Can Am­gen beat down pay­er re­sis­tance to Repatha with its own cost analy­sis?

PC­SK9 heart drugs have proven to be one of the biggest com­mer­cial dis­ap­point­ments in re­cent bio­phar­ma times. Held back by pay­ers who don’t see the val­ue in pro­vid­ing the drugs even at a hefty 30% or so dis­count on the list price, sales of Am­gen’s Repatha and Pralu­ent from Re­gen­eron/Sanofi have lan­guished — fur­ther ham­pered by cost analy­sis stud­ies that as­cribe a rel­a­tive­ly small val­ue to the drugs.

So now, in a wide­ly an­tic­i­pat­ed study of its own, Am­gen is lay­ing out the eco­nom­ic ar­gu­ment to jus­ti­fy its $14,523 list price of Repatha (evolocum­ab).

In what is bound to be quick­ly la­beled as a self-serv­ing analy­sis, Am­gen in­sists that if most pay­ers are re­fus­ing to pro­vide Repatha at less than $10,000 a year with the dis­count, many of their mem­bers may well be los­ing out.

Gregg Fonarow

To get to that con­clu­sion, the promi­nent in­ves­ti­ga­tors en­list­ed for this study used re­al-world num­bers on high-risk pa­tients, peg­ging it to qual­i­ty ad­just­ed life years (QALY), a con­tro­ver­sial, vari­able and com­mon­ly used mea­sure of as­sess­ment. They al­so not­ed that stick­ing with the pa­tient pop­u­la­tion re­cruit­ed for Am­gen’s big FOURI­ER study, the dis­count off the list price would need to be more than 50%.

That FOURI­ER study was sup­posed to be Am­gen’s tick­et to the block­buster sales that the lead­ers in this field feel they are due drugs that can sig­nif­i­cant­ly re­duce the risk of ma­jor car­dio events. In­stead, the lack of a mor­tal­i­ty ben­e­fit — fail­ing to sig­nif­i­cant­ly re­duce the risk of death — helped prompt pay­ers to hold fast with treat­ment guide­lines that con­tin­ue to de­ny cov­er­age to the vast ma­jor­i­ty of pa­tients in this huge mar­ket.

FOURI­ER is now used to demon­strate why Am­gen’s price should be far low­er than it is. In a study pub­lished in the Jour­nal of the Amer­i­can Med­ical As­so­ci­a­tion ear­li­er this week, re­searchers con­clud­ed that drug is worth no more than $4,536 a year, less than a third of its cur­rent list price.

Am­gen — which has been work­ing out mon­ey-back guar­an­tees in the event of a heart at­tack — dis­putes those num­bers. Giv­en pay­ers’ tren­chant at­ti­tudes, and the range of cost-jus­ti­fi­ca­tion re­ports now avail­able, their chance of suc­cess ap­pears lim­it­ed.

That pay­er kick­back is a key con­cern for every­one in drug de­vel­op­ment, where the opaque drug pric­ing sys­tem in the US — in­flat­ed list prices and un­cer­tain net amounts tend to cre­ate more con­fu­sion than clar­i­ty — is be­dev­il­ing the fu­ture of many drugs now in de­vel­op­ment. Get­ting the da­ta need­ed for an ap­proval is no guar­an­tee of com­mer­cial suc­cess, and that is a key is­sue that will bear in­creas­ing in­flu­ence in Big Phar­ma’s de­ci­sions about what it wants in the pipeline.

Am­gen’s as­sess­ment in­cludes this bot­tom line as­sess­ment of the val­ue of a drug that slash­es LDL by about 60% on av­er­age.

Sev­er­al clin­i­cal sce­nar­ios were ex­plored to de­ter­mine whether Repatha is worth the in­vest­ment, in­clud­ing event rates seen in re­al world clin­i­cal prac­tice set­tings, as well as event rates seen in the ac­tu­al out­comes tri­al, and in pa­tients who have high­er base­line LDL lev­els. The re­searchers de­ter­mined that to re­main be­low gen­er­al­ly ac­cept­ed cost-ef­fec­tive­ness thresh­olds in a typ­i­cal U.S. AS­CVD pop­u­la­tion ($150,000 per qual­i­ty ad­just­ed life year (QALY)), the an­nu­al net price for Repatha would need to be at or be­low $9,669. Tar­get­ing the more se­vere clin­i­cal prac­tice pop­u­la­tion with base­line LDL lev­els ≥100 mg/dL de­spite max­i­mal in­ten­si­ty statin ther­a­py re­veals that an an­nu­al net price of $13,225 is cost-ef­fec­tive. In a pop­u­la­tion with event rates seen in the Repatha out­comes study, $6,780 is cost-ef­fec­tive.

