Opin­ion: CMS needs to do FDA’s job and lim­it the use of Bio­gen’s pricey new Alzheimer’s drug

Now that the FDA has ap­proved Bio­gen’s ex­pen­sive new Alzheimer’s drug with lit­tle clin­i­cal ben­e­fit po­ten­tial, but plen­ty of po­ten­tial to speed the bank­rupt­cy of the gov­ern­ment’s health care pro­gram for se­niors, it’ll be up to the Cen­ters for Medicare and Med­ic­aid Ser­vices to lim­it pay­ment for it and dri­ve the con­ver­sa­tion on ac­cess.

While such ac­cess de­ci­sions are nor­mal­ly re­served for the FDA, the agency ab­di­cat­ed its re­spon­si­bil­i­ty be­cause of the wide la­bel and be­cause it’s al­low­ing Bio­gen to take al­most a decade to fig­ure out if ad­u­canum­ab ac­tu­al­ly works.

But what CMS does now will have a mas­sive im­pact on the fi­nan­cial health and well-be­ing of its Medicare pro­gram, es­pe­cial­ly as Aduhelm may speed Medicare’s path to in­sol­ven­cy, cur­rent­ly set for 2026.

The fi­nan­cial im­pli­ca­tions of Aduhelm range from daunt­ing to down­right dis­turb­ing.

Ron­ny Gal

As Bern­stein biotech Ron­ny Gal point­ed out, “The to­tal cost of Medicare part B drugs was $37B in 2019, grow­ing at 13%. This is the bud­get seg­ment Pres­i­dent Trump called ‘out of con­trol’. If 1M pa­tients use Aduhelm at the cur­rent price point, to­tal Medicare part B spend­ing would dou­ble.”

While Bio­gen has said they will have the man­u­fac­tur­ing ca­pac­i­ty to treat 1 mil­lion Alzheimer’s pa­tients, Gal not­ed that the fi­nan­cial im­pli­ca­tions of a more re­al­is­tic sce­nario of just half that pop­u­la­tion “is equal­ly mind-bog­gling.”

If 50% of new­ly di­ag­nosed pa­tients start Aduhelm at the cur­rent price, then the to­tal cost to Medicare will be equal to the top five drugs in Medicare Part B com­bined (Mer­ck’s Keytru­da, Re­gen­eron’s Eylea, BMS’ Op­di­vo, Roche’s Rit­ux­an and Am­gen’s Pro­lia – at $10.7B based on the Medicare dash­board), ac­cord­ing to Gal.

“We can eas­i­ly see a sit­u­a­tion where the com­bi­na­tion of the un­re­strict­ed la­bel and the high price could lead in-pa­tient fa­cil­i­ties treat­ing se­vere AD pa­tients to [get] as many of their res­i­dents on treat­ment as pos­si­ble,” he wrote in an in­vestor note ear­li­er this month.

Rep. Pe­ter Welch (D-VT) sent a let­ter to Bio­gen’s CEO Michel Vounatsos on Tues­day, not­ing, “In 2019, the to­tal amount of spend­ing for all Part B drugs was on­ly $37 bil­lion, so Bio­gen’s price for one drug and one treat­ment would cost near­ly $20 bil­lion more than the en­tire ex­ist­ing Medicare Part B drug pro­gram for all ben­e­fi­cia­ries.”

The ma­jor­i­ty of Amer­i­cans suf­fer­ing from Alzheimer’s— more than 6 mil­lion peo­ple—are Medicare ben­e­fi­cia­ries, Welch added, not­ing, “Bio­gen has abused the pric­ing pow­er that it holds.”

So what will CMS need to do?

The cen­ters may be tasked with in­sti­tut­ing what’s known as a Medicare Na­tion­al Cov­er­age De­ter­mi­na­tion on Aduhelm, which is usu­al­ly re­served for Medicare ser­vices and not new drugs. But such a de­ter­mi­na­tion could pro­tect Medicare ben­e­fi­cia­ries from FDA’s wide la­bel, and re­strict the use of the drug to on­ly the sub­groups of se­niors who might ben­e­fit and who were stud­ied in Bio­gen’s failed tri­als.

