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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

Bartley Griffith (University of Maryland School of Medicine)

New re­port de­tails se­quence of events lead­ing up to death of man who re­ceived pig heart trans­plant

Doctors still don’t know why the man who received a pig heart transplant died.

In a new paper in NEJM, the team of doctors from the University of Maryland, led by Bartley Griffith, detailed the sequence of events that followed after 57-year-old David Bennett received a pig heart, which failed 49 days after it was transplanted. Bennett died shortly after, 60 days after he received the genetically modified heart.

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EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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