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.

Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors. 

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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Part club, part guide, part land­lord: Arie Bellde­grun is blue­print­ing a string of be­spoke biotech com­plex­es in glob­al boom­towns — start­ing with Boston

The biotech industry is getting a landlord, unlike anything it’s ever known before.

Inspired by his recent experiences scrounging for space in Boston and the Bay Area, master biotech builder, investor, and global dealmaker Arie Belldegrun has organized a new venture to build a new, 250,000 square foot biopharma building in Boston’s Seaport district — home to Vertex and a number of up-and-coming biotech players.

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