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Wednesday, November 29, 2017

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, where we keep you on top of the latest in biotech. For more in-depth coverage of biopharma, subscribe to STAT Plus. On Twitter: @damiangarde@megkesh, and @statnews.

The ‘treacherous seduction’ of mining failed trials

Among the dirtiest of words in biotech is “subpopulation,” a multisyllabic red flag that companies use when they’re not ready to reach for the white one.

Basically, whenever a trial fails, there’s almost always a group of patients — left-handed Peruvian men who hate the taste of basil, say — in whom it works. Drawing conclusions from such analyses is statistically fraught, to say the least, so why do scientists so often do so?

“One answer — unpleasant but real — is that pharmaceutical companies want to put a positive spin on their drugs, even when the trials fail to show benefit,” writes Dr. Siddhartha Mukherjee, who has personal experience in the subject.

But there’s a “less cynical” answer, too, he writes: Sometimes slicing and dicing data can provide clues on how to design future trials, and sometimes — sometimes — those follow-up studies come up positive, making the whole exercise worthwhile.

Just don’t take your subpopulation analyses to the FDA.

A call for collaboration

Pharma has a productivity crisis. One solution: Support startup growth.  

“Pharmaceutical companies are some of the most conservative organizations on the planet and are typically slow to embrace change,” writes Steve Arlington, president of the nonprofit Pistoia Alliance, which promotes R&D. 

Biopharma development is infinitely more complex than it was in the 80s, when a lot of this stuff was done in-house. These days, it’s not just about acquiring startup technology. Now, multidisciplinary teams — with non-big-pharma members — are critical to the innovation engine, Arlington argues. 

Read more

A viral backlog that could stall gene therapies

Gene therapies are the next big thing. Or at least they could be, if they could get into the clinic. 

The problem: There’s a critical shortage of viral vectors, the New York Times writes. These disabled pathogens are used to deliver genes into patient cells — and there’s a logjam as more and more biotechs clamor to buy them from a limited number of suppliers. The wait can stretch years. 

Novartis, whose CAR-T drug uses these vectors, had to set up a contract years in advance to secure enough virus supply for its drug, Kymriah. And they are paying a premium. Not all biotechs have the same cash — and will simply have to wait.

Meanwhile, at Valeant...

“This isn’t like flipping a light switch,” said Joe Papa, the CEO of Valeant Pharmaceuticals, who is trying to rescue the company from the debt-laden tailspin that has transformed it from the toast of Wall Street into a cautionary tale.

And he’s probably right. Looking at Valeant’s recent history reads like a one-company anthology of the biopharma industry's PR failings. There are controversial price hikes, a series of overpriced acquisitions, accusations of managerial incompetence, and even an accounting scandal.

And Papa, hired about 18 months ago, is here to fix all that. In an interview with The Wall Street Journal, the former Perrigo CEO made it clear that’s no easy task.

“You can’t walk in and say, ‘All good now,’” he said. “It’s going to take some time.” 

More reads

  • Derek Lowe takes a look at the latest (grim) analysis of the drug industry's output. (In the Pipeline)
  • FDA boss aims to close EpiPen loophole. (Forbes)
  • Pfizer's cancer immunotherapy flops in "high-risk" stomach cancer trial. (FiercePharma)
  • Drug makers' reward for success? A piece of paper worth $100 million or more. (SF Business Times)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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