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.”

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP Images)

Up­dat­ing the Covid-19 vac­cine: FDA of­fers a qual­i­fied thumbs-up ahead of ad­comm

The FDA’s adcomm of outside vaccine experts will meet tomorrow to discuss how to protect the US from a likely coming wave of Omicron cases in the fall and winter, and whether to deploy vaccines that specifically target the Omicron variant.

While the data so far are limited, the FDA sounded an upbeat tone in the briefing documents on Pfizer/BioNTech’s candidates, released this weekend ahead of the VRBPAC meeting.

Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

As court case looms, Bris­tol My­ers touts la­bel ex­pan­sion for Breyanzi

As Bristol Myers Squibb braces for a court battle over a costly delay — at least for Celgene shareholders — for its CAR-T lymphoma treatment Breyanzi, the pharma giant is touting a label expansion in the second-line setting.

Breyanzi, also known as liso-cel, snagged a win on Friday in adults with large B-cell lymphoma (LBCL) who: don’t respond to chemotherapy, or relapse within 12 months; don’t respond or relapse after 12 months; or are not eligible for hematopoietic stem cell transplant after chemo due to their age or comorbidities.

Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.