Faster, prici­er and of­ten worth­less: Study slams reg­u­la­tors for wave of ques­tion­able can­cer drug OKs

The ex­plo­sion of new can­cer drugs that’s oc­curred over the past decade in­cludes some ma­jor stand­outs that in­clude ther­a­pies that have reg­is­tered some jaw-drop­ping re­sults in clin­i­cal tri­als. But when re­searchers at King’s Col­lege Lon­don and the Lon­don School of Eco­nom­ics took a step back and ex­am­ined 68 can­cer in­di­ca­tions ap­proved in Eu­rope over a 5-year pe­ri­od through 2013, they found that many were OK’d on on­ly flim­sy da­ta de­rived from un­re­li­able tri­al de­signs and at least 10 of these OKs have nev­er proved to have any re­al val­ue for pa­tients.

Kick­ing back at in­dus­try’s crit­i­cism of groups like NICE, which of­ten balk at cov­er­ing high-priced can­cer drugs, the bio­phar­ma crit­ics say that the vogue of fast-track­ing on­col­o­gy ther­a­pies has put a long line­up of drugs in­to reg­u­lar use, even though the man­u­fac­tur­ers haven’t pro­duced sol­id ev­i­dence of ef­fi­ca­cy four or more years lat­er.

Their study was pub­lished in the BMJ.

Court­ney Davis

The two au­thors — Court­ney Davis at King’s Col­lege in Lon­don, and Huseyin Naci of LSE Health — say 39 in­di­ca­tions (57%) were sanc­tioned “with­out ev­i­dence of a sur­vival or qual­i­ty of life ben­e­fit.” And they ex­co­ri­at­ed the use of sur­ro­gate end­points, say­ing they are un­re­li­able at best.

While 24 of these 68 in­di­ca­tions of­fered ev­i­dence for in­creased sur­vival, the av­er­age was less than 3 months. And on­ly 7 of 68 pro­vid­ed da­ta demon­strat­ing that they im­proved qual­i­ty of life.

“When ex­pen­sive drugs that lack clin­i­cal­ly mean­ing­ful ben­e­fits are ap­proved and re­im­bursed with­in pub­licly fund­ed health­care sys­tems, in­di­vid­ual pa­tients may be harmed, im­por­tant re­sources wast­ed, and the de­liv­ery of eq­ui­table and af­ford­able care is un­der­mined,” the re­searchers say. They added that while reg­u­la­tors have the au­thor­i­ty to pull a drug back from the mar­ket if it doesn’t demon­strate ef­fi­ca­cy, they don’t do it.

Can­cer Re­search UK cau­tioned, though, that the sys­tem on ap­prov­ing and pay­ing for can­cer drugs has un­der­gone some change in re­cent years, with a greater em­pha­sis on some of the weak spots high­light­ed in the study.

Of course, many of these same drugs are al­so ap­proved in the US. And the au­thors’ list of ques­tion­able drugs cov­ers some block­busters. It in­cludes:

→ Avastin, OK’d for ovar­i­an can­cer by the EU in 2011, they say, slap­ping re­searchers for switch­ing from over­all sur­vival to pro­gres­sion-free sur­vival.

Vinay Prasad Wash­ing­ton Post

→ Her­ceptin al­so comes in for crit­i­cism, with the re­searchers flag­ging an ap­proval for breast can­cer long be­fore it failed to demon­strate a sur­vival ben­e­fit.

→ Cel­gene’s Po­m­a­lyst was OK’d in Eu­rope af­ter reg­u­la­tors crit­i­cized their com­par­i­son of the drug com­bined with low dose dex­am­etha­sone against high dose dex­am­etha­sone.

“The ex­pense and tox­i­c­i­ty of can­cer drugs means we have an oblig­a­tion to ex­pose pa­tients to treat­ment on­ly when they can rea­son­ably ex­pect an im­prove­ment in sur­vival or qual­i­ty of life,” in­dus­try crit­ic Vinay Prasad told CRUK. The find­ings sug­gest “we may be falling far short of this im­por­tant bench­mark”

Tesla and SpaceX founder Elon Musk gestures to the audience after being recognized by President Trump following the successful launch of a Falcon 9 rocket at the Kennedy Space Center. (via Getty Images)

Tes­la chief Elon Musk teams up with Covid-19 play­er Cure­Vac to build 'R­NA mi­cro­fac­to­ries'

Elon Musk has joined the global tech crusade now underway to revolutionize vaccine manufacturing — now aimed at delivering billions of doses of a new mRNA vaccine to fight Covid-19. And he’s cutting right to the front.

In a late-night tweet Wednesday, the Tesla chief announced:

Tesla, as a side project, is building RNA microfactories for CureVac & possibly others.

That’s not a lot to go on. But the tweet comes a year after Tesla’s German division in Grohmann and CureVac filed a patent on a “bioreactor for RNA in vitro transcription, a method for RNA in vitro transcription, a module for transcribing DNA into RNA and an automated apparatus for RNA manufacturing.” CureVac, in the meantime, has discussed a variety of plans to build microfactories that can speed up the whole process for a global supply chain.

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Elias Zerhouni (Photo by Vincent Isore/IP3/Getty Images)

Elias Zer­houni dis­cuss­es ‘am­a­teur hour’ in DC, the de­struc­tion of in­fec­tious dis­ease R&D and how we need to prep for the next time

Elias Zerhouni favors blunt talk, and in a recent discussion with NPR, the ex-Sanofi R&D and ex-NIH chief had some tough points to make regarding the pandemic response.

