Out­spo­ken re­searcher Pe­ter Bach urges CMS to con­sid­er ways of bring­ing down CAR-T prices — and of­fers a few sug­ges­tions

When it comes to de­ter­min­ing cov­er­age for ex­pen­sive — but po­ten­tial­ly life­sav­ing — CAR-T ther­a­pies, CMS doesn’t have to roll over and let drug­mak­ers dom­i­nate the con­ver­sa­tion. The agency, in­stead, should lever­age com­pe­ti­tion to bring down the cost of these still-im­per­fect meds.

That’s ac­cord­ing to Pe­ter Bach, the di­rec­tor of Memo­r­i­al Sloan Ket­ter­ing’s Cen­ter for Health Pol­i­cy and Out­comes in New York. Known for of­ten speak­ing out on drug pric­ing, Bach de­tailed his thoughts on the mat­ter in a pa­per pub­lished in The New Eng­land Jour­nal of Med­i­cine.

“CAR-T ther­a­pies have bro­ken new ground on many fronts — they have shown ef­fi­ca­cy in pa­tients who pre­vi­ous­ly had few op­tions, but they cost mul­ti­ple times what any pre­vi­ous­ly ap­proved can­cer ther­a­py costs,” Bach wrote. “Their rapid ap­proval based on small, un­con­trolled stud­ies re­flects their promise. But they are no panacea.”

In the pa­per, he points to drugs like Gilead’s Yescar­ta and No­var­tis’ Kym­ri­ah, which each cost about $400,000. Bach al­so notes that an­cil­lary costs as­so­ci­at­ed with CAR-T treat­ments could add up to an ex­tra $33,000 or more, as pa­tients of­ten must be hos­pi­tal­ized and are re­quired to take im­muno­sup­pres­sive drugs in tan­dem.

Bach has sev­er­al ideas for bring­ing down the price of CAR-Ts, which he out­lines in the chart be­low. But CMS must an­swer two ques­tions be­fore choos­ing strate­gies to low­er those prices, he said: Are the net ben­e­fits of CAR-T drugs sim­i­lar? Do the an­cil­lary ser­vices cost the same for each drug?

De­ter­min­ing the net ben­e­fit would be tough, though. That con­clu­sion would be based off ob­ser­va­tions from on­ly a hand­ful of pa­tients 65 or old­er (and thus age-el­i­gi­ble for Medicare). But as­sum­ing CMS does de­cide there’s an equal net ben­e­fit, CMS “need not sim­ply say yes to cov­er­age,” Bach says.

(CMS) could lim­it which providers and hos­pi­tals are el­i­gi­ble to ad­min­is­ter CAR-T, as it does for some or­gan trans­plan­ta­tion, for in­stance, or it could use the Cov­er­age with Ev­i­dence De­vel­op­ment (CED) des­ig­na­tion, which can ei­ther in­clude a re­quire­ment that fur­ther out­come da­ta be col­lect­ed in a reg­istry or lim­it cov­er­age to pa­tients en­rolled in clin­i­cal tri­als. In ei­ther case, CED aims to but­tress the ev­i­dence rel­e­vant to the “rea­son­able and nec­es­sary” ques­tion.

Al­so, they could de­sign a pay­ment sys­tem for CAR-Ts that pro­motes com­pe­ti­tion based on price — an im­prove­ment over the cur­rent sys­tem, which in­cludes such ther­a­py in Part B drug re­im­burse­ment and pro­vides doc­tors and hos­pi­tals with larg­er prof­its when the treat­ment costs more.

Bach built this chart to help de­ter­mine the best strate­gies for CMS to im­ple­ment. Read more in his full pa­per at NE­JM.


Im­age: Pe­ter Bach. MEMO­R­I­AL SLOAN KET­TER­ING

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Amid mon­key­pox fears, biotechs spring to ac­tion; Mod­er­na’s CFO trou­ble; Cuts, cuts every­where; Craft­ing the right pro­teins; 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.

It’s always a bittersweet moment saying goodbye, but as Josh Sullivan goes off to new adventures we are grateful for the way he’s built up the Endpoints Manufacturing section — which the rest of the team will now carry forward. If you’re not already, this may be a good time to sign up for your weekly dose of drug manufacturing news. Thank you for reading and wish you a restful weekend.

