FDA chief Scott Got­tlieb promis­es to stream­line drug de­vel­op­ment path­way for bio­phar­ma

FDA Com­mis­sion­er Scott Got­tlieb on Tues­day told a House Ap­pro­pri­a­tions sub­com­mit­tee that the agency plans to “sharply in­crease” its re­lease of dis­ease-fo­cused guid­ance doc­u­ments to help drug­mak­ers de­vel­op new treat­ments.

“Among some of the many ar­eas we’re work­ing on right now are new guid­ance to lay out mod­ern cri­te­ria for the de­vel­op­ment of drugs tar­get­ed to ul­cer­a­tive col­i­tis; rare pe­di­atric can­cers; pe­di­atric HIV; and se­ri­ous, life-threat­en­ing and non-can­cer blood dis­or­ders like aplas­tic ane­mia,” ac­cord­ing to Got­tlieb’s writ­ten tes­ti­mo­ny on the 2019 bud­get.

The new guid­ance doc­u­ments will aim to make drug de­vel­op­ment more ef­fi­cient and fo­cus more on de­vel­op­ing drugs tar­get­ed to less-com­mon con­di­tions where there’s a lack of avail­able ther­a­py and de­vel­op­ment path­ways can be chal­leng­ing.

“For ex­am­ple, the guid­ance on blood dis­or­ders will al­low drug de­vel­op­ers to re­duce the use of an­i­mal test­ing and will out­line ways to mea­sure ben­e­fit that may per­mit more ef­fi­cient de­vel­op­ment pro­grams and ear­li­er ap­provals,” his tes­ti­mo­ny said.

He al­so not­ed how clin­i­cal tri­al de­sign “can be par­tic­u­lar­ly hard and guid­ance in this area will re­duce reg­u­la­to­ry un­cer­tain­ty and pro­vide clar­i­ty to drug de­vel­op­ers. The fo­cus of this pol­i­cy work mir­rors the gen­er­al di­rec­tion of drug de­vel­op­ment, where new sci­ence is en­abling us to dis­cov­er nov­el, safe and ef­fec­tive treat­ments for pre­vi­ous­ly in­tractable ill­ness­es.”

In ad­di­tion to the guid­ance, he said FDA is look­ing to set up a pol­i­cy of­fice‎ in­side the Of­fice of New Drugs in CDER that will “dis­till and align reg­u­la­to­ry, clin­i­cal and sci­en­tif­ic rea­son­ing of re­view di­vi­sions to pro­mote pol­i­cy trans­paren­cy and con­sis­ten­cy.”

Dis­cus­sions be­tween rep­re­sen­ta­tives and Got­tlieb in Tues­day’s hear­ing cen­tered around FDA’s work to reg­u­late elec­tron­ic cig­a­rettes, the opi­oid epi­dem­ic, con­tin­u­ous man­u­fac­tur­ing in­no­va­tion and med­ical de­vice safe­ty.

First pub­lished here. Reg­u­la­to­ry Fo­cus is the flag­ship on­line pub­li­ca­tion of the Reg­u­la­to­ry Af­fairs Pro­fes­sion­als So­ci­ety (RAPS), the largest glob­al or­ga­ni­za­tion of and for those in­volved with the reg­u­la­tion of health­care and re­lat­ed prod­ucts, in­clud­ing med­ical de­vices, phar­ma­ceu­ti­cals, bi­o­log­ics and nu­tri­tion­al prod­ucts. Email news@raps.org for more in­for­ma­tion. 

Im­age: Scott Got­tlieb. AP IM­AGES

The Price of Re­lief: Ex­plor­ing So­lu­tions to the Ris­ing Costs of On­col­o­gy Drugs

In 2020, The National Cancer Institute estimated about 1.8 million new cases of cancer diagnosed in the United States, while the costs associated with treatment therapies continued to escalate. Given the current legislative climate on drug pricing, it’s never been more important to look at the evolution of drug pricing globally and control concerns of sustainable and affordable treatments in oncology.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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UP­DAT­ED: CMS to re­strict cov­er­age of Bio­gen's con­tro­ver­sial Alzheimer's drug to on­ly clin­i­cal tri­als

The Centers for Medicare and Medicaid Services on Tuesday said it will only pay for Biogen’s Aduhelm and other FDA-approved anti-amyloid monoclonal antibodies for Alzheimer’s disease under CMS-approved randomized controlled trials.

The draft national coverage decision, which insurers nationwide are likely to follow, makes clear that CMS will be looking for randomized controlled trials that “demonstrate a clinically meaningful benefit in cognition and function.” That will be a tough task for Biogen, which previously showed conflicting benefits from past Aduhelm trials that were initially cut short due to futility and then resurrected for the accelerated approval.

CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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