There’s one end­point that the boom­ing bio­phar­ma in­dus­try has failed at mis­er­ably: fi­nan­cial tox­i­c­i­ty

Bioreg­num Opin­ion Col­umn by John Car­roll

One of the big themes in R&D over the past few years has been the on­slaught of spend­ing on de­vel­op­ing new on­col­o­gy drugs. The FDA has en­cour­aged ear­ly ap­provals, open­ing the door to small­er tri­als as an on­slaught of in­vest­ment cash made it pos­si­ble for small play­ers to go the dis­tance.

Big Phar­ma, mean­while, has en­joyed the com­fort of bet­ter sci­en­tif­ic in­sights and the ar­rival of some huge new PD-1s on the scene. Next up: A tsuna­mi of com­bo ther­a­pies com­ing at you from the US, Eu­rope and Chi­na — the new fac­tor in drug hunt­ing.

For the in­dus­try, that means a ma­jor new source of rev­enue from com­ing ther­a­pies. For US pa­tients, that means a much bet­ter shot at longer sur­vival and pos­si­bly even a cure. As well as bank­rupt­cy.

Wait. What?

Re­searchers just pub­lished a new study in The Amer­i­can Jour­nal of Med­i­cine high­light­ing that 42% of can­cer pa­tients ex­haust their sav­ings with­in 2 years of di­ag­no­sis. And 62% of the 9.5 mil­lion can­cer pa­tients they re­viewed were in debt af­ter ther­a­py, with 40% to 85% quit­ting work due to can­cer. Af­ter 4 years of ther­a­py, 38.2% were in­sol­vent.

You hear a lot every time a new drug is ap­proved about what drug com­pa­nies are do­ing to make their ther­a­pies ac­ces­si­ble, but the sim­ple fact is that in the US large per­cent­ages of pa­tients are be­ing crushed by the price of brand­ed drugs. And while the new drugs be­ing in­tro­duced may be more im­por­tant than ever, the un­var­nished truth is that ba­sic pric­ing strate­gies are more about max­i­miz­ing rev­enue than ac­com­mo­dat­ing pa­tients.

The re­sult­ing fi­nan­cial tox­i­c­i­ty is enor­mous.

Let’s re­mem­ber that one of the rea­sons we’re see­ing all the in­vest­ment cash pour­ing in is that Wall Street has em­braced a big wave of biotech IPOs. And that’s where ex­ecs are fo­cused when they price new drugs.

That’s not a wild guess, ei­ther.

This is the bot­tom line re­searchers tot­ted up af­ter dis­cussing pric­ing strate­gies on new drugs for mul­ti­ple scle­ro­sis with 4 biotech ex­ecs, pub­lished in Neu­rol­o­gy this week.

Par­tic­i­pants con­sis­tent­ly stat­ed that ini­tial price de­ci­sions were dic­tat­ed by the price of ex­ist­ing com­peti­tors in the mar­ket. Rev­enue max­i­miza­tion and cor­po­rate growth were dri­vers of price es­ca­la­tions in the ab­sence of con­tin­ued mar­ket pen­e­tra­tion. Low­er rev­enue pre­dic­tions out­side the Unit­ed States al­so in­formed pric­ing strate­gies. The grow­ing com­plex­i­ty and clout of drug dis­tri­b­u­tion and sup­ply chan­nels were al­so cit­ed as con­tribut­ing fac­tors. Al­though de­ci­sions to raise prices were mo­ti­vat­ed by the need to at­tract in­vest­ment for fu­ture in­no­va­tion, re­coup­ing drug-spe­cif­ic re­search and de­vel­op­ment costs as a jus­ti­fi­ca­tion was not strong­ly en­dorsed as hav­ing a sig­nif­i­cant in­flu­ence on pric­ing de­ci­sions.

So while the in­dus­try likes to talk a lot about the pric­ing lev­els need­ed to back in­no­va­tion, it’s just not an ac­cu­rate re­flec­tion of re­al­i­ty.

In just a few weeks, we’re go­ing to wake up to a new year that will be dom­i­nat­ed by drug pric­ing dis­cus­sions. We’re go­ing to be do­ing some of this our­selves at JP Mor­gan.

