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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Alaa Halawaa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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Rohan Palekar, 89bio CEO

89bio’s PhII da­ta add to quick suc­ces­sion of NASH read­outs as field seeks turn­around

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.

Flare Therapeutics biochemists Yong Li (L) and Valerie Vivat

A $123M Flare will get Third Rock on­col­o­gy biotech in­to the clin­ic this year

Flare Therapeutics will start its first human trial this year with an investigational urothelial cancer drug after pulling together a $123 million Series B from Big Pharmas, VCs and its incubator, Third Rock Ventures.

Launched in 2021 on the idea that a biotech could finally succeed at drugging the much-sought-after but stubborn transcription factor, Flare Therapeutics said Wednesday it is now primed for the clinic after closing its large financing haul earlier this year. The raise is a relatively stark figure in a tough startup financing environment but further buoys the upbeat signals coming out of other Third Rock biotechs in recent weeks, including the $200 million CARGO Therapeutics and $100 million Rapport Therapeutics rounds.

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Roche and Lil­ly team up to de­vel­op blood test to de­tect ear­ly signs of Alzheimer's

Eli Lilly is teaming up with Roche to help develop a blood test to detect early signs of Alzheimer’s disease and determine whether a patient should go for further confirmatory testing.

Roche’s Elecsys Amyloid Plasma Panel (EAPP) measures pTau 181 protein assay and APOE E4 assay in human blood plasma – elevations in pTau 181 are present in the early stages of Alzheimer’s, while the presence of APO E4 is the most common genetic risk factor for the disease.

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Francesco Marincola, newly-appointed Sonata Therapeutics CSO

Kite's head of re­search leaves for Flag­ship start­up Sonata

Another leader is departing Kite Pharma, and will to spend the “last part” of his career exploring how cancer evades the immune system.

Kite’s senior VP and global head of cell therapy research Francesco Marincola left the Gilead CAR-T unit last week for Sonata Therapeutics. Flagship last May unveiled the startup, which was pieced together from two fledgling biotechs Inzen and Cygnal Therapeutics. As CSO, Marincola will lead Sonata’s push to reprogram cancer cells to make them more immunogenic.

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FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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NIH re­jects an­oth­er at­tempt to 'march-in' on Astel­las' prostate can­cer drug over ex­ces­sive price

The National Institutes of Health has again declined to use so-called “march-in” rights to lower the price of Astellas and Pfizer’s prostate cancer drug Xtandi despite being invented at UCLA with grants from the US Army and NIH.

“Given the remaining patent life and the lengthy administrative process involved for a march-in proceeding, NIH does not believe that use of the march-in authority would be an effective means of lowering the price of the drug,” NIH told prostate cancer patients Robert Sachs and Clare Love, in a letter shared with Endpoints News. The institutes’ analyses found Xtandi “to be widely available to the public,” an indication that there was not a pressing need for the US to act.

No­vo Nordisk re­mains un­der UK scruti­ny as MHRA con­ducts its own re­view in 'in­cred­i­bly rare' case

The UK’s Medicines and Healthcare products Regulatory Agency is now reviewing Novo Nordisk’s marketing violation that resulted in its loss of UK trade group membership last week. Novo Nordisk was suspended on Thursday from the Association of the British Pharmaceutical Industry (ABPI) for two years after an investigation by its regulatory arm found the pharma broke its conduct rules.

MHRA said on Tuesday that its review of the Prescription Medicines Code of Practice Authority (PMCPA) investigation is standard practice. An MHRA spokesperson emphasized in an email to Endpoints News that the situation with Novo Nordisk is “incredibly rare” while also noting ABPI took “swift and proportionate action.”

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