WATCH: Scott Got­tlieb vows to shake up the FDA, back­ing a trend to­ward faster drug de­vel­op­ment

FDA com­mis­sion­er Scott Got­tlieb has sound­ed a crys­tal clear warn­ing over the high — and grow­ing — cost of drug de­vel­op­ment. And in a speech to reg­u­la­to­ry ex­ecs on Mon­day, Got­tlieb com­mit­ted the FDA to back­ing up more ef­fi­cient drug de­vel­op­ment pro­grams with new mea­sures to clear the reg­u­la­to­ry path for de­vel­op­ers bar­rel­ing ahead to rel­a­tive­ly swift piv­otal da­ta in search of an ac­cel­er­at­ed OK.

Got­tlieb start­ed by out­lin­ing a bleak pic­ture in drug R&D, not­ing that the eco­nom­ic mod­el for drug de­vel­op­ment is bro­ken. It costs too much to de­vel­op a drug so it can be ap­proved for mar­ket­ing. And costs are swelling fast at the dis­cov­ery end of the busi­ness, which will help swamp a sys­tem that al­ready doesn’t work par­tic­u­lar­ly well.

As he has in the past, Got­tlieb held up some of the rapid-fire clin­i­cal tri­als we’ve been see­ing in the can­cer field as a mod­el for what can work, paving the way to the ac­cel­er­at­ed ap­proval path­way at the FDA. And he be­lieves — though there is pre­cious lit­tle ev­i­dence to back it up — that mov­ing drug de­vel­op­ment in­to the fast lane can re­duce R&D costs and there­by al­low bio­phar­ma com­pa­nies to pass on sav­ings to pa­tients through low­er costs.

To help de­vel­op­ers, Got­tlieb vowed that the FDA, through CDER chief Janet Wood­cock and the Of­fice of New Drugs, will adapt the reg­u­la­to­ry path­way to en­able drug de­vel­op­ment at a more mod­er­ate cost.

Said Got­tlieb:

Com­pa­ra­ble reg­u­la­to­ry mile­stones need to be built in­to the new seam­less clin­i­cal tri­al process. We need to en­sure we pro­vide com­pa­ra­ble in­ter­ac­tions and over­sight.

He al­so not­ed that as de­vel­op­ers move to­ward faster, seam­less stud­ies — drop­ping the tra­di­tion­al Phase I through Phase III de­vel­op­ment plan — reg­u­la­tors al­so need to up­date pa­tients’ aware­ness of the risks in­volved in pro­vid­ing their con­sent for par­tic­i­pat­ing in these stud­ies.


Watch Got­tlieb’s speech and Q&A

Cred­it: RAPS


Here are some ex­cerpts from the speech, start­ing with an out­line of the trend to­ward a sin­gle de­vel­op­ment pro­gram for new drugs.

Ow­ing in part to these lead­er­ship ef­forts, we’ve seen more spon­sors de­vel­op on­col­o­gy drugs that for­go the con­ven­tion­al three se­quen­tial phas­es of drug de­vel­op­ment. They opt in­stead for seam­less ap­proach­es. Un­der these tri­al de­signs, they’ll typ­i­cal­ly add co­horts to a first-in-hu­man tri­al to in­ves­ti­gate dos­es and ac­tiv­i­ty in a va­ri­ety of can­cers.

We’ve seen ex­am­ples where this ap­proach has al­lowed the rapid de­vel­op­ment of drugs in mul­ti­ple dif­fer­ent tu­mor types. If we had to stop and start for­mal Phase II tri­als in each dif­fer­ent or­gan sys­tem where a can­cer arose, it could have been a pro­tract­ed process. This ap­proach is well suit­ed to the kinds of drugs that are be­ing de­vel­oped now, where drugs in­ter­vene on com­mon el­e­ments found across mul­ti­ple kinds of dis­ease states. At FDA, we’ve iden­ti­fied more than 40 ac­tive com­mer­cial in­ves­ti­ga­tion­al new drug ap­pli­ca­tions for large first-in-hu­man on­col­o­gy tri­als alone that use these seam­less strate­gies.

Got­tlieb al­so talked about us­ing broad pro­to­cols that al­low de­vel­op­ers to tack­le mul­ti­ple tar­gets at once.

We’re al­so ad­vanc­ing the use of ‘Mas­ter Pro­to­cols’ to en­able more co­or­di­nat­ed ways to use the same tri­al struc­ture to eval­u­ate treat­ments in more than one sub­type of a dis­ease or type of pa­tient.

