As FDA looks to speed re­views even more, 2 pol­i­cy ex­perts want to re­strict the price of drugs that win an ac­cel­er­at­ed OK

Even af­ter the FDA added reg­u­la­to­ry path­ways for drug de­vel­op­ers to win ac­cel­er­at­ed ap­provals for new drugs, the po­lit­i­cal pres­sure in Wash­ing­ton to speed up drug re­views con­tin­ues to grow.

In tes­ti­mo­ny be­fore a House sub­com­mit­tee yes­ter­day, FDA com­mis­sion­er Scott Got­tlieb as­sured law­mak­ers that bio­mark­ers, new tech­nolo­gies and more ef­fi­cient tri­al de­signs made it pos­si­ble to short­en the reg­u­la­to­ry process as he vowed to urge all the FDA to repli­cate the fast pace of the agency’s on­col­o­gy di­vi­sion, which has re­con­fig­ured can­cer drug de­vel­op­ment pro­grams over the past 3 years.

But should drugs ap­proved ear­ly with on­ly part of the da­ta that was once re­quired for an OK be able to fetch the full re­tail price that man­u­fac­tur­ers ex­pect to­day?

Two health pol­i­cy ex­perts say no.

Aaron Kessel­heim
Walid Gel­lad

In an op-ed for The New Eng­land Jour­nal of Med­i­cine, Walid Gel­lad from the Uni­ver­si­ty of Pitts­burgh and Har­vard’s Aaron Kessel­heim ar­gue that any bio­phar­ma com­pa­ny that wins an ac­cel­er­at­ed ap­proval should be sub­ject to cer­tain price re­stric­tions. And they’ve of­fered a few ex­am­ples of how that could work. You could, for ex­am­ple:

— Re­quire drug mak­ers to of­fer pub­lic pay­ers a set dis­count on drugs that get an ear­ly OK ahead of con­fir­ma­to­ry stud­ies. Med­ic­aid could get a statu­to­ry price re­duc­tion on top of the dis­counts it al­ready qual­i­fies for.

— Hold a por­tion of the rev­enue from these drugs in es­crow, un­til they prove they work as as­sumed based on the pre­lim­i­nary da­ta. Drug mak­ers can win it on a pos­i­tive Phase III, or lose it all as the cash is used to re­im­burse pay­ers.

— To avoid any gam­ing of this sys­tem, hik­ing the whole­sale price to make sure sell­ers make what they want from the dis­count­ed fig­ure, man­u­fac­tur­ers could be forced to switch to a cost-plus sys­tem, with set mar­gins.

The au­thors al­so call for a new sys­tem where de­vel­op­ers are held ac­count­able to see­ing their late-stage tri­als through on sched­ule. A sys­tem of re­wards and penal­ties can be put in place for com­pa­nies as they set out to achieve spe­cif­ic mile­stones in their stud­ies. And no more long run­ways, they say. New tri­als should start with­in months of an ac­cel­er­at­ed OK. And these con­fir­ma­to­ry stud­ies should be ex­pect­ed to wrap in a rea­son­able amount of time, not ex­tend for years in­to the fu­ture.

We be­lieve there should be plans in place to be­gin con­fir­ma­to­ry tri­als with­in 3 months af­ter ap­proval, with track­ing of tri­al progress through Clin­i­cal­Tri­als.gov. Though the rar­i­ty of the dis­ease and oth­er fac­tors might rea­son­ably af­fect tri­al ac­cru­al times, there should al­so be mean­ing­ful reper­cus­sions for miss­ing mile­stones such as hav­ing a pro­to­col in place or hit­ting re­cruit­ment tar­gets, cul­mi­nat­ing in with­draw­al of the drug if the tri­al is un­nec­es­sar­i­ly de­layed for an ex­tend­ed pe­ri­od. The FDA can, un­der cur­rent law, as­sess fi­nan­cial penal­ties or with­draw an ac­cel­er­at­ed-ap­proval drug from the mar­ket if the man­u­fac­tur­er fails to con­duct its con­fir­ma­to­ry tri­al or fails to do so with “due dili­gence,” a bench­mark that the FDA can fur­ther clar­i­fy with stake­hold­er in­put.

Even more con­tro­ver­sial­ly, they sug­gest that an eco­nom­ic im­pact study should be used to eval­u­ate these drugs af­ter one or two years on the mar­ket, to see if the val­ue of a drug giv­en an ac­cel­er­at­ed ap­proval is lost to the fi­nan­cial tur­moil it can cause.

As far as the in­dus­try is con­cerned, there isn’t any­thing here that would slip un­der the radar. It would all be fought tooth and nail. Ag­gres­sive gov­ern­ment reg­u­la­tions re­strict­ing prices and gov­ern­ing tri­als is anath­e­ma to bio­phar­ma, which much prefers vol­un­tary re­straint in the US. But as the de­bate over drug prices con­tin­ues to boil in Wash­ing­ton DC, it’s an­oth­er set of “so­lu­tions” like­ly to trig­ger fresh de­bate at a time ac­cel­er­at­ed ap­provals may just be get­ting start­ed.

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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