UP­DAT­ED: CMS to re­strict cov­er­age of Bio­gen's con­tro­ver­sial Alzheimer's drug to on­ly clin­i­cal tri­als

The Cen­ters for Medicare and Med­ic­aid Ser­vices on Tues­day said it will on­ly pay for Bio­gen’s Aduhelm and oth­er FDA-ap­proved an­ti-amy­loid mon­o­clon­al an­ti­bod­ies for Alzheimer’s dis­ease un­der CMS-ap­proved ran­dom­ized con­trolled tri­als.

The draft na­tion­al cov­er­age de­ci­sion, which in­sur­ers na­tion­wide are like­ly to fol­low, makes clear that CMS will be look­ing for ran­dom­ized con­trolled tri­als that “demon­strate a clin­i­cal­ly mean­ing­ful ben­e­fit in cog­ni­tion and func­tion.” That will be a tough task for Bio­gen, which pre­vi­ous­ly showed con­flict­ing ben­e­fits from past Aduhelm tri­als that were ini­tial­ly cut short due to fu­til­i­ty and then res­ur­rect­ed for the ac­cel­er­at­ed ap­proval.

“None of the tri­als com­plet­ed as of the pub­li­ca­tion of this NCA has con­vinc­ing­ly demon­strat­ed that use of an­ti­amy­loid mAbs re­sult in a mean­ing­ful im­prove­ment in health out­comes for AD pa­tients,” CMS said in its draft de­ci­sion, which is now open for 30 days of more com­ments, and will be fi­nal­ized in April.

Hol­ly Fer­nan­dez Lynch, a bioethics pro­fes­sor at the Uni­ver­si­ty of Penn­syl­va­nia, told End­points News that she thinks CMS made the right de­ci­sion in go­ing for cov­er­age with ev­i­dence de­vel­op­ment (CED) and re­quir­ing the tri­al, al­though she not­ed how odd it is that the gov­ern­ment will be es­sen­tial­ly pay­ing Bio­gen to run a tri­al.

“I didn’t think out­right re­fusal of any cov­er­age was gen­uine­ly on the ta­ble. This is a pret­ty strong re­buke of FDA as it is,” she said.

The an­nounce­ment is a blow for Bio­gen, which will like­ly have to de­sign and run an­oth­er clin­i­cal tri­al in ad­di­tion to its con­fir­ma­to­ry tri­al to sat­is­fy the FDA’s ac­cel­er­at­ed ap­proval re­quire­ments. Bio­gen’s stock dropped about 8% Tues­day af­ter­noon.

“This draft cov­er­age de­ter­mi­na­tion de­nies the dai­ly bur­den of peo­ple liv­ing with Alzheimer’s dis­ease,” Bio­gen said in a state­ment emailed to End­points, adding:

Cov­er­age with ev­i­dence de­vel­op­ment (CED) un­der a ran­dom­ized clin­i­cal tri­al will ex­clude al­most all pa­tients who may ben­e­fit. This will sig­nif­i­cant­ly lim­it pa­tient ac­cess to an FDA-ap­proved treat­ment, es­pe­cial­ly for un­der­served pa­tients as ev­i­denced in oth­er CED de­ter­mi­na­tions. CEDs can take months to years to ini­ti­ate, and hun­dreds of Alzheimer’s pa­tients – the ma­jor­i­ty of whom are Medicare ben­e­fi­cia­ries – are pro­gress­ing each day from mild to mod­er­ate dis­ease stages, where treat­ment may no longer be an op­tion.

But Lon Schnei­der, who di­rects the Uni­ver­si­ty of South­ern Cal­i­for­nia’s State of Cal­i­for­nia Alzheimer’s Dis­ease Cen­ter, told End­points that 80% of pa­tients in the Bio­gen tri­als had pro­dro­mal AD, which is “de­f­i­n­i­tion­al­ly, vir­tu­al­ly no dai­ly bur­den (a bit of mem­o­ry im­pair­ment, yes, a bit of amy­loid? Yes). And the tri­als couldn’t demon­strate that any one pa­tient ben­e­fit­ted from ad­u­canum­ab, so who’s be­ing de­prived ben­e­fit?”

He called the move “a smack­down” as CMS “al­so told Lil­ly that FDA might give you AA [ac­cel­er­at­ed ap­proval] or BTD [break­through des­ig­na­tion] but we’ll wait to see pos­i­tive tri­als re­sults.”

In­dus­try group PhRMA of­fered a sim­i­lar char­ac­ter­i­za­tion, not­ing, “With this pro­pos­al, CMS is writ­ing off an en­tire class of med­i­cines be­fore mul­ti­ple prod­ucts have even been re­viewed by FDA, po­si­tion­ing it­self and not FDA as the key ar­biter of clin­i­cal ev­i­dence.”

The FDA de­clined to com­ment on the de­ci­sion by CMS.

