Scott Got­tlieb aced his con­fir­ma­tion hear­ing, vow­ing to main­tain R&D gold stan­dard

Scott Got­tlieb – April 5, 2017 Zach Gib­son/Get­ty Im­ages


Sen­ate De­moc­rats didn’t waste any time in rais­ing their ob­jec­tions to Scott Got­tlieb as the next FDA com­mis­sion­er.

Sen­a­tor Pat­ty Mur­ray (D-Wash­ing­ton) start­ed her re­marks at Got­tlieb’s con­fir­ma­tion hear­ing this morn­ing voic­ing her con­cerns about “whether you can with­stand po­lit­i­cal pres­sure” in main­tain­ing an un­bi­ased, sci­ence-based ap­proach in main­tain­ing the gold stan­dard on safe­ty and ef­fi­ca­cy re­views of new drugs.

Mur­ray com­plained that sen­a­tors had on­ly days to re­view Got­tlieb’s “un­prece­dent­ed fi­nan­cial en­tan­gle­ments” in the phar­ma in­dus­try — with dozens of drugs from the com­pa­nies he’s worked with head­ed to the FDA — as well as more than 800 list­ed pub­li­ca­tions which “raise con­cerns about po­ten­tial con­flicts of in­ter­est.”

Scott Got­tlieb this morn­ing on Capi­tol Hill at his nom­i­na­tion hear­ing. (CRED­IT: C-SPAN/End­points News)

Got­tlieb, though, came pre­pared to re­but those sug­ges­tions, mak­ing promis­es to main­tain an in­de­pen­dent FDA com­mit­ted to find­ing new ef­fi­cien­cies at the agency while mak­ing sure that all the new prod­ucts the agency ap­proves are safe and ef­fec­tive, meet­ing the agency’s long-term gold stan­dard on R&D.

“I’ve seen the im­por­tance of the FDA’s work as both a doc­tor and a pa­tient,” Got­tlieb, a can­cer sur­vivor, said in his open­ing state­ment. He added that his in­vest­ments and en­deav­ors were aimed at im­prov­ing health­care in the US. Some have worked, oth­ers didn’t and many oth­ers are still in de­vel­op­ment.

Re­gard­less of those ties, he vowed to be an “im­par­tial and in­de­pen­dent ad­vo­cate for the pub­lic health” main­tain­ing a rig­or­ous ap­proach to re­view­ing drugs. “We’re at an in­flec­tion point in bio­med­ical sci­ence,” he added, and the chal­lenge at the FDA will be get­ting the most bang for its reg­u­la­to­ry bucks.

“We should re­ject the false di­choto­my that it all boils down to a choice be­tween speed and safe­ty.” Bet­ter ef­fi­cien­cy and safe­ty are both pos­si­ble, he said, while main­tain­ing the gold stan­dard. And he added that “it’s in­cum­bent on us to have a world class work force,” hir­ing the best for reg­u­la­to­ry work, which Sen­a­tor Lamar Alexan­der not­ed was Got­tlieb’s pick as his most im­por­tant chal­lenge.

Got­tlieb al­so helped soothe any in­dus­try con­cerns that he might want to come in as an abra­sive re­former, work­ing at odds with staff.

“I think every­thing is a bot­tom-up ap­proach in the agency,” he told the law­mak­ers. “The ideas re­al­ly need to come from the ca­reer staff in the cen­ters.” That’s go­ing to be a chal­lenge as the agency re­cruits re­place­ments for a line­up of se­nior agency of­fi­cials who are due for re­tire­ment.

Got­tlieb promised to bring best prac­tices to those ar­eas in the FDA that have been lag­ging. There’s been an “un­even adop­tion” of the break­through drug pro­gram, he added, which now re­quires broad adop­tion.  The prospec­tive com­mis­sion­er al­so said that while he was not crit­i­cal of Phase III, there is a pos­si­bil­i­ty with new tech­nol­o­gy to com­press Phase II and Phase III in­to an adap­tive de­sign.

And, as ex­pect­ed, he rel­ished the prospect of tack­ling a slew of com­plex gener­ics await­ing an ap­proval at the FDA. Many drugs are sold at high prices, he said, but should be sub­ject to com­pe­ti­tion.

A few days ago Got­tlieb care­ful­ly de­lin­eat­ed ties to some two dozen bio­phar­ma com­pa­nies he’s been work­ing with over the past eight years of the Oba­ma ad­min­is­tra­tion. He’s worked with Glax­o­SmithK­line, Ver­tex and Bris­tol-My­ers, so you can check the box on in­dus­try con­flicts of in­ter­est that De­moc­rats will con­demn.

Among Got­tlieb’s oth­er lines of work, the for­mer deputy FDA com­mis­sion­er penned a se­ries of ar­ti­cles on bio­phar­ma for Forbes, and the Dems have been giv­ing them a close look. Two pieces float­ed to the top of the read­ing list. One cen­tered on his pro­pos­al to move re­spon­si­bil­i­ty for iden­ti­fy­ing the opi­oid sup­pli­ers who were break­ing the law from the DEA to HHS. The oth­er dealt with his crit­i­cism of the REMS pro­grams, which set up post-mar­ket­ing safe­ty pro­grams to lim­it risks seen in cer­tain drugs.

Add it all up, says Sher­rod Brown (D-Ohio), and you find some­one who will “roll over for his Big Phar­ma friends,” as The Hill re­ports.

