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.”


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

RWE chal­lenges for to­day's bio­phar­ma

The rapid development of technology — and the resulting avalanche of data — are catalysts for significant change in the biopharmaceutical industry. This translates into urgent pressures for today’s biopharma, including a need to quickly and affordably develop products with proven therapeutic efficacy and value. This urgency is expedited by the growth of value-based contracting, where access to reimbursement and profit depends on these abilities.

UP­DAT­ED: In a stun­ning turn­around, Bio­gen says that ad­u­canum­ab does work for Alzheimer's — but da­ta min­ing in­cites con­tro­ver­sy and ques­tions

Biogen has confounded the biotech world one more time.

In a stunning about-face, the company and its partners at Eisai say that a new analysis of a larger dataset on aducanumab has restored its faith in the drug as a game-changer for Alzheimer’s and, after talking it over with the FDA, they’ll now be filing for an approval of a drug that had been given up for dead.

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As shares suf­fer from a lin­ger­ing slump, a bruised Alk­er­mes slash­es 160 jobs in R&D re­struc­tur­ing

With its share price in a deep slump after suffering through a regulatory debacle over their depression drug ALKS 5461, Alkermes CEO Richard Pops is taking the ax to its R&D organization in a restructuring aimed at cutting costs ahead of its next attempt at a rollout in a tough field.

Richard Pops, Endpoints via Youtube

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Acor­da's Ron Co­hen brings the ax back out as new drug sales on­ly trick­le in while cash cow is led to the slaugh­ter

With its new drug earning meager sums and its one-time cash cow reduced to a bony shadow of its former self, Acorda Therapeutics today is rolling out a new restructuring aimed at slashing the staff and cutting costs to get through the hard times ahead.

The biotech is chopping a quarter of its staff today, carving back R&D as well as SG&A expenses. And CEO Ron Cohen is cutting deep.

Under the new austerity budget, Acorda’s R&D expenses for the full year 2019 are expected to be $55 – $60 million, reduced from $70 – $80 million. SG&A expenses for the full year 2019 are expected to be $185 – $190 million, reduced from $200 – $210 million. R&D expenses for the full year 2020 are expected to be $20 – $25 million and SG&A
expenses for the full year 2020 are expected to be $160 – $165 million.

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RAPT Ther­a­peu­tics re­turns to Wall Street to re­vive IPO bid

On May 24, FLX Bio, a small cancer and inflammation biotech with backing from GV, changed its name to RAPT Therapeutics and filed confidentially for an IPO. On July 5th, they filed to raise up to $86 million. On July 22, they announced the IPO with a $75 million goal.  And on August 1, they abruptly and without explanation called it all off.

Now, without explanation, they’re reviving the bid, filing again for a $75 million IPO, this time with a new bookrunner and a new drug candidate in the clinic. The terms will be the same: 5 million shares at $14-$16 per share. It would give them a diluted market value of $351 million.

EY vet set to re­place re­tir­ing Am­gen CFO Meline

Ahead of its third-quarter results next week, Amgen on Tuesday disclosed the planned retirement of David Meline, who has served as the company’s chief financial officer since 2014.

Meline will be replaced by Ernst & Young vet, Peter Griffith, as CFO come January 1, 2020 — but until then Griffith will serve as executive vice president, finance.

“Over the last 5 years at Amgen, Meline instituted many major changes that led to operational efficiencies and margin expansion while successfully returning cash to shareholders. Now that Amgen is on solid footing, it was a good time to step away,” Cowen’s Yaron Werber wrote in a note. “We do not anticipate any major changes to strategy or operations immediately due to this transition as Amgen is on solid footing.”

Eli Lil­ly’s USA, di­a­betes chief En­rique Con­ter­no is head­ing out af­ter 27 years, and he’s be­ing re­placed by a com­pa­ny in­sid­er

Close to 3 years after Eli Lilly CEO Dave Ricks added the title of president of the US operations to Enrique Conterno’s resume, which included his helmsmanship of the diabetes franchise, the Peruvian born exec is set to retire after a 27-year run at the pharma giant.

Lilly put out the news just as it was posting Q3 results, with a mix of upbeat and downbeat results in the latest set of numbers from Lilly.
Conterno — a grizzled, deeply experienced and sometimes gruff veteran of the pharma world — was a high-profile figure at Lilly, stepping up to expanded duties as the company was forced to deal with intense pricing pressure on the diabetes side of the business. He had replaced outgoing US president Alex Azar, who later popped up as head of Health and Human Services in the Trump administration.
As head of the diabetes unit, Conterno had to deal with an extraordinarily competitive field as payers demanded bigger discounts. Trulicity’s success helped generate new revenue for the company, but Q3’s miss on revenue had a lot to do with the need for discounting the drug ahead of Novo Nordisk’s rival therapy, Rybelsus, which was priced on the wholesale level at an almost identical rate.

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No­var­tis hands off $80M in cash to part­ner up with a top biotech play­er in the fi­bro­sis sec­tor

Never underestimate the power of a good showing at a scientific conference.
In a presentation late last year, the researchers at Pliant Therapeutics launched a series of discussions about the preclinical data they were pulling together around their work on their small-molecule integrin inhibitor aimed at transforming growth factor beta, or TGF-β, a key pathway involved in fibrosis.
And they got some serious attention for the work.
“We got interest from pharma partners and at the end Novartis basically made it,” says Pliant CEO Bernard Coulie.

Vas Narasimhan. Getty Images

UP­DAT­ED: Failed PhI­II fe­vip­iprant tri­als pour more cold wa­ter on No­var­tis' block­buster R&D en­gine — and briefly spread the chill to a high-pro­file biotech

Back in July, during an investor call where Novartis execs ran through an upbeat assessment of their Q2 performance, CEO Vas Narasimhan and development chief John Tsai were pressed to predict which of the two looming Phase III readouts — involving cardio drug Entresto and asthma therapy fevipiprant, respectively — had a higher likelihood of success. Tsai gave the PARAGON-HF study with Entresto minimally better odds, but Narasimhan emphasized that their strategy of giving fevipiprant to more severe patients gave them confidence.

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