Rather than interpret them, I thought it would be best to provide snippets straight from the interview.

On the Trump administration response:

It was basically amateur hour. There is no central concept of operations for preparedness, for pandemics, period. This administration doesn’t want to or has no concept of what it takes to protect the American people and the world because it is codependent. You can’t close your borders and say, “OK, we’re going to be safe.” You’re not going to be able to do that in this world. So it’s a lack of vision, basically just a lack of understanding, of what it takes to protect the American people.

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George Yancopoulos (Regeneron)

UP­DAT­ED: Re­gen­eron co-founder George Yan­copou­los of­fers a com­bat­ive de­fense of the po­lice at a high school com­mence­ment. It didn’t go well

Typically, the commencement speech at Yorktown Central School District in Westchester — like most high schools — is an opportunity to encourage students to face the future with confidence and hope. Regeneron president and co-founder George Yancopoulos, though, went a different route.

In a fiery speech, the outspoken billionaire defended the police against the “prejudice and bias against law enforcement” that has erupted around the country in street protests from coast to coast. And for many who attended the commencement, Yancopoulos struck the wrong note at the wrong time, especially when he combatively challenged someone for interrupting his speech with a honk for “another act of cowardness.”

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Vas Narasimhan, Novartis CEO (Patrick Straub/​EPA-EFE/​Shutterstock)

No­var­tis pays $678M for kick­back scheme as Vas Narasimhan tries to dis­tance phar­ma gi­ant from shady be­hav­ior

Novartis has reached another large settlement to resolve misconduct allegations, agreeing to pay more than $678 million to settle claims that it had spent hundreds of millions of dollars on lavish dinners, so-called speaking fees and expensive alcohol “that were nothing more than bribes” to get doctors to prescribe Novartis medications.

The top-shelf alcohol and lavish meals included a $3,250 per person night at Nobu in Dallas, a $672-per person dinner at Washington DC’s Smith & Wollensky and a $314 per person meal at Sushi Roku in Pasadena, according to the Justice Department complaint. There were at least 7 trips to Hooters and fishing trips in Alaska and off the Florida coast. Each of these events were supposed to be “speaker programs” where doctors educated other doctors on a drug, but the DOJ alleged many were “bogus” wine-and-dine events where the drug was barely mentioned, if at all.  (“Nobody presented slides on the fishing trips,” the complaint says.)

Sec­ond death trig­gers hold on Astel­las' $3B gene ther­a­py biotech's lead pro­gram, rais­ing fresh con­cerns about AAV

Seven months after Astellas shelled out $3 billion to acquire the gene therapy player Audentes, the biotech company’s lead program has been put on hold following the death of 2 patients taking a high dose of their treatment. And there was another serious adverse event recorded in the study as well, with a total of 3 “older” patients in the study affected.

The incidents are derailing plans to file for a near-term approval, which had been expected right about now.

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An ex­pe­ri­enced biotech is stitched to­geth­er from transpa­cif­ic parts, with 265 staffers and a fo­cus on ‘new bi­ol­o­gy’

Over the past few years, different teams at a pair of US-based biotechs and in labs in Japan have labored to piece together a group of cancer drug programs, sharing a single corporate umbrella with research colleagues in Japan. But now their far-flung operations have been knit together into a single unit, creating a pipeline with 10 cancer drug development programs — going from early-stage right into Phase III — and a host of discovery projects managed by a collective staff of some 265 people.

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New stan­dard of care? FDA hands Pfiz­er, Mer­ck KGaA an OK for Baven­cio in blad­der can­cer

The breakthrough therapy designation Pfizer and Merck KGaA notched for Bavencio in bladder cancer has quickly paved way for a full approval.

The PD-L1 drug is now sanctioned as a first-line maintenance treatment for patients with locally advanced or metastatic urothelial carcinoma, applicable in cases where cancer hasn’t progressed after platinum-containing chemotherapy.

Petros Grivas, the principal investigator of the supporting Phase III JAVELIN Bladder 100, called the approval “one of the most significant advances in the treatment paradigm in this setting in 30 years.”

Look­ing for 'ex­ter­nal in­no­va­tion,' Boehringer In­gel­heim re­serves $500M+ for new Shang­hai hub

Now that Boehringer Ingelheim’s bet on contract manufacturing in China has paid off, the German drugmaker is anteing up more to get into the research game.

Boehringer has set aside $507.9 million (€451 million) for a new External Innovation Hub to be built in Shanghai over five years. The site will become one of its “strategic pillars” as the team strives to get 71 approvals — either for new products or indications — by 2030, said Felix Gutsche, president and CEO of Boehringer Ingelheim China.

No­vavax snags Ben Machielse for CMC and pro­motes a trio of staffers; Mar­ty Du­vall lands an­oth­er CEO post at On­copep­tides

Novavax has been making waves recently by securing a $384 million commitment from CEPI to cover R&D and manufacturing for its Covid-19 vaccine while also spending $167 million on a 150,000 square-foot facility. The Maryland biotech continues to shore up its leadership team as well, bringing in Ben Machielse as their EVP of CMC just a couple weeks after nabbing AstraZeneca vet Filip Dubrovsky as their new CMO. Machielse was president and CEO of Vtesse from 2014-17, and before that, he also spent more than 11 years at MedImmune and was EVP of operations for the back half of his tenure.

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