Joe Biden on the North Lawn of the White House, April 27, 2020 (Evan Vucci/AP Images)

Opin­ion: Mean­ing­ful drug pric­ing re­forms are head­ed nowhere fast

Pres­i­dent Biden is tak­ing a page out of for­mer Pres­i­dent Trump’s drug pric­ing play­book: of­fer big promis­es around tough ne­go­ti­a­tions with drug­mak­ers, but fail to of­fer a vi­able path for get­ting it done.

Warn­ing signs that drug pric­ing would no longer be a pri­or­i­ty for the pres­i­dent’s mar­quee leg­is­la­tion came over the past week from the Wash­ing­ton Post and Wall Street Jour­nal. To the cha­grin of House Speak­er Nan­cy Pelosi (D-CA) and oth­er De­moc­rats, Pres­i­dent Biden’s mas­sive, $1.8 tril­lion so­cial safe­ty net plan to help Amer­i­can fam­i­lies, de­tails of which were un­veiled Wednes­day morn­ing, does not in­clude a ma­jor push to cur­tail drug prices.

Al­though Biden is sup­posed to of­fer more specifics in his speech to Con­gress tonight, in­clud­ing a de­mand for leg­is­la­tion on price ne­go­ti­a­tions this year, stand­alone leg­is­la­tion on ne­go­ti­a­tions is not like­ly to make head­way in the dead­locked Sen­ate, es­pe­cial­ly if it needs 60 votes for pas­sage.

Biden whiffed on an op­por­tu­ni­ty to spell out what he wants in writ­ing.

“Pres­i­dent Biden has a plan to build on the Af­ford­able Care Act and low­er pre­scrip­tion drug costs for every­one by let­ting Medicare ne­go­ti­ate prices,” the White House said Wednes­day. That was the on­ly men­tion of drug prices in the White House fact sheet on the safe­ty net over­haul.

Se­nior Biden of­fi­cials tried to down­play this gap­ing hole where Biden should ex­plain what he wants on drug prices, telling re­porters on a call on Tues­day evening that Biden “has been very, very clear that he re­mains ful­ly com­mit­ted to ne­go­ti­a­tions to re­duce pre­scrip­tion drug prices — that, you will hear him re­it­er­ate as a very top pri­or­i­ty and some­thing he deems ur­gent.”

But the broad­er nar­ra­tive is that drug pric­ing as a pri­or­i­ty is slip­ping, and no one knows why ex­act­ly, con­sid­er­ing the sup­port from House and Sen­ate De­moc­rats and even some Re­pub­li­cans. Per­haps the phar­ma­ceu­ti­cal lob­by has al­ready got­ten to Biden? Per­haps the Biden team doesn’t want to pun­ish drug­mak­ers af­ter how quick­ly they were able to de­vel­op Covid vac­cines?

What­ev­er the rea­son, those as­sur­ances from se­nior staffers mean very lit­tle as the House and Sen­ate re­main di­vid­ed on how to pur­sue drug prices. House De­moc­rats last week rein­tro­duced their land­mark (or dead in the wa­ter, de­pend­ing on which par­ty you lis­ten to) drug pric­ing bill, HR 3, which passed along par­ty lines in the House in De­cem­ber 2019 but was nev­er con­sid­ered by the Sen­ate.

At­tempts to re­vive that bill and oth­ers will be­gin in earnest next week at a House En­er­gy & Com­merce Com­mit­tee hear­ing. But if drug pric­ing leg­is­la­tion is re­al­ly go­ing to make head­way, the Sen­ate will need to weigh in.

Some Sen­ate Re­pub­li­cans have al­ready raised red flags about gov­ern­ment price ne­go­ti­a­tions (even as the US is the on­ly coun­try in the world to not ne­go­ti­ate) and fo­cused their plans in­stead on low­er­ing out-of-pock­et costs.

How the leg­is­la­tion makes it across the fin­ish line re­mains murky, but the need for such leg­is­la­tion is as clear as ever.

A new GAO analy­sis on Wednes­day found that for 20 brand-name drugs in 2020, es­ti­mat­ed US prices paid at the re­tail lev­el by con­sumers and oth­er pay­ers (such as in­sur­ers) were more than two to four times high­er than prices in Aus­tralia, Cana­da and France.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

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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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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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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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.

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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‘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.

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.

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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