Rachel Sachs

“If CMS were to use the NCD process to re­strict cov­er­age for Aduhelm be­yond the FDA’s broad la­bel for the drug, it would set a very im­por­tant prece­dent by delink­ing the FDA’s de­ci­sion to ap­prove a drug from CMS’s de­ci­sion to pro­vide re­im­burse­ment for it,” Rachel Sachs, law pro­fes­sor at Wash­ing­ton Uni­ver­si­ty in St. Louis, wrote re­cent­ly in Health Af­fairs.

Biden’s new­ly con­firmed CMS ad­min­is­tra­tor Chiq­ui­ta Brooks-La­Sure said she’s look­ing in­to all the ev­i­dence in de­cid­ing whether to make a na­tion­al cov­er­age de­ter­mi­na­tion.

Oth­ers are sug­gest­ing that CMS con­duct its own re­search on pay­ing for Aduhelm.

Pe­ter Bach, di­rec­tor of the Drug Pric­ing Lab at Memo­r­i­al Sloan Ket­ter­ing Can­cer Cen­ter, and Craig Garth­waite, di­rec­tor of the Pro­gram on Health­care at the Kel­logg School of Man­age­ment at North­west­ern Uni­ver­si­ty, called on Medicare’s re­search cen­ter, the Cen­ter for Medicare and Med­ic­aid In­no­va­tion, to eval­u­ate whether CMS should pay for the drug and whether those pay­ments are cost-ef­fec­tive.

To eval­u­ate such pay­ments while Bio­gen’s 9-year con­fir­ma­to­ry tri­al is on­go­ing, Bach and Garth­waite called on CMS to se­lect some coun­ties where it would re­im­burse Aduhelm’s cur­rent price, and oth­ers where it would re­im­burse $0, and see what the dif­fer­ences are.

“Medicare must study ad­u­canum­ab re­im­burse­ment now, and pre­pare to do the same when­ev­er the FDA ap­proves oth­er cost­ly, im­por­tant drugs that lack ev­i­dence of ef­fec­tive­ness,” Bach and Garth­waite wrote. “Drug com­pa­nies should know that if their med­i­cines reach the mar­ket be­fore the ev­i­dence is in, pay­ment will not be au­to­mat­ic.”

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; 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.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Janet Woodcock (AP Images)

Janet Wood­cock plots her fu­ture at FDA, with se­nior ad­vi­sor role to fall back on if Califf wins con­fir­ma­tion

Acting FDA commissioner Janet Woodcock has been the face of just about every drug approval decision at the agency since the turn of the century. Since the pandemic began, she’s moved between the top of the drugs center to the head of therapeutics at Operation Warp Speed, leading the drive for work on Covid-targeted mAbs and antivirals.

Looking forward — and pending a quick Senate confirmation to cement Rob Califf’s return to the top of FDA early next year — Woodcock’s role at the agency will again be in flux.

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Richard Pazdur (via AACR)

Ac­cel­er­at­ed ap­proval re­forms need mean­ing­ful con­fir­ma­to­ry tri­al im­prove­ments, pro­fes­sors write in Sci­ence

Outside of Covid-19, 2021 has been the year of the accelerated approval.

Beginning last spring, FDA openly challenged six “dangling” accelerated approvals (hadn’t confirmed their clinical benefit yet), three of which were later pulled by the companies.

Then in June, FDA pulled out the accelerated approval pathway, seemingly out of nowhere, to sign off on Biogen’s controversial Alzheimer’s drug Aduhelm. It hadn’t even been mentioned at the drug’s adcomm.

Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News. 

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Lan Huang, BeyondSpring CEO

Months af­ter shock­ing in­vestors with lung can­cer win, Be­yond­Spring's lead drug hits road­block at the FDA

BeyondSpring shocked investors in early August after its once-marginal lead drug suddenly showed a lot of promise in a common form of lung cancer. With hopes high, the FDA has now slammed the door on that drug in another indication — does that spell bad news for BeyondSpring’s Cinderella story?

The FDA issued BeyondSpring a complete response letter for its plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia, effectively shutting down the drug’s immediate chances at a marketing approval, the biotech said Wednesday.

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