CMS al­so lays out very spe­cif­ic tri­al re­quire­ments that Bio­gen, Roche, Eli Lil­ly and any oth­er amy­loid con­tenders will have to meet, which is not what CMS has done in pre­vi­ous na­tion­al cov­er­age de­ci­sions, not­ing that it’s aware of at least three oth­er an­ti-amy­loid mAbs cur­rent­ly ap­proach­ing Phase 3 tri­als.

“The di­ver­si­ty of pa­tients in­clud­ed in each tri­al must be rep­re­sen­ta­tive of the na­tion­al pop­u­la­tion di­ag­nosed with AD,” CMS says, not­ing that the tri­als must ad­dress, “at a min­i­mum,” whether the  use of mAbs di­rect­ed against amy­loid re­sult in a sta­tis­ti­cal­ly sig­nif­i­cant and clin­i­cal­ly mean­ing­ful dif­fer­ence in de­cline in cog­ni­tion and func­tion, and whether they ad­dress the ad­verse events as­so­ci­at­ed with the use of these an­ti-amy­loid mAbs.

The news comes as Bio­gen re­cent­ly cut Aduhelm’s price in half, af­ter sell­ing just $300,000 of Aduhelm in the ear­ly months, and com­ing un­der heavy crit­i­cism for the $56,000 price tag that forced CMS to prep for record-high pre­mi­um in­creas­es for se­niors. HHS Sec­re­tary Xavier Be­cer­ra ear­li­er this week called on CMS to re­con­sid­er those steep pre­mi­um in­creas­es now that Aduhelm’s price has been re­duced.

Sen. Bernie Sanders of Ver­mont said in a state­ment that CMS’ de­ci­sion “is an im­por­tant step for­ward, es­pe­cial­ly fol­low­ing Sec­re­tary Be­cer­ra’s move yes­ter­day to di­rect Medicare to re­con­sid­er a pre­mi­um rate hike caused in part by the out­ra­geous price of Aduhelm.”

Oth­ers who be­lieve that Aduhelm does work were dis­ap­point­ed by the de­ci­sion from CMS, which will like­ly mean on­ly a lim­it­ed num­ber of pa­tients are cov­ered un­der a CMS-ap­proved clin­i­cal tri­al.

“This is clear­ly dis­ap­point­ing news, as CED would sig­nif­i­cant­ly lim­it the num­ber of pa­tients who can ac­cess Aduhelm and oth­er an­ti-Abe­ta mAbs, and in this sce­nario, we would ex­pect a pro­longed process to ini­ti­ate and en­roll pa­tients in­to the CED clin­i­cal tri­als which need to be re­viewed and ap­proved by CMS,” SVB Leerink told in­vestors.

Ad­vo­ca­cy or­ga­ni­za­tions were sim­i­lar­ly not pleased.

Har­ry Johns, Alzheimer’s As­so­ci­a­tion CEO, said in a state­ment, “To­day’s draft de­ci­sion by the Cen­ters for Medicare & Med­ic­aid Ser­vices (CMS) is shock­ing dis­crim­i­na­tion against every­one with Alzheimer’s dis­ease, es­pe­cial­ly those who are al­ready dis­pro­por­tion­ate­ly im­pact­ed by this fa­tal dis­ease, in­clud­ing women, Blacks and His­pan­ics.”

George Vraden­burg, chair and co-founder of Us­Again­stAlzheimer’s, added, “This is ab­solute­ly un­ac­cept­able. If this de­ci­sion stands, for the first time in his­to­ry, mil­lions of Amer­i­cans will be de­nied cov­er­age not just to a drug, but to a whole class of drugs—not by the agency that reg­u­lates drugs but by the fed­er­al in­sur­ance bu­reau­cra­cy.”

Ed­i­tor’s note: Ar­ti­cle up­dat­ed with ad­di­tion­al com­ments.

Mar­ket­ingRx roundup: Pfiz­er de­buts Pre­vnar 20 TV ads; Lil­ly gets first FDA 2022 pro­mo slap down let­ter

Pfizer debuted its first TV ad for its Prevnar 20 next-generation pneumococcal pneumonia vaccine. In the 60-second spot, several people (actor portrayals) with their ages listed as 65 or older are shown walking into a clinic as they turn to say they’re getting vaccinated with Prevnar 20 because they’re at risk.

The update to Pfizer’s blockbuster Prevnar 13 vaccine was approved in June, and as its name suggests is a vaccine for 20 serotypes — the original 13 plus seven more that cause pneumococcal disease. Pfizer used to spend heavily on TV ads to promote Prevnar 13 in 2018 and 2019 but cut back its TV budgets in the past two fall and winter seasonal spending cycles. Prevnar had been Pfizer’s top-selling drug, notching sales of just under $6 billion in 2020, and was the world’s top-selling vaccine before the Covid-19 vaccines came to market last year.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 129,600+ biopharma pros reading Endpoints daily — and it's free.