The in­dus­try still has a shot at com­ing up with some kind of work­able re­form on drug prices. Bar­ring a mar­ket so­lu­tion, though, you can ex­pect plen­ty of un­work­able and de­struc­tive sug­ges­tions on drug im­por­ta­tion and com­pul­so­ry li­cens­ing and more. And some­day, law­mak­ers will do some­thing about it.

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Stephen Hahn, AP

The FDA has de­val­ued the gold stan­dard on R&D. And that threat­ens every­one in drug de­vel­op­ment

Bioregnum Opinion Column by John Carroll

A few weeks ago, when Stephen Hahn was being lightly queried by Senators in his confirmation hearing as the new commissioner of the FDA, he made the usual vow to maintain the gold standard in drug development.

Neatly summarized, that standard requires the agency to sign off on clinical data — usually from two, well-controlled human studies — that prove a drug’s benefit outweighs any risks.

Over the last few years, biopharma has enjoyed an unprecedented loosening over just what it takes to clear that bar. Regulators are more willing to drop the second trial requirement ahead of an accelerated approval — particularly if they have an unmet medical need where patients are clamoring for a therapy.

That confirmatory trial the FDA demands can wait a few years. And most everyone in biopharma would tell you that’s the right thing for patients. They know its a tonic for everyone in the industry faced with pushing a drug through clinical development. And it’s helped inspire a global biotech boom.

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UP­DAT­ED: New play­ers are jump­ing in­to the scram­ble to de­vel­op a vac­cine as pan­dem­ic pan­ic spreads fast

When the CNN news crew in Wuhan caught wind of the Chinese government’s plan to quarantine the city of 11 million people, they made a run for one of the last trains out — their Atlanta colleagues urging them on. On the way to the train station, they were forced to skirt the local seafood market, where the coronavirus at the heart of a brewing outbreak may have taken root.

And they breathlessly reported every moment of the early morning dash.

In shuttering the city, triggering an exodus of masked residents who caught wind of the quarantine ahead of time, China signaled that they were prepared to take extreme actions to stop the spread of a virus that has claimed 17 lives, sickened many more and panicked people around the globe.

CNN helped illustrate how hard all that can be.

The early reaction in the biotech industry has been classic, with small-cap companies scrambling to headline efforts to step in fast. But there are also new players in the field with new tech that has been introduced since the last of a series of pandemic panics that could change the usual storylines. And they’re volunteering for a crash course in speeding up vaccine development — a field where overnight solutions have been impossible to prove.

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Mer­ck KGaA spin­out gets first fund­ing to bring dual-act­ing can­cer mol­e­cules in­to the clin­ic

Two and a half years after launch, Merck KGaA spinout iOnctura is getting its first major round of funding.

The oncology startup raised €15 million ($16.6 million) to put its lead drug into the clinic and get its second drug past IND-enabling tests. INKEF Capital and VI Partners co-led the round and were joined by the biotech’s longtime backer M Ventures, an arm of Merck KGaA, and Schroder Adveq.

UP­DAT­ED: Eli Lil­ly’s $1.6B can­cer drug failed to spark even the slight­est pos­i­tive gain for pa­tients in its 1st PhI­II

Eli Lilly had high hopes for its pegylated IL-10 drug pegilodecakin when it bought Armo last year for $1.6 billion in cash. But after reporting a few months ago that it had failed a Phase III in pancreatic cancer, without the data, its likely value has plunged. And now we’re getting some exact data that underscore just how little positive effect it had.

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Am­gen aug­ments Asia foothold by tak­ing over Astel­las joint ven­ture in Japan

California-based Amgen, which does the bulk of its business in the United States, made its ambition to reinvigorate its growth prospects by expanding its presence in Asia clear at the sidelines of the JP Morgan healthcare conference in San Francisco earlier this month.

The Thousand Oaks-based company on Thursday executed its plan to dissolve the joint venture with Astellas — created in 2013 — to operate the unit independently in Japan. With its rapidly aging population, the region represents an appealing market for Amgen’s osteoporosis treatments Prolia and Evenity as well as a cholesterol-lowering injection Repatha.

Daphne Zohar (PureTech)

PureTech bags $200M from sale of Karuna shares — still siz­zling from promis­ing schiz­o­phre­nia da­ta

Cashing in on the exuberance around Karuna Therapeutics and its potential blockbuster CNS drug, PureTech has sold a chunk of the biotech’s shares to Goldman Sachs for $200 million.

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