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.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.

Stanley Erck, Novavax CEO (Andrew Harnik/AP Images)

No­vavax pulls out of Covid-19 vac­cine al­liance with Gavi

Novavax is pulling out of its Covid-19 vaccine deal with Gavi, the Vaccine Alliance, a global partnership tasked with ensuring vaccine access in lower-income countries, following an alleged contract violation.

The Maryland-based company claimed on Friday that Gavi failed to purchase at least 350 million doses of its protein-based vaccine Nuvaxovid by the end of the year, per an advanced purchase agreement. Gavi, the World Health Organization and the Coalition for Epidemic Preparedness Innovations (CEPI) are co-leaders of COVAX, an effort to ensure that all participating countries, regardless of income levels, have access to vaccines.

Fu­ji­film to build $188M man­u­fac­tur­ing plant in North Car­oli­na’s re­search tri­an­gle

As the Japanese conglomerate Fujifilm continues to invest heavily in its CDMO arm, one of its manufacturing divisions is teeing up a major investment.

Fujifilm Irvine Scientific announced on Tuesday that parent Fujifilm is making a $188 million investment to build a cell culture media manufacturing site in the Research Triangle Park in North Carolina. The new site will mark Fujifilm Irvine’s fifth manufacturing site globally and its second in the US.