“This is the first cost-ef­fec­tive­ness as­sess­ment of evolocum­ab us­ing a mod­el based on a high- qual­i­ty out­comes tri­al, com­bined with U.S. clin­i­cal prac­tice da­ta. The analy­sis iden­ti­fies the types of high-risk pa­tients for whom this ther­a­py is both clin­i­cal­ly ben­e­fi­cial and cost-ef­fec­tive,” said Gregg Fonarow, pro­fes­sor of med­i­cine at UCLA David Gef­fen School of Med­i­cine and the study’s prin­ci­pal au­thor. “This study pro­vides a crit­i­cal in­put to the over­all cost-ef­fec­tive­ness de­bate that has sur­round­ed PC­SK9 in­hibitors.”

The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Pablo Legorreta, founder and CEO of Royalty Pharma AG, speaks at the annual Milken Institute Global Conference in Beverly Hills, California (Patrick T. Fallon/Bloomberg via Getty Images)

Cap­i­tal­iz­ing Pablo: The world’s biggest drug roy­al­ty buy­er is go­ing pub­lic. And the low-key CEO di­vulges a few se­crets along the way

Pablo Legorreta is one of the most influential players in biopharma you likely never heard of.

Over the last 24 years, Legorreta’s Royalty Pharma group has become, by its own reckoning, the biggest buyer of drug royalties in the world. The CEO and founder has bought up a stake in a lengthy list of the world’s biggest drug franchises, spending $18 billion in the process — $2.2 billion last year alone. And he’s become one of the best-paid execs in the industry, reaping $28 million from the cash flow last year while reserving 20% of the cash flow, less expenses, for himself.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,800+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,800+ biopharma pros reading Endpoints daily — and it's free.

Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,800+ biopharma pros reading Endpoints daily — and it's free.

As­traZeneca’s $7B ADC suc­ceeds where Roche failed, im­prov­ing sur­vival in gas­tric can­cer

Another day, another win for Enhertu.

The antibody-drug conjugate AstraZeneca promised up-to $7 billion to partner on has had a quite a few months, beginning with splashy results in a Phase II breast cancer trial, a rapid approval and, earlier this month, breakthrough designations in both non-small cell lung cancer and gastric cancer.

Now, at ASCO, the British pharma and their Japanese partner, Daiichi Sankyo, have shown off the data that led to the gastric cancer designation, which they’ll take back to the FDA. In a pivotal, 187-person Phase II trial, Enhertu shrunk tumors in 42.9% of third-line patients with HER2-positive stomach cancer, compared with 12.5% in a control arm where doctors prescribed their choice of therapy. Progression-free survival was 5.4 months for Enhertu compared to 3.5 months for the control.

Once a gem, now just a rock, Take­da punts PhI­II IBD drug as ri­vals mus­cle ahead

Back in 2016, when then-Shire CEO Flemming Ørnskov picked up a promising clinical-stage IBD drug from Pfizer, the Boston-based biotech dubbed it SHP647 and moved it into the gem section of the pipeline, with rosy expectations of registration-worthy Phase III data ahead.

This was a drug that the EC wanted Takeda to commit to selling off before it gave their blessing to its acquisition of Shire, to settle some deep-seated concerns revolving around the potential market overlap with their blockbuster rival Entyvio. And Takeda, which took on a heavy debt load to buy Shire, clearly wanted the cash to pay down debt.

David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,800+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er, Mer­ck KGaA ce­ment Baven­cio blad­der can­cer win with OS da­ta — while carv­ing an­oth­er niche in rare can­cer

Pfizer and Merck KGaA have detailed the Phase III data that inspired FDA regulators to designate Bavencio a “breakthrough” for first-line advanced bladder cancer and offered an early glance at how the PD-L1 can help patients with a rare gynecological cancer — carving out niches in the checkpoint space for itself after being shut out of numerous others.

In JAVELIN Bladder 100, Bavencio led to a 31% reduction in risk of death compared to standard care alone. It also extended median survival by more than seven months — a historic feat in this setting, according to investigators at Queen Mary University of London.

Sanofi brings in 4 new ex­ec­u­tives in con­tin­ued shake-up, as vac­cines and con­sumer health chief head out the door

In the middle of Sanofi’s multi-pronged race to develop a Covid-19 vaccine, David Loew, the head of their sprawling vaccines unit, is leaving – part of the final flurry of moves in the French giant’ months-long corporate shuffle that will give them new-look leadership under new CEO Paul Hudson.

The company also said today that Alan Main, the head of their consumer healthcare unit, is out, and they named 4 executives to fill new or newly vacated positions, 3 of whom come from both outside both Sanofi and from Pharma.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 81,800+ biopharma pros reading Endpoints daily — and it's free.