Re­pub­li­cans, though, were large­ly con­tent to lim­it their com­ments to praise for the nom­i­nee and his three young daugh­ters, who were seat­ed be­hind the can­di­date. And it’s the Re­pub­li­cans who have the votes to make this hap­pen.

The in­dus­try, mean­while, has made it clear that af­ter hear­ing some of the ideas backed by oth­er com­mis­sion­er can­di­dates like Jim O’Neill – who’s sug­gest­ed ap­prov­ing drugs as soon as they’re de­ter­mined safe — Got­tlieb is the best can­di­date by far. In a snap poll of qual­i­fied End­points News sub­scribers, 87% of the 580 who reg­is­tered an opin­ion backed Got­tlieb’s nom­i­na­tion.

So you can ex­pect most peo­ple in bio­phar­ma to keep their fin­gers crossed for Got­tlieb.

Bot­tom line: Got­tlieb came well pre­pared for every ques­tion, care­ful not to step be­yond the bound­aries of the com­mis­sion­er’s job when in­vit­ed to com­ment on Oba­macare. His an­swers were pol­ished and prac­ticed, leav­ing lit­tle room for un­ex­pect­ed at­tacks. And he de­flect­ed crit­i­cism eas­i­ly, promis­ing to avoid any po­ten­tial con­flicts of in­ter­est.

Bar­ring a last minute sur­prise, Got­tlieb’s con­fir­ma­tion as the next FDA com­mis­sion­er is all but guar­an­teed.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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UP­DAT­ED: Stay tuned: Bio­gen’s num­bers are great — it’s their wor­ri­some fu­ture that leaves an­a­lysts skit­tish

Biogen came out with an upbeat assessment of their Q2 numbers today, discounting the arrival of a key rival for its blockbuster Spinraza franchise. But the top execs remain grimly determined to not say much anything new about the sore points that have dragged down its stock, including the future of its big investment in Alzheimer’s or how it plans to invest the considerable cash that the big biotech continues to reap.

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Why wait? Cel­gene re­struc­tures a big Jounce pact — ze­ro­ing in on new I/O path­way with $530M deal and bump­ing ICOS

Celgene’s business team isn’t waiting for the big merger with Bristol-Myers Squibb to go through before syncing its strategy with the new mother ship.

Tuesday evening the big biotech unveiled a $530 million deal — $50 million in upfront cash — to amend their alliance with Jounce Therapeutics $JNCE to gain worldwide rights to JTX-8064, an antibody that targets the LILRB2 receptor on macrophages. Their old, $2.6 billion deal is being scrapped, leaving Jounce with a pipeline that includes the lead drug, the ICOS-targeting vopratelimab.

PACT Phar­ma says it's per­fect­ed the tech to se­lect neoanti­gens for per­son­al­ized ther­a­py — now on­to the clin­ic

At PACT Pharma, the lofty goal to unleash a “tsunami” of T cells personalized for each patient has hinged on the ability to correctly identify the neoantigens that form something of a fingerprint for each tumor, and extract the small group of T cells primed to attack the cancer. It still has a long way to go testing a treatment in humans, but the biotech says it has nailed that highly technical piece of the process.

UP­DAT­ED: My­ovan­t's uter­ine fi­broid drug looks com­pet­i­tive in PhI­II — but can they van­quish mighty Ab­b­Vie?

Vivek Ramaswamy’s Myovant $MYOV has closely matched its positive first round of Phase III data for their uterine fibroid drug relugolix, setting up a head-to-head rivalry with pharma giant AbbVie as the little biotech steers to the market with a planned filing in Q4.

Here’s how Myovant plans to prevail over the AbbVie $ABBV empire.

In the study, 71.2% of women receiving once-daily relugolix combination therapy achieved the clinical response they were looking for, compared to only 14.7% in the control arm. The data comfortably reflected the same outcomes in the first Phase III — 73.4% of women receiving once-daily oral relugolix combination therapy achieved the responder criteria compared with 18.9% of women receiving placebo — which will reassure regulators that they are getting the carefully randomized data that qualifies for the FDA’s gold standard for success.

Lit­tle Mar­i­nus sees its shares eclipsed as the Sage ri­val fails to com­pare on PPD in PhII

The executive team at Sage $SAGE have skirted another potential pitfall on its way to racking up a big future for its depression drug Zulresso.

Little Marinus Pharmaceuticals $MRNS had sought to challenge the Sage drug with an IV formulation — followed by an oral version — of ganaxolone for postpartum depression. But researchers say their Phase II study failed to positively differentiate itself from a placebo at 28 days — leaving them to hold up “clinically meaningful” data within the first day of administration compared to the control arm.

Roche cuts loose Tam­i­flu OTC rights, hand­ing Sanofi the keys as the phar­ma gi­ant dou­bles down on Xofluza

Roche set out to make a better flu medicine than Tamiflu as that franchise was headed to a generic showdown. Now they’ll see just how well Xofluza stacks up against the mainstay drug after handing off over-the-counter rights in the US to Sanofi.

Sanofi $SNY says it will now step in to negotiate a deal with the FDA to steer Tamiflu into the OTC market, a role that could well involve new studies to ease passage of the drug out of doctor’s hands and into the consumer end of the market. And the French pharma giant will have first dibs over “selected” OTC markets around the world as they push ahead.

Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.