Sen­a­tors ques­tion how HHS plans to roll out new drug pric­ing re­forms

While CMS drug price ne­go­ti­a­tions won’t ac­tu­al­ly kick off un­til 2026, a group of more than two dozen Re­pub­li­can sen­a­tors is al­ready rais­ing ques­tions about how this new law will be im­ple­ment­ed.

The par­ti­san law (all De­moc­rats in the House and Sen­ate vot­ed for it, and all Re­pub­li­cans vot­ed against it) in­cludes not on­ly Medicare price ne­go­ti­a­tions but manda­to­ry in­fla­tion-re­lat­ed re­bates that will be­gin next year, as well as mil­lion-dol­lar fines for non­com­pli­ance.

In a let­ter sent ear­li­er this week to both HHS Sec­re­tary Xavier Be­cer­ra and CMS ad­min­is­tra­tor Chiq­ui­ta Brooks-La­Sure, the group of 25 Re­pub­li­can sen­a­tors ex­pressed “con­cerns with the dis­rup­tive and dis­tortive ad­min­is­tra­tive un­der­tak­ing that the im­ple­men­ta­tion of this far-reach­ing fed­er­al ex­pan­sion will in­evitably rep­re­sent.”

They al­so note both the CBO’s point that the ne­go­ti­a­tions could in­crease launch prices for new drugs, and re­duce the num­ber of new drug ap­provals in the com­ing years.

The law’s true im­pact on bio­phar­ma R&D may be dif­fi­cult to track, giv­en the fact that there are usu­al­ly mul­ti­ple rea­sons a drug’s de­vel­op­ment might be dis­con­tin­ued. The CBO has said that over the next 30 years, of the ap­prox­i­mate­ly 1,300 drugs ap­proved, about 15 drugs may be cut from bio­phar­ma com­pa­nies’ col­lec­tive pipelines, in­clud­ing two over the 2023-2032 pe­ri­od, about five over the sub­se­quent decade, and about eight over the third decade.

“Oth­er in­de­pen­dent analy­ses have pro­ject­ed even more cat­a­stroph­ic con­se­quences, from hun­dreds of thou­sands of do­mes­tic job loss­es to hun­dreds of bil­lions in for­gone R&D dol­lars,” the Re­pub­li­cans wrote.

Com­pa­nies like Eli Lil­ly, No­vo Nordisk and As­traZeneca may be dis­pro­por­tion­ate­ly af­fect­ed by the law.

The Re­pub­li­cans, which in­clud­ed Utah’s Mitt Rom­ney, North Car­oli­na’s Thom Tillis, and West Vir­ginia’s Shel­ley Moore Capi­to, al­so note that the law’s im­ple­men­ta­tion doesn’t use the stan­dard no­tice-and-com­ment rule­mak­ing process typ­i­cal­ly re­quired for such “sub­stan­tive reg­u­la­to­ry pol­i­cy­mak­ing.” This “ex­clu­sion of pa­tient voic­es from pol­i­cy­mak­ing pos­es po­ten­tial­ly cat­a­stroph­ic con­se­quences, par­tic­u­lar­ly for in­di­vid­u­als liv­ing with rare dis­eases or with dis­abil­i­ties,” they wrote, re­quest­ing de­tails on how those voic­es will be heard.

The group al­so seeks in­fo on plans to “en­force the leg­is­la­tion’s ban on the use of dis­crim­i­na­to­ry com­par­a­tive ef­fec­tive­ness re­search” and ques­tions on “the lack of a floor for Sec­re­tary-dic­tat­ed prices.”

And the fi­nal ques­tion from the group is on how the ad­min­is­tra­tion plans to mon­i­tor these wide-rang­ing ef­fects of the new pro­gram, on every­thing from pa­tient ac­cess to R&D in­vest­ments, launch prices, new drug ap­provals and do­mes­tic man­u­fac­tur­ing jobs.

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Benjamine Liu, TrialSpark CEO

Paul Hud­son and Tri­alSpark's mu­tu­al de­sire to speed up de­vel­op­ment con­verges in three-year, six-drug goal

A unicorn startup that originally set out to hasten clinical studies for biopharma partners dug further into its revised path of internal drug development by linking arms with Sanofi in a pact that the biotech’s CEO said originated from the top.

TrialSpark and the Big Pharma on Tuesday committed to in-licensing and/or acquiring six Phase II/Phase III drugs within the next three years.

“I’ve known Paul Hudson for a while and we were discussing the opportunity to really re-imagine a lot of different parts of pharma,” TrialSpark CEO Benjamine Liu told Endpoints News, “and one of the things that we discussed was this opportunity to accelerate the development of new medicines in mutual areas of interest.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Take­da to pull key hy­poparathy­roidism drug from the mar­ket en­tire­ly by end of 2024 af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024.

The decision to not re-commercialize Natpara will be a blow to not only the 2,400 people who were awaiting supplies of their reliable injection since 2019, but also the additional nearly 400 people who were accessing the drugs via the company’s Special Use Program as Takeda sought to resolve these manufacturing issues over the past five years.

Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP)

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,300+ biopharma pros reading Endpoints daily — and it's free.

Astel­las, Pan­th­er­na add or­gan to mR­NA tie-up; Rock­et launch­es sale of six fig­ures worth of stock

Astellas and Pantherna have expanded their November 2021 pact surrounding the latter’s mRNA platform to include a new target organ, the duo announced Tuesday morning, though they did not specify what that target is.

German biotech Pantherna is home to two platform technologies — one that designs mRNAs for non-vaccine therapies and another that designs LNPs. Astellas and Pantherna’s deal appears to mainly revolve around the first platform, which Astellas said it is using to research direct reprogramming, or turning cells from one kind into another without an intermediate stem cell phase.