Bio­phar­ma ex­ecs give Scott Got­tlieb’s nom­i­na­tion for FDA com­mish (al­most) a stand­ing O in End­points poll

A big ma­jor­i­ty of bio­phar­ma ex­ec­u­tives over­all are clear­ly en­thu­si­as­tic about Pres­i­dent Trump’s de­ci­sion to nom­i­nate Scott Got­tlieb as the next FDA com­mis­sion­er.

In our snap poll over the week­end Got­tlieb picked up 87% sup­port among the 580 qual­i­fied sub­scribers who vot­ed on the de­ci­sion, with 504 ap­prov­ing the choice against 76 who large­ly hat­ed it.

Keep in mind, so far the Trump ad­min­is­tra­tion has earned a sol­id neg­a­tive rat­ing in the drug de­vel­op­ment busi­ness as ex­ecs in over­whelm­ing­ly De­mo­c­ra­t­ic re­gions reg­is­tered their dis­dain for his trav­el ban, re­ject­ed the no­tion of gut­ting reg­u­la­tions and ex­pressed fears that a new ap­proach on H-1B visas could make it much hard­er to re­cruit top tal­ent from be­yond US bor­ders. Got­tlieb, though, looks like a breath of fresh air in an in­dus­try that’s been feel­ing bad­ly choked in Wash­ing­ton, DC for the past two months.

“Ab­solute­ly!” cheered Al­ny­lam CEO John Maraganore when he was asked whether he ap­proved of the nom­i­na­tion. “First good thing Trump has done.”

The full spec­trum of pro-Got­tlieb sen­ti­ment ran the gamut from what could be de­scribed as the best of a bad lot to a big thumb’s up for a physi­cian who has ex­pe­ri­ence at the FDA and a deep un­der­stand­ing of the de­vel­op­ment and re­view process, which he will now push in­to high gear.

You could prac­ti­cal­ly hear the sigh of re­lief that fol­lowed deep-seat­ed fears that Trump might choose a Lib­er­tar­i­an like Jim O’Neill, who has sound­ed ready to drop the bar on new ap­provals about as low as it can get.

“I think it is a re­lief that Got­tlieb is ex­pe­ri­enced in reg­u­la­to­ry mat­ters and at the FDA, un­der­stands that a strong FDA is the best way to get med­i­cines to pa­tients and al­so sup­ports us­ing the best new sci­ence to im­prove the FDA’s ap­proval process,” said Basil Dahiy­at, the CEO of an­ti­body en­gi­neer­ing com­pa­ny Xen­cor. “Ra­tio­nal and rig­or­ous physi­cians and doc­tors who want to try new ideas are what in­dus­try needs at the FDA. Ap­point­ing some­body who wants to make the FDA bet­ter, rather than im­pose to­tal­ly un­found­ed ide­ol­o­gy to gut good reg­u­la­tion like some of the oth­er can­di­dates float­ed, is promis­ing.  Strange days in­deed that the al­ter­na­tive was even re­al­ly con­sid­ered.”

“Un­der­stands agency and in­dus­try needs,” not­ed one re­sponse. “The one ra­tio­nal choice amongst the can­di­dates men­tioned.”

A few oth­er com­ments from the pro-Got­tlieb side:

“He seems like a rea­son­able can­di­date, will­ing to lis­ten, and I see his ties to in­dus­try as a ben­e­fit, in terms of his un­der­stand­ing of the busi­ness.”

“He’s ex­pe­ri­enced and will bring a thought­ful ap­proach to the com­plex chal­lenges of run­ning FDA. This is not a job for a light­weight.”

