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.

How one start­up fore­told the neu­ro­science re­nais­sance af­ter '50 years of shit­show'

In the past couple of years, something curious has happened: Pharma and VC dollars started gushing into neuroscience research.

Biogen’s controversial new Alzheimer’s drug Aduhelm has been approved on the basis of removing amyloid plaque from the brain, but the new neuro-focused pharma and biotechs have much loftier aims. Significantly curbing or even curing the most notorious disorders would prove the Holy Grail for a complex system that has tied the world’s best drug developers in knots for decades.

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Ryan Watts, Denali CEO

De­nali slips as a snap­shot of ear­ly da­ta rais­es some trou­bling ques­tions on its pi­o­neer­ing blood-brain bar­ri­er neu­ro work

Denali Therapeutics had drummed up considerable hype for their blood-brain barrier technology since launching over six years ago, hype that’s only intensified in the last 14 months following the publications of a pair of papers last spring and proof of concept data earlier this year. On Sunday, the South San Francisco-based biotech gave the biopharma world the next look at in-human data for its lead candidate in Hunter syndrome.

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Why is On­col­o­gy Drug De­vel­op­ment Re­search Late to the Dig­i­tal Bio­mark­ers Game?

During the recent Annual ASCO Meeting, thousands of cancer researchers and clinicians from across the globe joined together virtually to present and discuss the latest findings and breakthroughs in cancer research and care. There were more than 5000+ scientific abstracts presented during this event, yet only a handful involved the use of motion-tracking wearables to collect digital measures relating to activity, sleep, mobility, functional status, and/or quality of life. Although these results were a bit disappointing, they should come as no surprise to those of us in the wearable technology field.

Bob Bradway, Amgen CEO (Scott Eisen/Bloomberg via Getty Images)

Am­gen bel­lies back up to the M&A ta­ble for an­oth­er biotech buy­out, this time with a $2.5B deal for an an­ti­body play­er fo­cused on PS­MA

Five months after Amgen CEO Bob Bradway stepped up to the M&A table and acquired Five Prime for $1.9 billion, following up with the smaller Rodeo acquisition, he’s gone back in for another biotech buyout.

This time around, Amgen is paying $900 million cash while committing up to $1.6 billion in milestones to bag the privately held Teneobio, an antibody drug developer that has expertise in developing new bispecifics and multispecifics. In addition, Amgen cited Teneobio’s “T-cell engager platform, which expands on Amgen’s existing leadership position in bispecific T-cell engagers by providing a differentiated, but complementary, approach to Amgen’s current BiTE platform.”

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J&J’s Rem­i­cade — the poster child for how to block biosim­i­lars — fi­nal­ly set­tles Pfiz­er suit

Biosimilars have proven time and again (although mostly in Europe) that competition works to bring down the cost of a once-pricey biologic, and can even expand its use.

J&J’s Remicade, however, has always proven to be an outlier.

Back in 2016, Pfizer won FDA approval for its infliximab biosimilar, known as Inflectra, but when the launch foundered, the company sued J&J, claiming that the company’s plan to block biosimilar competition worked incredibly well. Pfizer even went on to win FDA approval for a second infliximab biosimilar in 2017, known as Ixifi, but decided to never launch it.

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Art Levinson (Calico)

Google-backed Cal­i­co dou­bles down on an­ti-ag­ing R&D pact with Ab­b­Vie as part­ners ante up $1B, start to de­tail drug tar­gets

Seven years after striking up a major R&D alliance, AbbVie and Google-backed anti-aging specialist Calico are doubling down on their work with a joint, $1 billion commitment to continuing their work together. And they’re also beginning to offer some details on where this project is taking them in the clinic.

According to their statement, each of the two players is putting up $500 million more to keep the labs humming.

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UP­DAT­ED: Pan­el of neu­ro­science ex­perts lays out the com­pli­ca­tions with us­ing Bio­gen's new Alzheimer's drug

Treatment of early Alzheimer’s patients with Biogen’s new drug Aduhelm should closely resemble how the drug was studied in its pivotal clinical trials, according to new recommendations from a panel of neuroscience experts led by UNLV’s Jeffrey Cummings.

“Those considering aducanumab therapy should understand that the expected benefit is slowing of cognitive and functional decline; improvement of the current clinical state is not anticipated,” they wrote Tuesday in The Journal of Prevention of Alzheimer’s Disease, noting that some of their recommendations are more specific or more restrictive than the information provided in the FDA’s prescribing information.

Busi­ness­es and schools can man­date the use of Covid-19 vac­cines un­der EUAs, DOJ says

As public and private companies stare down the reality of the Delta variant, many are now requiring that their employees or students be vaccinated against Covid-19 prior to attending school or to returning or starting a new job. Claims that such mandates are illegal or cannot be used for vaccines under emergency use authorizations have now been dismissed.

Setting the record straight, the Department of Justice on Monday called the mandates legal in a new memo, even when used for people with vaccines that remain subject to EUAs.

Ugur Sahin, BioNTech CEO (Bernd von Jutrczenka/dpa via AP Images)

BioN­Tech is spear­head­ing an mR­NA vac­cine de­vel­op­ment pro­gram for malar­ia, with a tech trans­fer planned for Africa

Flush with the success of its mRNA Covid-19 vaccine, BioNTech is now gearing up for one of the biggest challenges in vaccine development — which comes without potential profit.

The German mRNA pioneer says it plans to work on a jab for malaria, then transfer the tech to the African continent, where it will work with partners on developing the manufacturing ops needed to make this and other vaccines.

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