This ap­proach is par­tic­u­lar­ly rel­e­vant when it comes to tar­get­ed drugs. These are drugs that may in­ter­vene on mark­ers that are rel­e­vant across many dif­fer­ent dis­ease sub­types. We may, for ex­am­ple, want to eval­u­ate these dif­fer­ent tar­gets si­mul­ta­ne­ous­ly, as part of one large study. This could give us a bet­ter way to un­der­stand the com­par­a­tive ben­e­fits of a drug across dif­fer­ent set­tings. To en­able these mas­ter pro­to­cols, it’s of­ten im­por­tant to do mol­e­c­u­lar pa­tient screen­ing. This can lead to the de­vel­op­ment of a di­ag­nos­tic that can al­so be used to guide pa­tient care.

Got­tlieb out­lined plans to in­vest more of the FDA’s mon­ey in new tech­nol­o­gy that can as­sist this faster/bet­ter/cheap­er ap­proach to drug de­vel­op­ment.

On the sec­ond point that I want­ed to high­light to­day, we’re al­so tak­ing new steps to mod­ern­ize how spon­sors can eval­u­ate clin­i­cal in­for­ma­tion, and how FDA re­views this da­ta as part of our reg­u­la­to­ry process.

This starts with bet­ter use of more ad­vanced com­put­ing tools, and more so­phis­ti­cat­ed sta­tis­ti­cal and com­pu­ta­tion­al method­olo­gies, as part of the drug de­vel­op­ment and the drug re­view process. This in­cludes more wide­spread use of mod­el­ing and sim­u­la­tion, and high per­for­mance com­put­ing clus­ters in­side FDA.

FDA al­ready has high per­for­mance com­put­ing clus­ters. These tools help us de­vel­op more so­phis­ti­cat­ed meth­ods for eval­u­at­ing the da­ta that’s sub­mit­ted to us from clin­i­cal tri­als. The com­put­ing tools al­so en­able us to prop­er­ly eval­u­ate the more so­phis­ti­cat­ed com­po­nents that are sub­mit­ted to us as part of prod­uct re­view ap­pli­ca­tions.

Got­tlieb al­so talked about shak­ing up the R&D ap­proach to some spe­cif­ic dis­eases that have proved par­tic­u­lar­ly hard to deal with.

Ad­di­tion­al­ly, to bet­ter de­lin­eate how we’re go­ing to ap­proach the over­all de­vel­op­ment and eval­u­a­tion of drugs tar­get­ed to cer­tain un­met med­ical needs, we plan to be­gin work on at least ten new dis­ease-spe­cif­ic guid­ance doc­u­ments over the next year. Some of these doc­u­ments are al­ready un­der­way. Among the dis­eases we’re tar­get­ing are ar­eas of sig­nif­i­cant un­met need like Amy­otroph­ic Lat­er­al Scle­ro­sis (ALS).

Re­vamp­ing the eco­nom­ics of drug R&D is no sim­ple task.

The on­col­o­gy field has been able to move far­ther and faster than oth­er dis­ease fields due to its abil­i­ty to test new drugs on pa­tients with ad­vanced dis­ease and dwin­dling hope of sur­vival. So it won’t be easy to trans­late that same ap­proach to mass mar­ket dis­eases like di­a­betes and car­dio, where mil­lions are treat­ed for years for chron­ic dis­ease.

An­oth­er big ques­tion is whether bio­phar­ma com­pa­nies will ac­tu­al­ly pass along any sav­ings they get from a more ef­fi­cient de­vel­op­ment path­way to pay­ers and con­sumers. The en­tire in­dus­try has been tip­ping more and more of its de­vel­op­ment dol­lars to can­cer in part be­cause of the big re­wards that come from fast ap­provals. And the FDA has no con­trol what­so­ev­er over the fi­nal price drug de­vel­op­ers use for their new drugs.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Top an­a­lyst finds a sil­ver lin­ing in Ab­b­Vie’s $63B Al­ler­gan buy­out — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took a 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

Endpoints News

Basic subscription required

Unlock this story instantly and join 53,600+ biopharma pros reading Endpoints daily — and it's free.

While Ako­rn works to re­vive its for­tunes, the FDA hits it with an­oth­er warn­ing let­ter

Ako­rn just can’t dig it­self out of its hole.

The spe­cial­ty gener­ic drug­mak­er has re­ceived yet an­oth­er warn­ing let­ter from the FDA this year. With­out dis­clos­ing any specifics, the Lake For­est, Illi­nois-based drug­mak­er on Wednes­day said the US reg­u­la­tor had is­sued the let­ter, cit­ing an in­spec­tion of its Som­er­set, New Jer­sey man­u­fac­tur­ing fa­cil­i­ty in Ju­ly and Au­gust of 2018. The com­pa­ny’s shares $AKRX dipped about 1.7% to $4.65 be­fore the bell.