Committee Chair Patty Murray (D-WA) (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­a­tors grill in­dus­try groups over FDA user fee agree­ments to fund the agency through 2027

Mem­bers of the Sen­ate health com­mit­tee on Tues­day fired off ques­tions at drug in­dus­try reps in the first of two hear­ings to deal with the lat­est five-year fund­ing agree­ments that Con­gress has to re-au­tho­rize by the end of Sep­tem­ber and that the FDA re­lies on to re­view new drug, gener­ic drug, bi­o­log­ic and biosim­i­lar ap­pli­ca­tions.

Com­mit­tee Chair Pat­ty Mur­ray (D-WA) made clear in her open­ing re­marks that the FDA has done a su­pe­ri­or job, par­tic­u­lar­ly when it’s come to the pan­dem­ic, liv­ing up to its “gold stan­dard” tagline, but “FDA doesn’t run on grat­i­tude.”

Now in its sev­enth it­er­a­tion, Pre­scrip­tion Drug User Fee Act or PDU­FA has mor­phed in­to a way to pro­vide ad­di­tion­al rev­enue to the agency so it can hire more staff, im­prove its sys­tems and es­tab­lish a bet­ter-man­aged, more re­li­able re­view process to make im­por­tant drugs avail­able to pa­tients soon­er.

While the FDA’s re­mit doesn’t in­clude drug pric­ing, Mur­ray stressed the need to fight sky­rock­et­ing health costs and stop com­pa­nies from deny­ing gener­ics and biosim­i­lars ac­cess to the mar­ket.

Richard Burr

But on the oth­er side of the aisle, rank­ing mem­ber Richard Burr (R-NC) ques­tioned the need for these ever-ex­pand­ing user fee agree­ments, which pro­vide for the ma­jor­i­ty of the FDA’s to­tal fund­ing. In 1993, he not­ed, PDU­FA was first au­tho­rized and in­clud­ed on­ly about $35 mil­lion for FDA. To­day, the agree­ment is worth more than $1 bil­lion.

Burr stressed that the FDA has con­tin­ued to see in­creas­es in fund­ing with each new -UFA reau­tho­riza­tion, even as the FDA has strug­gled to meet cer­tain agreed-to goals, in­clud­ing those around sched­ul­ing meet­ings and staffing up.

Much of this round’s new fund­ing will go to­ward hir­ing more FDA staff, Burr not­ed, and while the FDA is com­mit­ting to 884 new hires over the next 5 years, the agency still has around 700 va­can­cies out­stand­ing.

CDER Di­rec­tor Pa­trizia Cavaz­zoni yes­ter­day al­so called on Con­gress to pro­vide more funds for ad­di­tion­al hires af­ter a spike in staff loss­es last sum­mer.

“The FDA is in­creas­ing­ly re­mov­ing it­self from Con­gress’ reach and my col­leagues should be con­cerned about this,” Burr said, stress­ing that the agency should be held ac­count­able for its com­mit­ments.

Sens. Tim Kaine (D-VA) and Su­san Collins (R-ME) al­so ques­tioned in­dus­try group BIO’s rep re­gard­ing the FDA’s plans for a new pi­lot pro­gram to sup­port ef­fi­ca­cy end­point de­vel­op­ment for rare dis­ease drugs by of­fer­ing ad­di­tion­al en­gage­ment op­por­tu­ni­ties.

BIO CSO Carti­er Es­ham said the pi­lot will build off work the FDA has done on pi­lots re­lat­ed to mod­el-in­formed drug de­vel­op­ment and com­plex in­no­v­a­tive tri­al de­signs, and help the FDA and spon­sors con­nect on what’s need­ed to sup­port the uti­liza­tion of a new end­point.

Mean­while, Pew’s health care di­rec­tor Liz Richard­son told the com­mit­tee that while her NGO sup­ports the reau­tho­riza­tion of these user fee agree­ments, the FDA needs more re­sources to pro­tect pub­lic health, and should be ac­count­able to the pub­lic, not just the in­dus­try it reg­u­lates.

Oth­er sen­a­tors raised small­er, more point­ed con­cerns re­gard­ing the FDA’s progress in cer­tain ar­eas.

For in­stance, Sen. Chris Mur­phy (D-CT) lament­ed the lack of new drugs to tack­le men­tal health con­di­tions over the past 8 years, while Sen. Mike Braun (R-IN) called for more of a fo­cus on the pa­tient voice.

David Gaugh, SVP at the gener­ic drug in­dus­try group, al­so ex­plained to Sen. Mag­gie Has­san (D-NH) how the lat­est GDU­FA will help in­crease gener­ic drug ap­provals as it in­cludes a pro­vi­sion that will al­low the FDA to take an “im­mi­nent ac­tion” route so as to avoid is­su­ing a com­plete re­sponse let­ter when a gener­ic drug could be ap­proved, but the agency may need an ad­di­tion­al 60 days to re­view the app.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Vlad Coric, Biohaven CEO

Vlad Coric charts course for new Bio­haven with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the new Biohaven post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

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Robert Califf, FDA commissioner (via AP Images)

User fees in ac­tion: FDA un­veils new short­ened sup­ple­ment re­view, rare dis­ease pi­lots

Thanks to PDUFA VII, signed into law last Friday by President Joe Biden, the FDA this week unveiled two new industry-friendly pilot programs to advance new rare disease endpoints via additional meetings, and to shorten FDA review times for supplemental apps aimed at unmet medical needs.

The agency this week released eagerly-awaited details behind the shortened pilot, known as the Split Real Time Application Review or STAR pilot program, which will speed up certain FDA reviews of efficacy supplements across all therapeutic areas (thanks to earlier submissions of data), but only for those that propose addressing an unmet medical need.

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Mar­ket­ingRx roundup: No­var­tis re­cruits NFL coach for Leqvio cam­paign; Pfiz­er pro­motes ‘Sci­ence’ merch on so­cial me­dia

Novartis is turning to a winning coach to talk about Leqvio and the struggles of high cholesterol — including his own. Bruce Arians, the retired NFL head coach of the Arizona Cardinals and Super Bowl-winning Tampa Bay Buccaneers, is partnering with the pharma for its “Coaching Cholesterol” digital, social and public relations effort.

In the campaign, Arians talks about the potential for “great comebacks” in football and heart health. Once nicknamed a “quarterback whisperer,” he is now retired from fulltime coaching (although still a front-office consultant for Tampa Bay), and did a round of media interviews for Novartis, including one with People and Forbes.

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Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: No­vo Nordisk dig­i­tal in­no­va­tion chief Amy West dis­cuss­es phar­ma pain points and a health­care 'easy but­ton’

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

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