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.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

Endpoints Premium

Premium subscription required

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

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Gilead bol­sters its case for block­buster hope­ful fil­go­tinib as FDA pon­ders its de­ci­sion

Before remdesivir soaked up the spotlight amid the coronavirus crisis, Gilead’s filgotinib was the star experimental drug tapped to rake in billions competing with other JAK inhibitors made by rivals including AbbVie and Eli Lilly.

Now, long term data on the drug — discovered by Gilead’s partners at Galapagos and posted as part of a virtual medical conference — have solidified the durability and safety of filgotinib in patients with rheumatoid arthritis, spanning data from three late-stage trials. An FDA decision on the drug is expected this year.

Joseph Kim, Inovio CEO (Andrew Harnik, AP Images)

Caught in a stand­off with its con­tract man­u­fac­tur­er over Covid-19 vac­cine, In­ovio files suit in an at­tempt to break free while ri­vals race ahead

Inovio was one of the first vaccine developers to snag attention for a jab that their execs said promised to end the Covid-19 pandemic. Using their own unique DNA tech, CEO Joseph Kim said it took just 3 hours to work it out.

But while rivals are racing to the finish line with ambitious plans to make vast quantities of their vaccines with billions of dollars of deals, Inovio is still stuck at the starting line on manufacturing.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 83,000+ biopharma pros reading Endpoints daily — and it's free.