As a promi­nent an­a­lyst pre­dicts (even­tu­al) drug price con­trols, bio­phar­ma braces for a blow while Trump balls his fist

Some­time this week, no one is sure when, Pres­i­dent Don­ald Trump is ex­pect­ed to give his long-await­ed speech on drug pric­ing. And a long line­up of key play­ers in the field has been scratch­ing their heads, anx­ious­ly try­ing to de­ter­mine just how far the na­tion’s most un­pre­dictable pres­i­dent could go.

Trump, of course, has been free with his pop­ulist brand of an­ti-in­dus­try rhetoric for more than a year now, af­ter es­tab­lish­ing the base­line with re­peat­ed as­ser­tions that phar­ma com­pa­nies have been get­ting away with mur­der for years. 

But there’s been lit­tle di­rect ac­tion, be­yond the work at the FDA to hus­tle up new gener­ic ap­provals while try­ing to use the pub­lic spot­light to whip up some fresh com­pe­ti­tion for the price gougers. 

FDA com­mis­sion Scott Got­tlieb like­ly tipped the ad­min­is­tra­tion’s hand on one point in a pre­view he of­fered on pric­ing last week, in­di­cat­ing that in­dus­try prac­tices in fight­ing a de­lay­ing ac­tion against gener­ics and biosim­i­lars will face some di­rect chal­lenges. But while sig­nif­i­cant, that by it­self won’t bend any longterm trends on pric­ing in fa­vor of pay­ers.

Mean­while, pub­lic anger over drug pric­ing is re­peat­ed­ly stoked with an un­end­ing se­ries of high pro­file as­saults on cer­tain play­ers that have used their pric­ing pow­er to jack up rev­enue from old drugs.

Just last night, Mallinck­rodt was back in the spot­light with Ac­thar, which has gone from $40 a vial in 2001 to $40,000, threat­en­ing the ba­sic po­lice and fire ser­vices pro­vid­ed by the town of Rock­ford, IL.

For­mer Rock­ford May­or Lar­ry Mor­ris­sey set the stage for a 60 Min­utes seg­ment with this quote: “‘Why is health­care so ex­pen­sive? Be­cause the fix is in. That’s the an­swer. That’s the short an­swer.”

And it’s not the kind of an­swer bio­phar­ma wants to hear right now, es­pe­cial­ly on 60 Min­utes.

The in­dus­try is still dis­grun­tled over the ad­min­is­tra­tion’s de­ci­sion to get phar­ma to cov­er part of the donut hole in Medicare drug prices. But they can live with that. There’s been a steady fo­cus at PhRMA on try­ing to shift the fo­cus from the man­u­fac­tur­ers to the in­sur­ers and PBMs that man­age the ben­e­fit. But it hasn’t worked. And re­cent­ly that tac­tic has pro­duced some painful­ly awk­ward sal­lies on so­cial me­dia that tends to at­tract as much spon­ta­neous laugh­ter as dis­cus­sion.

With pub­lic anger per­co­lat­ing at an un­prece­dent­ed rate — a ma­jor­i­ty told Kaiser Health that re­duc­ing drug pric­ing should be a top pri­or­i­ty of pol­i­cy mak­ers — some an­a­lysts be­lieve that what­ev­er Trump comes up with it won’t be enough to stop the one strat­e­gy that would make the bio­phar­ma in­dus­try reel: Price con­trols.

Se­nior Wells Far­go an­a­lyst David Maris put it in blunt terms a few days ago.

We be­lieve the US is on a longer-term arc to­ward price con­trols and low­er mar­gins, as there is an up­ris­ing that has been per­co­lat­ing for years against high drug prices. While the caus­es are many and the prob­lem much more com­pli­cat­ed and nu­anced than many oth­ers would lead read­ers to be­lieve, there is a re­bel­lion at hand. Ar­gu­ments about the cost to de­vel­op a drug or how oth­er sup­ply chain con­stituents are to blame are not ap­pre­ci­at­ed by the par­ent stand­ing at a phar­ma­cy counter aban­don­ing their child’s pre­scrip­tion be­cause they can­not af­ford it due to an out­ra­geous de­ducible or high co-pay. And ex­am­ples of un­con­scionable drug price in­creas­es have fur­ther hard­ened many con­sumer opin­ions. 

That day prob­a­bly won’t ar­rive this week. But if that’s the path we’re on, Trump is faced with ei­ther sat­is­fy­ing the anger with some grand com­pro­mis­es, or stok­ing it by pulling his punch­es.


Im­age: Pres­i­dent Don­ald Trump at a speech in Oc­to­ber, 2017. AP IM­AGES

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.

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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.

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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.

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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 help@endpointsnews.com with any issues.

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Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

BeiGene and Mus­tang nail down spe­cial FDA sta­tus for top drugs; Roche bags added cov­er­age for Hem­li­bra

→ BeiGene $BGNE is getting a boost in its drive to field a rival to Imbruvica. The FDA has offered an accelerated review to zanubrutinib, a BTK inhibitor that has posted positive results for mantle cell lymphoma. The PDUFA date lands on February 27, 2020. The drug scored breakthrough status at the beginning of the year.

→ BeiGene isn’t the only biopharma company to gain special regulatory status today. Mustang Bio $MBIO and St. Jude Children’s Research Hospital announced that MB-107, a lentiviral gene therapy for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, has been granted Regenerative Medicine Advanced Therapy status.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.

Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.