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Friday, December 1, 2017

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, where we keep you on top of the latest in biotech. Don't forget to sign up for our pop-up newsletter during the American Society of Hematology conference: ASH in 30 Seconds. Quick reminder, too, that Adam Feuerstein is doing a live chat to preview ASH — and answer your questions — on Monday at 1 p.m. for STAT Plus subscribers. Register here.

Gottlieb's fast track plan

It looks as though FDA chief Scott Gottlieb plans on downgrading the “gold standard” of clinical trials for a few special candidates. Certain drugs that show “clear and outsized treatment effect” might be able to skip a few regulatory steps and achieve approval — with further evaluation conducted largely in post-market study. 

And that’s raised some red flags, including from the ever-bellicose Dr. Vinay Prasad.  

“Randomized trials are needed to understand cancer drugs, the same way we have races in the Olympics to pick the winner,” Prasad told STAT. “We don't have people run on different tracks in their home countries and submit their times, which are compared to Jesse Owens and then used to declare who should really win the 1936 Olympic games.” 

Read more on STAT Plus.

Drug pricing is cool again

After months of handwringing about tax law and health care policy, the world suddenly seems to care about drug prices once more.

First, on Tuesday, a Senate committee was pointed and persistent in its grilling of President Trump's nominee to run HHS, repeatedly prodding him on the issue of increasingly expensive medicines.

Then yesterday the National Academies of Sciences, Engineering, and Medicine came out with eight recommendations on how to make drugs more affordable, pretty much all of which would be repulsive to pharma.

And, as if on cue, Alnylam Pharmaceuticals CEO John Maraganore took the unprecedented step of promising not to raise (beyond inflation) the price of a drug that hasn't even been approved yet, adding that "price increases are an indefensible act."

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Get ready for ASH with our STAT Plus Intelligence Briefing

As you prepare for this year’s American Society of Hematology (ASH) annual meeting, join STAT Reporter Adam Feuerstein for an online intelligence briefing, December 4 at 1PM ET. He’ll look ahead at what he expects at ASH — from new therapies and research to patient care. Questions welcome. Subscribe today to register.

Test tube glioblastomas for personalized medicine

Human micro-brains may help us unravel the most puzzling secrets of glioblastoma: Why these brain cancers appear and grow as they do, and why they are so challenging to treat.  

In the past few years, scientists have created pea-sized brain organoids, grown out of stem cells, that can mimic disease, and serve as a tool to develop new drugs. 

One recent discovery? Organ facsimiles can actually get cancer — and they may be more reliable than mouse models of the same disease. So one brain cancer researcher hopes to grow them to personalize glioblastoma care.

Read more.

The Biotech Devil's Dictionary

There’s a lot of jargon, coded language, and outright nonsense in biotech, and we want to clear up — and celebrate — as much of it as we can through this glossary. Have a phrase to contribute? Email it over. And, for your convenience, we've gone ahead and gathered all the past entries into a one-stop shop.

unlock value (v.): A bit of corporate argot used to describe the process of making money, implying that "value" has been cloistered away by some dastardly villain or is perhaps buried under sediment and thus in need of biotech's answer to fracking.

"While M&A remains Sanofi's top priority in 2017, we performed a sum-of-the-parts analysis to determine if there's value to be unlocked from the company’s separate business units." — Leerink analyst Seamus Fernandez

More reads

  • FDA announces approval, CMS proposes coverage of first breakthrough-designated test to detect extensive number of cancer biomarkers. (Press release)
  • This start-up raised millions to sell 'brain hacking' pills, but its own study found coffee works better. (CNBC)
  • In search of novelty: 2017’s new FDA drug approvals. (LifeSciVC)
  • The Senate’s tax bill is a sweeping change to every part of federal health care. (Vox)

Have a news tip or comment you want to send us?

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Thanks for reading! Until Monday,

Damian & Meghana

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