fec­tive gener­ic com­pe­ti­tion even long af­ter patent ex­piries. Re­form­ing the reg­u­la­to­ry path­way for com­plex gener­ic prod­ucts would ad­dress one key pol­i­cy de­fi­cien­cy that re­sults in un­nec­es­sary bar­ri­ers to the de­vel­op­ment and re­view of gener­ic com­peti­tors for some in­no­va­tor prod­ucts for which tra­di­tion­al bioe­quiv­a­lence and bioavail­abil­i­ty test­ing alone are some­times in­suf­fi­cient for prov­ing same­ness. FDA should al­so ex­plore op­tions to im­prove the ef­fi­cien­cy and con­sis­ten­cy of AN­DA re­view process­es and time­lines, so that fi­nan­cial spec­u­la­tors can­not en­gage in a reg­u­la­to­ry ar­bi­trage, by dra­mat­i­cal­ly hik­ing the price of some very old gener­ic drugs be­cause they know it can take years for new gener­ic com­peti­tors to en­ter the mar­ket.

Sen­a­tor Shel­don White­house (D-RI) not­ed that there have been oc­ca­sions when gener­ic man­u­fac­tur­ers protest­ed that brand­ed drug man­u­fac­tur­ers were rais­ing hur­dles on ac­cess­ing drugs to de­lay cheap knock­offs.

Part of Got­tlieb’s re­sponse:

If man­u­fac­tur­ers in­ap­pro­pri­ate­ly refuse to pro­vide their prod­uct to prospec­tive gener­ic com­peti­tors, this would be a con­cern to FDA and be­come a mat­ter for po­ten­tial en­force­ment ac­tion by the Fed­er­al Trade Com­mis­sion.

Al­so, he added, re­form­ing the path­way on com­plex gener­ics could al­so help ad­dress this.

Tam­my Bald­win (D-WI) en­cour­aged Got­tlieb to get be­hind new rules to use pa­tient-re­port­ed out­comes mea­sures in the re­view process, to “cap­ture da­ta be­yond just dis­ease symp­toms and phys­i­cal func­tion­ing to in­clude psy­choso­cial health mea­sures, in­clud­ing dis­tress screen­ing (e.g., con­cerns re­lat­ed to dis­rup­tion of work/fam­i­ly life [due to the reg­i­men], con­cerns re­lat­ed to nu­tri­tion, fi­nan­cial im­pact and oth­ers). This would pro­vide mean­ing­ful pa­tient feed­back about is­sues that may not be iden­ti­fied through the cur­rent mea­sures be­ing used in the clin­i­cal tri­al process.”

Got­tlieb:

I strong­ly agree that pa­tient ex­pe­ri­ences, pref­er­ences, and per­spec­tives should play an im­por­tant and ap­pro­pri­ate role in FDA’s reg­u­la­to­ry pol­i­cy-mak­ing and de­ci­sion-mak­ing. Among oth­er ap­proach­es, I have ad­vo­cat­ed that we con­tin­ue to ad­vance well-val­i­dat­ed, sci­en­tif­ic tools for in­cor­po­rat­ing Pa­tient Re­port­ed Out­comes (PROs) as end­points in clin­i­cal tri­als. I sup­port these and oth­er mea­sures that would al­low mean­ing­ful pa­tient feed­back to be in­cor­po­rat­ed in­to reg­u­la­to­ry de­ci­sion mak­ing to bet­ter de­fine pa­tient-ex­pe­ri­ence da­ta that may not be iden­ti­fied through the cur­rent mea­sures be­ing used in the clin­i­cal tri­al process.

Sen­a­tor Christo­pher Mur­phy (D-CT) raised Got­tlieb’s stand on trans­paren­cy, an is­sue that comes up rou­tine­ly for an agency barred by law from dis­cussing de­tails on drug da­ta — and much more.

I am a strong pro­po­nent of da­ta trans­paren­cy—for pa­tients, physi­cians, and man­u­fac­tur­ers. I have long ad­vo­cat­ed that the FDA re­lease more in­for­ma­tion re­lat­ed to its own re­view process that could be used to bet­ter in­form con­sumers and prod­uct de­vel­op­ers alike. If con­firmed, I will be com­mit­ted to work­ing with Con­gress, pa­tients, in­dus­try, and stake­hold­ers on the is­sue of da­ta trans­paren­cy and new ways that FDA could po­ten­tial­ly make im­por­tant of its own in­for­ma­tion and de­lib­er­a­tions more read­i­ly avail­able to the pub­lic.

And in an­oth­er an­swer to one of Sen­a­tor Eliz­a­beth War­ren’s (D-MA) ques­tions, he added an im­por­tant point on his com­mit­ment to open­ing up more.

This in­cludes the com­plete re­sponse let­ters, af­ter prop­er redac­tion of com­mer­cial con­fi­den­tial in­for­ma­tion.

Through it all, Got­tlieb re­peat­ed­ly com­mit­ted to main­tain­ing the agency’s stan­dards on prov­ing a drug’s ef­fi­ca­cy and safe­ty ahead of an ap­proval. And he stuck to that as Sen­a­tor Pat­ty Mur­ray (D-WA) ques­tioned where he would draw the line and new drug ap­provals, cit­ing some crit­i­cism from Got­tlieb five years ago.

FDA has made sig­nif­i­cant progress in re­cent years to en­sure that pa­tients in the U.S. have ac­cess to new, in­no­v­a­tive ther­a­pies thanks to new leg­isla­tive path­ways like Break­through Ther­a­py des­ig­na­tion. The adop­tion of the Break­through path­way ad­dressed many of the con­cerns I raised in that 2012 ar­ti­cle. I be­lieve we should con­tin­ue to look for ways to im­prove the ef­fi­cien­cy of FDA’s med­ical prod­uct re­view pro­gram, mod­ern­ize the sci­en­tif­ic stan­dards used in drug reg­u­la­tion, and seek more uni­form adop­tion of path­ways cre­at­ed by Con­gress like the Break­through Des­ig­na­tion, and build on these op­por­tu­ni­ties through adop­tion of the new pro­vi­sions in Con­gress in 21st Cen­tu­ry Cures. We need to do all these things while con­tin­u­ing to en­sure that new prod­ucts meet FDA’s Gold Stan­dard for safe­ty and ef­fi­ca­cy.

In an­oth­er re­sponse, he not­ed:

Main­tain­ing the Gold Stan­dard of safe­ty and ef­fi­ca­cy for med­ical prod­ucts is fun­da­men­tal to FDA’s mis­sion to pro­tect and pro­mote pub­lic health.

Got­tlieb al­so re­peat­ed that his of­ten cit­ed busi­ness ties to com­pa­nies like Glax­o­SmithK­line, which had paid him $60,000 a year plus trav­el ex­pens­es for his ad­vice, Ver­tex, Cel­gene and oth­ers were al­ready or about to be sev­ered, vow­ing to seek coun­sel from ethics ad­vis­ers af­ter re­cus­ing him­self from any de­ci­sions about his for­mer clients and busi­ness in­vest­ments.

Got­tlieb is dig­ging in for the du­ra­tion.


Im­age: Com­mis­sion­er-des­ig­nate Scott Got­tlieb at his nom­i­na­tion hear­ing in ear­ly April Zach Gib­son/Get­ty Im­ages

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Merck Research Laboratories CMO Roy Baynes

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