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

Charles Baum, Mirati CEO

Mi­rati plots a march to the FDA for its KRAS G12C drug, breath­ing down Am­gen’s neck with bet­ter da­ta

Mirati Therapeutics $MRTX took another closely-watched step toward a now clearly defined goal to file for an approval for its KRAS G12C cancer drug adagrasib (MRTX849), scoring a higher response rate than the last readout from the class-leading rival at Amgen but still leaving open a raft of important questions about its future.

Following a snapshot of the first handful of responses, where the drug scored a tumor response in 3 of 5 patients with non-small cell lung cancer, the response rate has now slid to 45% among a pooled group of 51 early-stage and Phase II patients, 43% — 6 of 14 — when looking solely at the Phase I/Ib. Those 14 patients had a median treatment duration of 8.2 months, with half still on therapy and 5 of 6 responders still in response.

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In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

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Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Michel Vounatsos, Biogen CEO (via YouTube)

Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: FDA anoints Gilead­'s remde­sivir as the Covid-19 treat­ment win­ner, hand­ing down full ap­proval — de­spite some deep skep­ti­cism

Seven months into the Covid-19 pandemic, the race to develop a treatment for the disease that’s proved to be the biggest health crisis in a century has an officially designated winner: Gilead. And they’re picking up the prize — worth billions in peak sales — despite a major study that concluded the drug was no help in reducing the number of people who die from the virus.

The FDA handed down a thumbs-up for remdesivir, the company announced Thursday afternoon, as the drug becomes the first fully approved treatment for Covid-19 in the US. Remdesivir, to be marketed as Veklury, will come with a label for treatment in adults and children older than 12 in Covid-19 cases that require hospitalization.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

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