For shame: ‘Phar­ma Bro’ Shkre­li is in prison, but Dara­prim’s price is still high

The move drew crit­i­cism from all cor­ners. Con­gress hauled Shkre­li in for ques­tion­ing on tele­vi­sion. Me­dia out­lets shamed the prac­tice. The Phar­ma­ceu­ti­cal Re­search and Man­u­fac­tur­ers of Amer­i­ca (PhRMA), the pow­er­ful trade group for brand­ed drugs, dis­tanced it­self, say­ing Tur­ing “does not rep­re­sent the val­ues of@PhRMA” and kicked off a cam­paign it de­scribed as “more lab coat, less hood­ie.”

Shkre­li, 35, is now serv­ing a sev­en-year prison term for se­cu­ri­ties fraud (un­re­lat­ed to Dara­prim). Tur­ing has re­named it­self Vy­era Phar­ma­ceu­ti­cals.

But Dara­prim, which costs pen­nies to make and is used to treat the par­a­sitic in­fec­tion tox­o­plas­mo­sis — which is rare in the Unit­ed States — still re­tails for more than $750 per pill, ac­cord­ing to drug web­site Vy­era did not re­spond to mul­ti­ple re­quests for com­ment.

The con­tin­ued high price of the drug is a cau­tion­ary tale to those who hope that pub­lic sham­ing of a few “bad ac­tors” can curb es­ca­lat­ing drug prices, be­cause the prob­lem is root­ed in the mar­ket’s un­der­ly­ing fi­nan­cial in­cen­tives.

Drug prices are “easy to raise and hard­er to low­er, par­tic­u­lar­ly if there’s no com­pe­ti­tion,” said Nichol­son Price, an as­sis­tant pro­fes­sor at the Uni­ver­si­ty of Michi­gan Law School. “The mys­tery isn’t, ‘Why don’t drug prices go down?’ It’s more, ‘Why don’t they go up more?’”

That’s es­pe­cial­ly the case with a prod­uct like Dara­prim, which ben­e­fits a rel­a­tive­ly small group of peo­ple — about 2,000 Amer­i­cans per year. That means less prof­it in­cen­tive for oth­er com­pa­nies to de­vel­op a com­peti­tor that could dri­ve down prices.

Joey Mat­ting­ly, an as­sis­tant pro­fes­sor at the Uni­ver­si­ty of Mary­land School of Phar­ma­cy, us­es Dara­prim as a case study in a uni­ver­si­ty course he teach­es on phar­ma­ceu­ti­cal busi­ness strat­e­gy, high­light­ing how the in­dus­try func­tions un­der cur­rent in­cen­tives.

“The mar­ket sort of sets it up where, if you need it, you have to pay for it,” he said. “A for-prof­it en­ti­ty is go­ing to raise the price.”

Brand­ed drugs like Dara­prim are more like­ly to be priced high with­out a clear jus­ti­fi­ca­tion, not­ed David Howard, a health econ­o­mist and pro­fes­sor at Emory Uni­ver­si­ty.

Dara­prim was first ap­proved by the Food and Drug Ad­min­is­tra­tion more than 50 years ago, and the patent has long since ex­pired for both the drug and its ac­tive in­gre­di­ent. But there’s no gener­ic equiv­a­lent in the Unit­ed States.

Even with gener­ic-drug com­pe­ti­tion, costs don’t al­ways drop. In 2015 alone, 300 gener­ic drugs — off-patent med­ica­tions, which are typ­i­cal­ly cheap to make — saw price in­creas­es of more than 100 per­cent, ac­cord­ing to a 2016 Gov­ern­ment Ac­count­abil­i­ty Of­fice re­port.

“We don’t have a good mod­el for pric­ing phar­ma­ceu­ti­cals in this coun­try and, as a re­sult, we keep spend­ing a lot more mon­ey,” Price said. “We avoid think­ing about it, or avoid deal­ing with it, and as a re­sult things get more prob­lem­at­ic.”

