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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

Alaa Halawa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Genen­tech to stop com­mer­cial man­u­fac­tur­ing at Cal­i­for­nia head­quar­ters

Genentech is halting commercial manufacturing at its California headquarters — and laying off several hundred employees.

The move is the result of a decision Genentech made in 2007 to relocate manufacturing operations from its South San Francisco headquarters location to other facilities or move the work to CDMOs, said Andi Goddard, Genentech’s SVP of quality and compliance for pharmaceutical technical operations, in an interview with Endpoints News. Genentech has made changes in capabilities and invested more in technology, so it doesn’t need as many large-scale manufacturing facilities as it did in the past, she said.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

In­cyte wins ac­cel­er­at­ed ap­proval for PD-1 in rare skin can­cer

Incyte touted an accelerated approval for its PD-1 retifanlimab in a rare skin cancer on Wednesday, roughly a year and a half after the drug suffered a rejection in squamous cell carcinoma of the anal canal (SCAC).

Retifanlimab, marketed as Zynyz, was approved for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC), a fast-growing skin cancer typically characterized by a single, painless nodule. It’s roughly 40 times rarer than melanoma, according to the nonprofit Skin Cancer Foundation — but incidence is growing, particularly among older adults, Incyte said in its announcement.

A new study finds that many patient influencers are sharing prescription drug experiences along with health information.

So­cial me­dia pa­tient in­flu­encers ‘danc­ing in the gray’ of phar­ma mar­ket­ing, more clar­i­ty need­ed, re­searcher says

It’s no surprise that patient influencers are talking about their health conditions on social media. However, what’s less clear is what role pharma companies are playing, how big the patient influencer industry is, and just how is information about prescription drugs from influencers relayed — and received — on social media.

While University of Colorado associate professor Erin Willis can’t answer all those questions, she’s been researching the issue for several years and recently published new research digging into the communication styles, strategies and thinking of patient influencers, many of whom partner with pharma companies.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

Drug short­age so­lu­tions brought be­fore Sen­ate Home­land Se­cu­ri­ty com­mit­tee

With more than 300 active drug shortages, the Senate Committee on Homeland Security and Governmental Affairs had its hands full on Wednesday with multiple experts testifying on drug shortages and possible solutions.

A picture of the shortage situation. presented by Erin Fox, an adjunct professor at the College of Pharmacy at the University of Utah, explained how some patients have died due to drug shortages, including with medication errors when substitutes were dosed incorrectly or when an emergency product was not available.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.

FDA re­jects Ab­b­Vie's in­fu­sion ther­a­py for Parkin­son's, re­quests more in­fo on pump de­vice

The FDA rejected AbbVie’s 24-hour infusion therapy for Parkinson’s, saying it needs more information on a device used to administer the treatment before it can clear it.

The Chicago-area drugmaker said in a press release that the complete response letter from the agency didn’t include any requests for more efficacy or safety trials related to the drug, known as ABBV-951. The company said it aims to “resubmit the NDA as soon as possible.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,500+ biopharma pros reading Endpoints daily — and it's free.