CMS kicks off 9-month process on whether to lim­it cov­er­age to Bio­gen's new Alzheimer's drug

The Cen­ters for Medicare & Med­ic­aid Ser­vices late Mon­day opened a for­mal process, rarely done for new ther­a­peu­tics, to care­ful­ly re­view whether it will lim­it cov­er­age na­tion­wide for mon­o­clon­al an­ti­bod­ies tar­get­ing amy­loid for the treat­ment of Alzheimer’s dis­ease, in­clud­ing Bio­gen’s new­ly ap­proved Aduhelm.

While it re­mains un­like­ly that CMS won’t end up pay­ing for Aduhelm (Medicare of­fi­cial­ly draws the line at care that is “not rea­son­able and nec­es­sary,” but CMS de­nies on­ly about 3% of claims sub­mit­ted by hos­pi­tals and physi­cians), the stakes are high as Bio­gen ex­pects that about 80% of pa­tients el­i­gi­ble for Aduhelm are cov­ered by Medicare, and pri­vate in­sur­ers are like­ly to fol­low the fed­er­al gov­ern­ment’s lead.

Al­though CMS main­tains that the cost of the treat­ment will not fac­tor in­to its fi­nal de­ci­sion, it may be dif­fi­cult for the agency to avoid dis­cussing the reper­cus­sions of the $56,000 price tag for Bio­gen’s drug, con­sid­er­ing how quick­ly it could push Medicare in­to in­sol­ven­cy if Aduhelm is used wide­ly.

A pro­posed de­ci­sion from CMS is due in 6 months, with a fi­nal one com­ing 3 months lat­er, and CMS says it will re­view the clin­i­cal ev­i­dence such as pub­lished clin­i­cal stud­ies (of which there aren’t any for Aduhelm), pro­fes­sion­al so­ci­ety guide­lines, and pub­lic com­ments (a pair of 2-hour lis­ten­ing ses­sions are slat­ed for lat­er this month) to make its de­ter­mi­na­tion.

CMS is so­lic­it­ing pub­lic com­ments that in­clude sci­en­tif­ic ev­i­dence and that ad­dress the fol­low­ing ques­tions re­gard­ing the treat­ment of in­di­vid­u­als with Alzheimer’s dis­ease with mon­o­clon­al an­ti­bod­ies di­rect­ed against amy­loid:

  1. Which health out­comes are im­por­tant, and what de­gree of im­prove­ment in them is mean­ing­ful for pa­tients re­ceiv­ing treat­ment?
  2. What char­ac­ter­is­tics of pa­tients with Alzheimer’s dis­ease are im­por­tant to op­ti­miz­ing the like­li­hood of pos­i­tive health out­comes from treat­ment?
  3. What is­sues of eq­ui­ty and in­clu­sion must be ac­count­ed for in the di­ag­no­sis and treat­ment of Alzheimer’s dis­ease?
  4. What health care providers should be in­clud­ed as part of the pa­tient’s treat­ment team? Should med­ical spe­cial­ists be in­clud­ed in the care team of pa­tients re­ceiv­ing treat­ment? If so, which spe­cial­ists should be in­clud­ed in the care?
  5. In what set­ting(s) should treat­ment and care be giv­en?

CMS said it has al­ready ini­ti­at­ed its own dis­cus­sions with not on­ly Bio­gen, but the Alzheimer’s As­so­ci­a­tion — which has raised con­cerns about the 9-year time­line for Bio­gen’s con­fir­ma­to­ry tri­al — Duke Uni­ver­si­ty’s Mar­go­lis Cen­ter for Health Pol­i­cy, and Johns Hop­kins Uni­ver­si­ty.

But such na­tion­al de­ter­mi­na­tions to lim­it cov­er­age for FDA-ap­proved drugs are rare. And even when the process be­gins, they of­ten don’t end up lead­ing to lim­its. For in­stance, with CAR-T cell ther­a­pies, CMS de­bat­ed whether to re­strict cov­er­age to on­ly when it is of­fered in a CMS-ap­proved reg­istry or clin­i­cal study, but even­tu­al­ly de­cid­ed against such a lim­it. CMS came to a sim­i­lar con­clu­sion to not lim­it na­tion­al cov­er­age for Den­dreon’s can­cer treat­ment Provenge, which end­ed up be­ing a com­mer­cial dud.

So­cial im­age cred­it: Shut­ter­stock

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

Covid-19 roundup: Pfiz­er im­pos­es vac­cine man­date for US work­ers; WHO calls for mora­to­ri­um on boost­ers, while some coun­tries make plans any­way — re­port

As the US struggles to keep pace with the fast-spreading Delta variant, big companies like Walmart and Disney are imposing vaccine mandates for some workers. It may come as no surprise that Pfizer — the Big Pharma behind the US’ first authorized Covid-19 vaccine — is joining them.

Pfizer will start requiring all US employees and contractors to get vaccinated, or participate in weekly Covid-19 testing, spokesperson Pamela Eisele told Reuters. Workers outside the US are strongly urged to get a vaccine if they can, according to the report. And those with medical conditions or religious objections can seek accommodations.

Tien Lee, Aardvark Therapeutics CEO

Emerg­ing from stealth mode, Aard­vark rounds up enough cash to put its lead drug through Prad­er-Willi PhII

When Aardvark Therapeutics CEO Tien Lee started his work on the biotech’s lead candidate, appetite suppression was the goal for the small molecule.  Soon after, his team started to see added benefits with lower blood glucose levels and anti-inflammatory activity. On the tail end of that, the company has emerged from stealth mode and announced today that they’ve raised enough cash in the B round to cover mid-stage development work.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.