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

Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

Daniel O'Day [via AP Images]

UP­DAT­ED: Gilead un­leash­es a $5B late-stage cash al­liance with Gala­pa­gos — lay­ing out O'­Day's R&D strat­e­gy

Daniel O’Day is executing his first major development deal since taking over as CEO of Gilead $GILD. And he’s going in deep to ally himself with a longstanding partner.

O’Day announced today that he is spending $5 billion in cash to add new late-stage drugs to Gilead’s pipeline, picking up rights to Galapagos’ $GLPG Phase III IPF drug GLPG1690 alongside adoption of the biotech’s Phase IIb drug GLPG1972 for osteoarthritis. And Gilead is also putting billions more on the table for milestones, gaining options for everything else in Galapagos’ pipeline, with a shot at all rights outside of Europe.

Altogether, Gilead is gaining rights to 6 clinical-stage assets, 20 preclinical programs and everything else being hatched in translation.

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Pe­ter Kolchin­sky and Raj Shah raise a $300M fund de­vot­ed to biotech star­tups

Peter Kolchinsky and Raj Shah have another $300 million-plus to play with on the biotech venture side of their investment business. 

The two announced Monday morning that they’ve put together their first pure-play venture fund at RA Capital Management, which has been known to bet on just about every angle in healthcare investing — from rounds to follow-on investments at public companies. This new fund of theirs arrives well into a go-go era of new startup financing, with a particular focus on building new biotechs.

Boehringer buys Swiss biotech in its lat­est M&A deal, go­ing the next-gen can­cer vac­cine route

Boehringer Ingelheim has snapped up a Swiss biotech startup and added their group as a new platform for the oncology pipeline. 

The German biopharma company has bagged Geneva-based AMAL Therapeutics, paying out an unspecified upfront in a $358 million deal — cash, milestones and everything else, all in. Plus there’s 100 million euros on the line for commercial milestones.

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Ab­b­Vie beefs up the on­col­o­gy pipeline, bag­ging an up­start STING play­er with its own unique ap­proach

AbbVie isn’t letting its $63 billion buyout of Allergan stop its M&A/deals team from continuing their work.

Monday morning we learned that the pharma giant is snapping up tiny Mavupharma out of Seattle, a Frazier-backed startup that has its own unique take on STING — which is on the threshold of their first clinical trial.

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Hal Bar­ron's team at GSK scores a win with pos­i­tive Ze­ju­la PhI­II front­line study — now comes the hard part

Score one for Hal Barron and the new R&D team steering GlaxoSmithKline’s pipeline.

The pharma giant reported this morning that its recently acquired PARP, Zejula (niraparib), hit the primary endpoint on progression-free survival in a frontline maintenance setting for women suffering ovarian cancer — following chemo and regardless of their BRCA status.

GSK bet $5 billion on the Tesaro buyout primarily to get this drug, drawing the shaking heads of biopharma. Why pay a big premium for a drug like this when AstraZeneca was going from strength to strength with Lynparza, ran the argument, having won a hugely important accelerated approval to jump out ahead — way ahead — of the rest of the PARP players? Lynparza — now co-owned by a powerhouse cancer team at Merck — won the first approval in frontline maintenance in ovarian cancer.

Billing it­self as the first AI biotech to launch hu­man tri­als, Re­cur­sion adds $121M C round

Billing itself as the first AI biotech with programs in the clinic, Salt Lake City-based Recursion now has a $121 million bankroll to start gathering human data to see if it’s on the right track. 

“We’re trying to build this discovery engine,” Recursion CEO Chris Gibson tells me ahead of the C round news. “We now have the first two programs in the clinic.” And that, he adds, qualifies as a first for any AI establishment “that actually have something in the clinic.”

FDA bats back As­traZeneca's SGLT di­a­betes drug for Type 1 di­a­betes — block­ing a class on safe­ty fears

The FDA has just fired its latest salvo at the SGLT class of diabetes drugs, blowing up some commercial opportunity at AstraZeneca as part of the collateral damage.

The pharma giant reported early Monday that the FDA has rejected its blockbuster drug Farxiga for Type 1 diabetes that can’t be controlled by insulin. And while the pharma giant maintained its usual grim silence in the face of a setback, this one should be easy to interpret.

Jonathan Symonds [via HSBC]

GSK to tap Jonathan Symonds as chair­man, lever­ag­ing Big Phar­ma ex­pe­ri­ence for con­sumer biz deal

Six months into its search for a new board chairman, GlaxoSmithKline has apparently found the perfect candidate in a seasoned executive groomed at AstraZeneca and Novartis. Jonathan Symonds is in the final stages of being appointed, Bloomberg reports.

In January Sir Philip Hampton announced his intention to step down and make way for a new leader to oversee the consumer health joint venture GSK is setting up with Pfizer. The deal — announced a month prior — would spin out the unit formerly headed by GSK CEO Emma Walmsley and merge it with the equivalent division at Pfizer to create a new entity to be listed separately.