Friday, November 18, 2016

The Readout by Damian Garde & Meghana Keshavan

Welcome to The Readout, where we keep you on top of all the biotech news. Follow us on Twitter: @damiangarde@megkesh, and @statnews.

How to weed out feeble cancer drugs

Clinical trials exist in a bubble, where data (and patient pools) can be selected, sorted, and tweaked to help design the best possible readout. So is it possible to gauge whether a cancer drug’s going to be effective in the real world? 

Unclear, according to an opinion piece in JAMA this week. A smattering of cancer drugs are truly transformative — but by and large, new medications have extended life only marginally. 

The paper’s suggestion? Instead of providing full approval to cancer drugs, the FDA could offer accelerated approval. That’d get new drugs to sick patients while also allowing the FDA to mandate postmarket studies that could validate a drug’s performance in the real world — not just in a pre-selected pool of patients. 

This would generate a heap of clinical data, “and ineffective drugs could later be removed from the market," write Sham Mailankody and Dr. Vinay Prasad. 


Califf for FDA chief, version 2.0?

Agency heads are readying themselves to hang up their hats when Donald Trump moves into the White House.

NIH director Francis Collins told Scientific American that he, “like every other presidential appointee and Senate-confirmed person,” has to send in a resignation letter by December 7.

Same goes for FDA Commissioner Robert Califf.

But Collins hinted he'd be open to staying on, if asked. And BIO Chair Dr. Ron Cohen is calling on Trump to extend Califf's tenure. “Assuming he wants to stay on, one of the things this [Trump] administration could do is reappoint him as head of FDA,” Cohen told RAPS.

Lest that send the wrong message, Cohen hastened to add that he wants to “emphasize that this isn’t because [Califf is] unduly friendly to the industry.” 

How do VCs make sure they’re not buying into bunk?

Being an early-stage biotech investor means sifting through a lot of straight-from-the-lab science whose on-paper promise may well fizzle on further examination. That’s a risky proposition.

The “dirty little secret,” as Paulina Hill of Polaris Partners put it during a Wall Street Journal biotech panel, is that “it’s very difficult oftentimes to repeat what has been done” in academic labs.

“I remember when I was a postdoc. You’re usually looking for one good figure to put in a publication,” Hill said. “You may maybe repeat it once, but it’s a whole different level of scrutiny and rigor when you’re trying to develop a therapeutic.”

And so early-stage VCs quietly spend a fair amount of time and money checking and rechecking quote-unquote breakthroughs long before whipping them into companies exposed to the world, enduring “a diamond-in-the-rough kind of process” in which “you spend a lot of time kissing frogs,” Patrick Heron of Frazier Healthcare told WSJ.

“It’s important not just that the mouse model you’re looking at improves but that it improves for the reasons you think it will improve,” Atlas Venture’s Mike Gladstone said. “And for the reasons that really guide your therapeutic hypothesis for the only species we really care about.”

By which he means people.

Sovaldi was the best drug of 2013. Shocked?

Yeah, probably not — but that’s the conclusion of a new drug index from Trinity Partners, a Massachusetts-based life sciences consultancy. Its methodology ranks a drug’s value based on how efficient its R&D process was, as well as whether it’s a commercial success and, well, works.

So based on those metrics, Sovaldi wins. (Small solace for Gilead, perhaps, as it’s had a pretty lousy week.) 

Here’s the top 10: 

More reads

  • How much longer will Gilead wait before buying its way out of the doldrums? (STAT)
  • Intel and the Broad Institute ink a $25 million pact for genomic data analysis. (GenomeWeb)
  • Newt Gingrich, a rumored future director of Health and Human Services, said he will not join the cabinet of President-elect Donald Trump. (Washington Post)
  • FDA approves new pasta shape. (The Onion)

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Thanks for reading! Until next week,

Damian & Meghana

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