Bay­er sounds re­treat from a $670 mil­lion CAR-T pact in the wake of a pa­tient death

Two months after Atara Biotherapeutics hit the hold button on its lead CAR-T 2.0 therapy following a patient death, putting the company under the watchful eye of the FDA, its Big Pharma partners at Bayer are bowing out of a $670 million global alliance. And the move is forcing a revamp of Atara’s pipeline plans, even as research execs vow to continue work on the two drugs allied with Bayer 18 months ago, which delivered a $60 million cash upfront.

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Sanofi and Re­gen­eron clear the fin­ish line in an in­flam­ma­to­ry esoph­a­gus dis­ease, leav­ing Take­da in the dust

With atopic dermatitis rivals breathing down Dupixent’s neck, Sanofi and Regeneron on Friday secured a first win in new territory in what Sanofi’s head of immunology and inflammation Naimish Patel called the fastest approval he’s ever seen.

The FDA approved Dupixent on Friday to treat patients 12 years and older with eosinophilic esophagitis (EoE), an inflammatory condition that causes swelling and scarring of the esophagus. The approval came just a couple months after regulators granted Dupixent priority review, and months ahead of its PDUFA date on Aug. 3.

Fu­ji­film con­tin­ues its biotech build­ing spree with new fa­cil­i­ty in Chi­na

A Japanese conglomerate is making a big play in China with the opening of a new facility, as it continues to expand.

Fujifilm Irvine Scientific has opened its new Innovation and Collaboration Center in Suzhou New District, China, an area in Jiangsu province specifically designated for technological and industrial development.

According to Fujifilm, the 12,000-square-foot site will be responsible for the company’s cell culture media optimization, analysis and design services. Cell culture media itself often requires customization of formulas and protocols to achieve the desired quantity and quality of therapeutic desired. Fujifilm Irvine Scientific is offering these services from its headquarters in California and Japan to its customers globally, as well as in China now.

Rob Etherington, Clene CEO

Mary­land of­fers loan to Clene de­spite ALS tri­al bumps

Even after Utah-based Clene failed to hit its primary endpoints for its ALS drug last year, the state of Maryland is putting its money at least behind Clene’s manufacturing facility.

The Maryland Board of Public Works has finalized a $3 million, 60-month loan facility with Clene Nanomedicine. The loan was provided by the state’s Neighborhood BusinessWorks program within the Maryland Department of Housing and Community Development.

Armon Sharei, SQZ founder and CEO

SQZ's out­side-the-box man­u­fac­tur­ing method slash­es pro­duc­tion time in ear­ly in study

At ASCO 2021 in June of last year, SQZ Biotech showcased a glimpse of its unorthodox cell therapy manufacturing tech. And on Wednesday, the Watertown, MA, company announced that its first-generation system showed comparable or better performance than a conventional clean-room-based manufacturing process.

The study was non-clinical. Clinical trials are expected by the first half of 2023.

SQZ’s device opens up a temporary window by cell-squeezing to deliver cargoes into cells. Its average processing time was less than six hours per batch, which is more than half the time than conventional methods. The company is planning to use the technology in its first red blood cell derived program for celiac disease. That IND is set to be submitted in the first half of 2023, the company said.

Bobby Sheng, Bora Pharmaceuticals CEO

With new ac­qui­si­tion, Bo­ra to ven­ture in­to bi­o­log­ics

Last week, Taiwan-based CDMO Bora Pharmaceuticals announced that it acquired Eden Biologics. Now, it says that purchase has helped established Bora Biologics, expanding into the biopharmaceutical market.

The acquisition of the company’s assets, which are located in the Hsinchu Biomedical Science Park in Taiwan, is helping Bora build its presence in the biopharma world by expanding production capacity of cell lines for the production of protein drugs. It will also improve the quality control and inspection specifications, as well as cell bank generation. The facility has four 500-liter bioreactors that have been approved by European and Taiwanese regulators.

Paul Chaplin, Bavarian Nordic president and CEO

With mon­key­pox cas­es ris­ing, one Eu­ro­pean coun­try is lock­ing down a small­pox vac­cine con­tract

As the global number of confirmed and suspected monkeypox cases continues to slowly climb, one country is trying to get a head start on potential vaccine stocking.

Bavarian Nordic signed a contract with an undisclosed European nation to supply its smallpox vaccine in response to new cases this month, the company announced Thursday morning. The continent saw its first monkeypox case confirmed about two weeks ago, with both the UK and Portugal seeing cases, according to the Washington Post.

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