Graphic: Alexander Lefterov for Endpoints News

Small biotechs with big drug am­bi­tions threat­en to up­end the tra­di­tion­al drug launch play­book

Of the countless decisions Vlad Coric had to make as Biohaven’s CEO over the past seven years, there was one that felt particularly nerve-wracking: Instead of selling to a Big Pharma, the company decided it would commercialize its migraine drug itself.

“I remember some investors yelling and pounding on the table like, you can’t do this. What are you thinking? You’re going to get crushed by AbbVie,” he recalled.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 129,600+ biopharma pros reading Endpoints daily — and it's free.

Albert Bourla (Photo by Steven Ferdman/Getty Images)

UP­DAT­ED: Pfiz­er fields a CRL for a $295M rare dis­ease play, giv­ing ri­val a big head start

Pfizer won’t be adding a new rare disease drug to the franchise club — for now, anyway.

The pharma giant put out word that their FDA application for the growth hormone therapy somatrogon got the regulatory heave-ho, though they didn’t even hint at a reason for the CRL. Following standard operating procedure, Pfizer said in a terse missive that they would be working with regulators on a followup.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 129,600+ biopharma pros reading Endpoints daily — and it's free.

Alexander Lefterov/Endpoints News

A new can­cer im­munother­a­py brings cau­tious hope for a field long await­ing the next big break­through

Bob Seibert sat silent across from his daughter at their favorite Spanish restaurant near his home in Charleston County, SC, their paella growing cold as he read through all the places in his body doctors found tumors.

He had texted his wife, a pediatric intensive care nurse, when he got the alert that his online chart was ready. Although he saw immediately it was bad, many of the terms — peritoneal, right iliac — were inscrutable. But she was five hours downstate, at a loud group dinner the night before another daughter’s cheer competition.

Endpoints Premium

Premium subscription required

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

Not cheap­er by the dozen: Bris­tol My­ers be­comes the 12th phar­ma com­pa­ny to re­strict 340B sales

Bristol Myers Squibb recently joined 11 of its peer pharma companies in limiting how many contract pharmacies can access certain drugs discounted by a federal program known as 340B.

Bristol Myers is just the latest in a series of high-profile pharma companies moving in their own direction as the Biden administration’s Health Resources and Services Administration struggles to rein in the drug discount program for the neediest Americans.

Joaquin Duato, J&J CEO (Photo by Charles Sykes/Invision/AP)

New J&J CEO Joaquin Du­a­to promis­es an ag­gres­sive M&A hunt in quest to grow phar­ma sales

Joaquin Duato stepped away from the sideline and directly into the spotlight on Tuesday, delivering his first quarterly review for J&J as its newly-tapped CEO after an 11-year run in senior posts. And he had some mixed financial news to deliver today while laying claim to a string of blockbuster drugs in the making and outlining an appetite for small and medium-sized M&A deals.

Duato also didn’t exactly shun large buyouts when asked about the future of the company’s medtech business — where they look to be in either the top or number 2 position in every segment they’re in — even though the bar for getting those deals done is so much higher.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 129,600+ biopharma pros reading Endpoints daily — and it's free.

Amgen's Twitter campaign #DearAsthma inspired thousands of people to express struggles and frustrations with the disease

Am­gen’s #Dear­Asth­ma spon­sored tweet lands big on game day, spark­ing thou­sands to re­spond

Amgen wanted to know how people with asthma really felt about daily life with the disease. So it bought a promoted tweet on Twitter noting the not-so-simple realities of life with asthma and ended the post with a #DearAsthma hashtag, a megaphone emoji and a re-tweet button.

That was just over one week ago and the responses haven’t stopped. More than 7,000 posts so far on Twitter replied to #DearAsthma to detail struggles of daily life, expressing humor, frustration and sometimes anger. More than a few f-bombs have been typed or gif-ed in reply to communicate just how much many people “hate” the disease.

Pfiz­er, Bris­tol My­ers dom­i­nate top 10 pre­dic­tions for the best-sell­ing drugs of 2022

The annual exercise where analysts try and predict which drugs will become blockbusters and make the most money tends to highlight the biggest trends in biopharma R&D. 2022 is no exception.

The team at Evaluate Vantage published its predictions for the top 10 selling drugs for the year — expecting tens of billions of dollars in sales and highlighting an industry-wide focus on certain diseases and indications.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 129,600+ biopharma pros reading Endpoints daily — and it's free.

Nabiha Saklayen, Cellino co-founder and CEO (via Cellino)

Backed by Bay­er's Leaps, Boston-based Celli­no lands $80M for cell ther­a­py-in-box

The summer before Cellino CEO and co-founder Nabiha Saklayen started at Harvard, she lost her grandmother following complications to diabetes. Before then, she hadn’t taken a biology class since ninth or tenth grade — the mark of a classic physicist — but it was then she decided she wanted the rest to sit at the intersection of the two for the rest of her career

Combine that with being across the way from the University’s stem cell institute in Cambridge, and you get the birth of Cellino, an autonomous cell therapy manufacturing company that just announced the closing of its Series A.