ICER plots ear­ly sci­en­tif­ic ad­vice pro­gram for bio­phar­ma

The In­sti­tute for Clin­i­cal and Eco­nom­ic Re­view (ICER) is look­ing to help the bio­phar­ma in­dus­try with ear­li­er re­views of clin­i­cal work, adding to their cur­rent in­de­pen­dent eval­u­a­tions of the clin­i­cal and eco­nom­ic val­ue of pre­scrip­tion drugs, med­ical tests and oth­er health in­no­va­tions.

David Whitrap

“For some time, ICER has been re­ceiv­ing re­quests from life sci­ences com­pa­nies to help them re­think clin­i­cal tri­al de­sign, so that the tri­als more ad­e­quate­ly mea­sure the types of out­comes that mat­ter most to pa­tients and their fam­i­lies,” David Whitrap, ICER VP of com­mu­ni­ca­tions and out­reach, told Fo­cus.

The idea float­ed is that bio­phar­ma com­pa­nies pay a fee for such a pre-mar­ket or pre-clin­i­cal re­view, though it’s un­clear at this stage what that fee would be or how such a re­view would be con­duct­ed.

“Some in­ter­na­tion­al health tech­nol­o­gy or­ga­ni­za­tions, such as NICE and CADTH, have of­fered this ‘ear­ly sci­en­tif­ic ad­vice’ to in­dus­try for many years with gen­er­al suc­cess. We are there­fore eval­u­at­ing the op­tions to pro­vide a sim­i­lar ser­vice but have not made any de­fin­i­tive plans,” Whitrap said.

Bern­stein biotech an­a­lyst Ron­ny Gal not­ed that the po­ten­tial move for ICER would be a fund­ing shift from their past re­liance on do­na­tions and con­fer­ence or­ga­niz­ing.

“We do not ques­tion ICER staff’s in­tegri­ty, but it is very hard to not grow to like your clients,” Gal said. “This fund­ing mech­a­nism is (re­spect­ful­ly) in­her­ent­ly prob­lem­at­ic for an or­ga­ni­za­tion whose mis­sion is to be­come the fair ar­biter of drug val­ue … It is in­cred­i­bly short­sight­ed of pay­ers (in­clud­ing and es­pe­cial­ly CMS) not to step up and fund ICER. An in­de­pen­dent eval­u­a­tor of drug val­ue is fi­nal­ly get­ting off the ground and you let it be­come fund­ed by Phar­ma?”

But Whitrap re­as­sured that if ICER does pur­sue such a pro­gram, “We would on­ly do so in a man­ner that would pre­serve ICER’s in­de­pen­dence and in­tegri­ty, and on­ly if of­fer­ing the ear­ly ad­vice to man­u­fac­tur­ers would fur­ther our core mis­sion of help­ing all pa­tients achieve ac­cess to high-val­ue care.”

Gal al­so called the idea “a pos­i­tive for the drug in­dus­try” as it will al­low drug de­vel­op­ers “to be the ones to ed­u­cate ICER ear­ly about the mer­it of their drug — they will get to com­pre­hen­sive­ly present their case and gain clar­i­ty as to what da­ta they need to put to­geth­er to ob­tain bet­ter prices.”

Whitrap added: “We’re still eval­u­at­ing all of our op­tions at this point re­gard­ing ear­ly sci­en­tif­ic ad­vice, in­clud­ing how to sup­port the staffing re­quire­ments it would en­tail … any po­ten­tial of­fer­ing in this area would be struc­tured in a way that would safe­guard the in­de­pen­dence of the drug as­sess­ments we con­duct at or around FDA ap­proval.”

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 for more in­for­ma­tion.


Zachary Brennan

managing editor, RAPS

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

Endpoints News

Basic subscription required

Unlock this story instantly and join 58,000+ biopharma pros reading Endpoints daily — and it's free.

Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.