As prices climb, Vy­era has fol­lowed what has be­come a fa­mil­iar phar­ma­ceu­ti­cal play­book to shift at­ten­tion and costs, launch­ing what it calls the Dara­prim Di­rect pro­gram.

Com­mer­cial­ly in­sured pa­tients can get a com­pa­ny-spon­sored coupon that guar­an­tees they’ll pay no more than $10 out-of-pock­et. Unin­sured pa­tients at 500 per­cent or less of the fed­er­al pover­ty lev­el — about $82,300 for a fam­i­ly of two — won’t pay any­thing.

Peo­ple with Medicare Part D cov­er­age can ap­ply for co­pay as­sis­tance from an “in­de­pen­dent char­i­ta­ble foun­da­tion” to which Vy­era has do­nat­ed mon­ey. This op­tion is list­ed on the Dara­prim Di­rect web­site. Tech­ni­cal­ly, Medicare ben­e­fi­cia­ries can­not use com­pa­ny coupons, but many drug com­pa­nies skirt that reg­u­la­tion by send­ing as­sis­tance through a sep­a­rate in­ter­me­di­ary — such as an in­de­pen­dent char­i­ty. It’s com­mon enough that the prac­tice has re­cent­ly come un­der fed­er­al scruti­ny.

Crit­ics are quick to point out that such pro­grams — of­ten de­ployed for high-priced drugs — may en­able pa­tient ac­cess but do noth­ing to ad­dress over­all ex­pense. Pri­vate in­sur­ers, Medicare or Med­ic­aid must pay the tab, whether through in­creased pre­mi­ums or strained pub­lic health bud­gets.

On av­er­age, Med­ic­aid pro­grams in 2017 paid $35,556.48 per Dara­prim pre­scrip­tion, ac­cord­ing to a Kaiser Health News analy­sis of fed­er­al da­ta cov­er­ing that year’s first three quar­ters.

That fig­ure doesn’t ac­count for any re­bates state Med­ic­aid pro­grams like­ly re­ceive from Vy­era, which is undis­closed pro­pri­etary in­for­ma­tion. In Mass­a­chu­setts, those deals mean the state’s net costs for Dara­prim have re­mained large­ly un­changed since 2014, though the price tag is 75 times what it was, said a spokes­woman for the agency’s Ex­ec­u­tive Of­fice of Health and Hu­man Ser­vices.

But states have vari­able ne­go­ti­at­ing lever­age and skills in press­ing for dis­counts. And pay­ing for high-cost drugs — es­pe­cial­ly those with­out a com­peti­tor — re­mains a se­ri­ous chal­lenge, she said.

Gen­er­al­ly, Med­ic­aid like­ly pays hun­dreds of dol­lars per Dara­prim pill, said Matt Sa­lo, ex­ec­u­tive di­rec­tor of the Na­tion­al As­so­ci­a­tion of Med­ic­aid Di­rec­tors. A stan­dard start­ing dose of two to three pills per day lasts one to three weeks. And that’s like­ly to gen­er­ate costs much high­er than they were be­fore Shkre­li start­ed sell­ing Dara­prim.

By She­fali Luthra with con­tri­bu­tion from Syd­ney Lup­kin. Orig­i­nal­ly post­ed at Kaiser Health News, a na­tion­al health pol­i­cy news ser­vice that is part of the non­par­ti­san Hen­ry J Kaiser Fam­i­ly Foun­da­tion.

Note: To de­ter­mine what Med­ic­aid paid for Dara­prim, Kaiser Health News used da­ta made pub­lic by the Cen­ters for Medicare & Med­ic­aid Ser­vices. This fig­ure rep­re­sents a weight­ed av­er­age of Med­ic­aid pay­ments per pre­scrip­tion, across var­i­ous strengths, pack­age sizes, routes and la­bels. It does not in­clude drug ver­sions (rep­re­sent­ed by Na­tion­al Drug Codes) with few­er than 11 pre­scrip­tion fills per quar­ter.

Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Why would the FDA ap­prove an­oth­er con­tro­ver­sial drug to spur a woman’s li­bido with these da­ta? And why no ex­pert pan­el re­view?

AMAG Pharmaceuticals’ newly approved drug for spurring women’s sexual desire may never make much money, but it’s a big hit at sparking media attention.

The therapy — Vyleesi (bremelanotide) — got the green light from regulators on Friday evening, swiftly lighting up a range of stories around the world, from The New York Times to The Guardian. Several headlines inevitably referred to it as the “female Viagra,” invoking Pfizer’s old erectile dysfunction blockbuster.

But the two drugs have little in common.

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Alex­ion wins pri­or­i­ty re­view for Ul­tomiris' aHUS in­di­ca­tion; FDA ex­pands ap­proval of Ver­tex's Symdeko

→ Alex­ion $ALXN has scored a speedy re­view for Ul­tomiris for pa­tients with atyp­i­cal he­molyt­ic ure­mic syn­drome (aHUS) af­ter post­ing pos­i­tive da­ta from a piv­otal study in Jan­u­ary. The drug is the rare dis­ease com­pa­ny’s shot at pro­tect­ing its block­buster blood dis­or­der fran­chise that is cur­rent­ly cen­tered around its flag­ship drug, Soliris, which is a com­ple­ment in­hibitor typ­i­cal­ly ad­min­is­tered every two weeks. Ul­tomiris has a sim­i­lar mech­a­nism of ac­tion but re­quires less-fre­quent dos­ing — every eight weeks. The de­ci­sion date has been set to Oc­to­ber 19. Late last year, Ul­tomiris se­cured ap­proval for noc­tur­nal he­mo­glo­bin­uria (PNH) pa­tients.

Bet­ter than Am­bi­en? Min­er­va soars on PhI­Ib up­date on sel­torex­ant for in­som­nia

A month af­ter roil­ing in­vestors with what skep­tics dis­missed as cher­ry pick­ing of its de­pres­sion da­ta, Min­er­va is back with a clean slate of da­ta from its Phase IIb in­som­nia tri­al.

In a de­tailed up­date, the Waltham, MA-based biotech said sel­torex­ant (MIN-202) hit both the pri­ma­ry and sev­er­al sec­ondary end­points, ef­fec­tive­ly im­prov­ing sleep in­duc­tion and pro­long­ing sleep du­ra­tion. In­ves­ti­ga­tors made a point to note that the ef­fects were con­sis­tent across the adult and el­der­ly pop­u­la­tions, with the lat­ter more prone to the sleep dis­or­der.

Gene ther­a­py biotech sees its stock rock­et high­er on promis­ing re­sults for rare cas­es of but­ter­fly dis­ease

Shares of Krys­tal Biotech took off this morn­ing $KRYS af­ter the lit­tle biotech re­port­ed promis­ing re­sults from its gene ther­a­py to treat a rare skin dis­ease called epi­der­mol­y­sis bul­losa.

Fo­cus­ing on an up­date with 4 new pa­tients, re­searchers spot­light­ed the suc­cess of KB103 in clos­ing some stub­born wounds. Krys­tal says that of 4 re­cur­ring and 2 chron­ic skin wounds treat­ed with the gene ther­a­py, the KB103 group saw the clo­sure of 5. The 6th — a chron­ic wound, de­fined as a wound that had re­mained open for more than 12 weeks — was par­tial­ly closed. That brings the to­tal so far to 8 treat­ed wounds, with 7 clo­sures.

UP­DAT­ED: In sur­prise switch, Bris­tol-My­ers is sell­ing off block­buster Ote­zla, promis­ing to com­plete Cel­gene ac­qui­si­tion — just lat­er

Apart from revealing its checkpoint inhibitor Opdivo blew a big liver cancer study on Monday, Bristol-Myers Squibb said its plans to swallow Celgene will require the sale of blockbuster psoriasis treatment Otezla to keep the Federal Trade Commission (FTC) at bay.

