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

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.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

Endpoints Premium

Premium subscription required

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

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.

More man­u­fac­tur­ing is­sues: Fen­nec preps for sec­ond CRL for po­ten­tial hear­ing loss drug

Shares of Fennec Pharmaceuticals stock were cut almost in half early Monday as the company said manufacturing issues caused another FDA rejection of its reformulated version of sodium thiosulfate, which is intended to help kids who lose hearing due to chemo treatment.

The biotech had resubmitted an NDA for the drug to treat platinum-based, chemo-related ototoxicity in young children earlier this year. The first NDA was denied by the FDA last year, with the agency citing manufacturing issues with the biotech’s supplier.

Like the flu vac­cine every year, the FDA could move quick­ly on a vari­ant-tar­get­ed Covid vac­cine

In the same way that the FDA signs off on flu vaccines every year without requiring large clinical trials to measure their efficacy, the FDA may employ a similar strategy in authorizing variant-focused versions of the mRNA vaccines.

As the world braces for more data on the latest variant Omicron, which may reduce vaccine efficacy, top vaccine developers like Moderna and Pfizer-BioNTech have promised they can pull together a new vaccine targeted against a specific Covid variant in about 100 days. Since Omicron emerged last week, Pfizer-BioNTech, Moderna and J&J have all said they’ve begun work on Omicron-specific vaccines, if needed.

Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; 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.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.

Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 124,200+ biopharma pros reading Endpoints daily — and it's free.