Dems, Re­pub­li­cans split on 340B drug pric­ing pro­gram in Sen­ate hear­ing

The di­vid­ing line be­tween Sen­ate De­moc­rats and Re­pub­li­cans on the 340B drug pric­ing pro­gram was set on Thurs­day, with De­moc­rats sid­ing with the hos­pi­tals, say­ing the sav­ings from the pro­gram is des­per­ate­ly need­ed for the poor­est pop­u­la­tions, while Re­pub­li­cans took the side of drug­mak­ers, say­ing the pro­gram is be­ing abused and needs to be re­formed.

But the hear­ing al­so put the spot­light on the lack of trans­paren­cy from both the phar­ma­ceu­ti­cal and hos­pi­tal in­dus­tries, as nei­ther side could agree to some ba­sic sta­tis­tics, such as what per­cent of the to­tal drug spend in the US goes in­to the 340B pro­gram.

Lamar Alexan­der

For in­stance, the Health Re­sources & Ser­vices Ad­min­is­tra­tion, which over­sees the pro­gram, says that some­where be­tween 1% and 2% of the na­tion’s drug spend is at­trib­uted to the 340B pro­gram.

But Sen Lamar Alexan­der (R-TN) on Thurs­day ques­tioned what per­cent of what Amer­i­cans spend on pre­scrip­tion drugs is avail­able to safe­ty net hos­pi­tals and clin­ics for the pur­pos­es of 340B.

“Is it $6 bil­lion or $8 bil­lion or $14 bil­lion? If it’s 1 or 2%, well that’s just a tax on phar­ma­ceu­ti­cal com­pa­nies that we’re spend­ing for a good pur­pose, but if it’s 6% to 8%, then that’s a pret­ty big tax. I’d like to get those fig­ures,” Alexan­der said.

Lori Reil­ly

Lori Reil­ly, ex­ec­u­tive vice pres­i­dent of the lob­by­ing group PhRMA, told law­mak­ers that the to­tal spend on dis­counts by phar­ma­ceu­ti­cal com­pa­nies was $8 bil­lion in 2016, and that the to­tal 2016 spend on phar­ma­ceu­ti­cals in the US was “in the $390 bil­lion range,” though she could not con­firm an ex­act fig­ure to Alexan­der.

Sen Eliz­a­beth War­ren (D-MA), mean­while, said the size of the US drug mar­ket in 2015 was $457 bil­lion, while point­ing out that phar­ma­ceu­ti­cal com­pa­nies’ mar­gins were sig­nif­i­cant­ly high­er (she said six times as high) than hos­pi­tals’ mar­gins.

Eliz­a­beth War­ren

Pew Char­i­ta­ble Trusts, mean­while, re­cent­ly spot­light­ed the var­i­ous es­ti­mates and pro­jec­tions on drug spend­ing in the US, not­ing the dif­fer­ences among dif­fer­ent groups.

And though PhRMA’s Reil­ly said ear­li­er in the hear­ing that phar­ma­ceu­ti­cal com­pa­nies “do not want this pro­gram to dis­ap­pear,” War­ren took is­sue with PhRMA’s ar­gu­ment that the 340B rais­es drug prices.

“If 340B didn’t ex­ist, drug com­pa­nies would have an ex­tra $6 bil­lion in their pock­et, that’s less than 1% of glob­al phar­ma­ceu­ti­cal sales rev­enue,” War­ren said. “The loss that they’re kick­ing and scream­ing about is a tiny frac­tion of the many bil­lions of dol­lars they pull down every year in prof­its.”

Rep­re­sen­ta­tives of health cen­ters and hos­pi­tals, mean­while, not­ed that re­forms to low­er what drug­mak­ers spend on 340B dis­counts would hurt.

How­ev­er, re­form and more over­sight may still come.

study pub­lished in Health Af­fairs in 2014 found that 340B hos­pi­tals are ex­pand­ing their base in­to com­mu­ni­ties that tend to be af­flu­ent and well-in­sured, which runs counter to the ob­jec­tives of the pro­gram. The Al­liance for In­tegri­ty and Re­form of 340B, backed by the bio­phar­ma in­dus­try and oth­er groups, al­so re­leased a re­port that found in 2015, 61% of par­tic­i­pants spent less on char­i­ty care com­pared to both 2014 and 2013 de­spite ad­di­tion­al rev­enue re­ceived.


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.

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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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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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.

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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