The announcement — which has potentially delayed the completion of the takeover to early 2020 — irked investors, triggering the New York-based drugmaker’s shares to tumble Monday morning in premarket trading.

Celgene’s Otezla, approved in 2014 for psoriasis and psoriatic arthritis, is a rising star. It generated global sales of $1.6 billion last year, up from the nearly $1.3 billion in 2017. Apart from the partial overlap of Bristol-Myers injectable Orencia, the company’s rival oral TYK2 psoriasis drug is in late-stage development, after the firm posted encouraging mid-stage data on the drug, BMS-986165, last fall. With Monday’s decision, it appears Bristol-Myers is favoring its experimental drug, and discounting Otezla’s future.

The move blindsided some analysts. Credit Suisse’s Vamil Divan noted just days ago:

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Bris­tol-My­ers star Op­di­vo fails sur­vival test in a matchup with Nex­avar aimed at shak­ing up the big HCC mar­ket

Bris­tol-My­ers Squibb has suf­fered an­oth­er painful set­back in its years-long quest to ex­pand the reach of Op­di­vo. The phar­ma gi­ant this morn­ing not­ed that their Check­mate-459 study com­par­ing Op­di­vo with Bay­er’s Nex­avar in front­line cas­es of he­pa­to­cel­lu­lar car­ci­no­ma — the most com­mon form of liv­er can­cer — failed to hit the pri­ma­ry end­point on over­all sur­vival.

This was a sig­nif­i­cant mile­stone in Bris­tol-My­ers’ tal­ly of PD-1 cat­a­lysts this year. Nex­avar (so­rafenib) has been the stan­dard of care in front­line HCC for the past decade, though Op­di­vo has been mak­ing head­way in sec­ond-line HCC cas­es, where it’s go­ing toe-to-toe with Bay­er’s Sti­var­ga (re­go­rafenib) af­ter re­cent ap­provals shook up the mar­ket.

Ab­b­Vie gets a green light to re­sume re­cruit­ing pa­tients for one myelo­ma study — but Ven­clex­ta re­mains un­der a cloud

Three months af­ter reg­u­la­tors at the FDA forced Ab­b­Vie to halt en­rolling pa­tients in its tri­als of a com­bi­na­tion us­ing Ven­clex­ta (vene­to­clax) to treat drug-re­sis­tant cas­es of mul­ti­ple myelo­ma, the agency has green-light­ed the re­sump­tion of one of those stud­ies, while keep­ing the rest on the side­lines.

The CANO­VA (M13-494) study can now get back in busi­ness re­cruit­ing pa­tients to test the drug for a pop­u­la­tion that shares a par­tic­u­lar ge­net­ic bio­mark­er. To get that per­mis­sion, Ab­b­Vie — which is part­nered with Roche on this pro­gram — was forced to re­vise the pro­to­col, mak­ing un­spec­i­fied changes in­volv­ing risk mit­i­ga­tion mea­sures, pro­to­col-spec­i­fied guide­lines and an up­dat­ed fu­til­i­ty cri­te­ria.

Mike Grey. Mirum

In $86M IPO pitch, Mirum spells out plans to turn Shire dis­cards in­to or­phan liv­er drug suc­cess­es

Mike Grey doesn’t have any time to waste. Hav­ing re­gained con­trol of two liv­er dis­ease drugs from Shire and po­si­tioned them for piv­otal stud­ies — five years af­ter first hand­ing them off in a deal to sell Lu­me­na, where he was CEO — Grey is steer­ing Mirum straight in­to an IPO with a $86 mil­lion ask.

Not that Mirum has spent much of its $120 mil­lion Se­ries A cash since launch­ing last No­vem­ber. Ac­cord­ing to the S-1, the Cal­i­forn­ian biotech has burned through $23.3 mil­lion as of March, but ex­pects ex­pens­es to pick up once their clin­i­cal work gath­ers steam.