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.”

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. The idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.

Ver­tex deal for Scot­land — no deal for Eng­land

Cystic fibrosis (CF) drug maker Vertex Pharmaceuticals — which is still locked in negotiation with NHS England to endorse the use of its medicines — has successfully negotiated a deal with Scottish authorities.

A month ago, the Scottish Medicines Consortium spurned two of the company’s medicines — Orkambi and Symkevi — citing uncertainty over their long-term efficacy in relation to their cost.

Tony Kulesa, Brian Baynes. Petri

Star founders, in­ves­ti­ga­tors hud­dle around new Boston ac­cel­er­a­tor spot­light­ing young en­tre­pre­neurs

As a widely recognized biotech hub, Boston is undoubtedly one of the best places to start a new company at the frontier of biology and engineering. With a dense network of incubators, venture capitalists and talent, seasoned company founders can have their pick of partners and models launching their latest startups.

But for young, aspiring entrepreneurs, it’s a very different scene.

Big VC firms might hire you to work on their ideas instead of yours, and accelerators may not offer the kind of deep technical expertise and guidance needed to make it in the field.

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From part­ner to knight in shin­ing ar­mor: Cas­tle Creek to buy Fi­bro­cell

In April, Castle Creek swooped in to partner with the embattled gene and cell therapy Fibrocell to shepherd its lead gene therapy for a type of “butterfly” disease into late-stage development. Now, the New Jersey-based dermatology company is acquiring its partner in a deal worth $63.3 million.

Pennsylvania-based Fibrocell last year initiated a review of strategic alternatives, including a sale.