Steve Holtz­man, the CEO of Deci­bel Ther­a­peu­tics, put it this way:

“While I am in many, many ways ide­o­log­i­cal­ly at the oth­er end of the spec­trum from Scott, I sup­port his ap­point­ment. He be­lieves in sci­ence-based reg­u­la­tion–ad­dress­ing BOTH ef­fi­ca­cy and safe­ty–and em­braces sci­en­tif­ic ad­vances that can, with good jus­ti­fi­ca­tion and with­out im­per­il­ing pa­tients, make clin­i­cal tri­als more ef­fi­cient. I al­so think that con­cerns about con­flict of in­ter­est, be­cause of his work with the bio­phar­ma­ceu­ti­cal in­dus­try, are ill-found­ed. The blan­ket ap­pli­ca­tion of this type of log­ic leads to calls to ex­clude from FDA Ad­vi­so­ry Pan­els those most qual­i­fied be­cause those same in­di­vid­u­als, for the very same rea­son of their ex­per­tise, are sought by in­dus­try as ad­vi­sors. –While no doubt true that many will sell their souls and rep­u­ta­tions for a buck, it is al­so the case that such an ac­cu­sa­tion, made against a spe­cif­ic in­di­vid­ual with­out any ev­i­dence, can tell us more about the ac­cuser than the ac­cused.”

An­oth­er sim­ply said: “Pro-in­dus­try” in giv­ing a thumb’s up.

But you could find the ex­act same re­mark re­peat­ed among the 13% of our sub­scribers who end­ed up shak­ing their heads over the nom­i­na­tion.

“Too wed to in­dus­try,” not­ed one neg­a­tive re­sponse to Got­tlieb.

That in­dus­try ex­pe­ri­ence that Got­tlieb has that so many found re­as­sur­ing looked like a se­ri­ous con­flict of in­ter­est to the ‘no’ vot­ers. And be­sides, in some eyes Trump – who clear­ly alarmed the in­dus­try with vows to dereg­u­late drug de­vel­op­ment — can’t do any­thing right.

“Too close with the in­dus­try & wants to re­duce or elim­i­nate the reg­u­la­tions that have worked to keep the US safe.”

“His ar­dor for dereg­u­la­tion could be harm­ful to con­sumers and ul­ti­mate­ly the in­dus­try. Pro­pos­al to cen­tral­ize re­view de­ci­sions could re­sult in fur­ther politi­ciz­ing ap­proval de­ci­sions.”

“Un­qual­i­fied,” said an­oth­er.

”Will in­sti­tute dan­ger­ous pol­i­cy and be be­hold­en to Trump.”

Got­tlieb hasn’t been shy about mak­ing con­crete sug­ges­tions for a new and im­proved FDA. As I’ve writ­ten, he’s backed a more fo­cused ap­proach on rare dis­ease drugs, pub­lish­ing com­plete re­sponse let­ters of re­jec­tion and quick­ly mov­ing com­plex gener­ics to the mar­ket, among oth­er things.

Now he has to start de­liv­er­ing on those promis­es — or run the risk of se­vere­ly dis­ap­point­ing an in­dus­try whose clear sup­port may have well been the de­cid­ing fac­tor be­hind his nom­i­na­tion.

 

Here’s just a few more com­ments from sub­scribers …

 

Yes

Ac­cel­er­a­tion and sim­pli­fi­ca­tion of the process and re­quire­ments for ap­proval of biosim­i­lars is des­per­ate­ly need­ed. Pub­li­ca­tion of CR let­ters is over­due. In­creased use of sur­ro­gate end­points is a po­ten­tial con­cern de­pend­ing on how low the bar would be set. Sur­ro­gate end­points should be agreed up­on pri­or to ini­ti­a­tion of stud­ies.


While I am in ide­o­log­i­cal­ly at the oth­er end of the spec­trum from Scott, I sup­port his ap­point­ment. He be­lieves in sci­ence-based reg­u­la­tion–ad­dress­ing BOTH ef­fi­ca­cy and safe­ty–and em­braces sci­en­tif­ic ad­vances that can, with good jus­ti­fi­ca­tion and with­out im­per­il­ing pa­tients, make clin­i­cal tri­als more ef­fi­cient. I al­so think that con­cerns about con­flict of in­ter­est, be­cause of his work with the bio­phar­ma­ceu­ti­cal in­dus­try, are ill-found­ed. The blan­ket ap­pli­ca­tion of this type of log­ic leads to calls to ex­clude from FDA Ad­vi­so­ry Pan­els those most qual­i­fied be­cause those same in­di­vid­u­als, for the very same rea­son of their ex­per­tise, are sought by in­dus­try as ad­vi­sors. While no doubt true that many will sell their souls and rep­u­ta­tions for a buck, it is al­so the case that such an ac­cu­sa­tion, made against a spe­cif­ic in­di­vid­ual with­out any ev­i­dence, can tell us more about the ac­cuser than the ac­cused.


Ex­cel­lent bal­ance of med­ical ex­per­tise, pri­or ex­pe­ri­ence and po­lit­i­cal deft­ness. Scott will force great change in cul­ture, or­ga­ni­za­tion, phi­los­o­phy and lead­er­ship at FDA. He will bring an es­pe­cial­ly pa­tient-cen­tric fo­cus to the FDA. We are par­tic­u­lar­ly ex­cit­ed about his be­lief in the need for a sep­a­rate re­view di­vi­sion for rare dis­eases at FDA. Great day for sci­ence, med­i­cine and for pa­tients. Not so great a day for FDAers who yearn for rigid, in­flex­i­ble and non-21st Cen­tu­ry drug de­vel­op­ment.


Doesn’t want to dis­man­tle agency he’s lead­ing.


He has the nec­es­sary ex­pe­ri­ence and deep un­der­stand­ing of both the agency and the in­dus­try. The changes he has been ad­vo­cat­ing in his pub­lished writ­ings have broad sup­port and were in­deed the type of bu­reau­crat­ic hur­dles that need­ed to be sim­pli­fied for the greater pub­lic good.


Calm per­sona. Reg­u­la­to­ry and med­ical back­ground. Has shown an un­der­stand­ing of da­ta.


Amer­i­cans don’t re­al­ize it but the pho­bia that the FDA and NIH staff have about in­dus­try in­puts and co­op­er­a­tions hurts the process of mov­ing re­search ad­vances to med­i­cines. For ex­am­ple, drug dis­cov­ery and clin­i­cal tri­als bio­mark­ers re­quires much bet­ter in­dus­try-NIH/FDA co­op­er­a­tions. (Cur­rent­ly, these are han­dled by the FNIH, con­sor­tia, PCORI and the IMI). Leg­is­la­tion that would al­low more pool­ing of NIH/FDA/in­dus­try funds is re­quired. A new Bio­mark­er Con­sor­tium that can move quick­ly would be good for med­i­cine, the pub­lic, and in­dus­try.

No

He be­lieves in de-em­pha­siz­ing safe­ty in fa­vor of med­ical in­no­va­tion. Both are im­por­tant, but with re­cent safe­ty set­backs as in the case of Juno, I fear that the safe­ty bar may be set a lit­tle low.


On a mat­ter of prin­ci­ple, it is al­ways bet­ter to have a watch­dog with as much neu­tral­i­ty and sep­a­ra­tion as pos­si­ble. You don’t want your watch­dog to be your best friend. They’re there to push back and bring the best out of you. Giv­en the choic­es we had, thank good­ness Scott was se­lect­ed, but that shouldn’t dis­tract us from the fact that when our friends be­come our over­sight, we erode the over­sight that does the in­sti­tu­tion jus­tice. The last­ing costs of non-neu­tral in­sid­er checks has been seen time and again through his­to­ry and across for­eign gov­ern­ments where a re­volv­ing door feeds cor­rup­tion and self-serv­ing bu­reau­cra­cies. Scott’s a great guy. He fun­da­men­tal­ly cares about pa­tients. He loves the in­dus­try and the FDA with pas­sion. But he should hum­ble him­self to his bias and re­cuse him­self. On­ly thir­ty years ago, the in­dus­try was clam­or­ing about ba­sic FDA rules and now the in­dus­try re­al­izes its im­por­tance. Thir­ty years from now, hope­ful­ly it’ll be fur­ther clear how im­por­tant it is to make sure what­ev­er checks come, we don’t write them our­selves.


Bet­ter than the al­ter­na­tives but still too dereg­u­la­to­ry.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

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