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The Readout Damian Garde

PhRMA’s top lobbyist is on the way out

As the pharmaceutical industry confronts some of the biggest threats to the status quo in Washington in recent memory, its chief lobbying group is drawing on a war chest to get its message out to Congress. Now, though, PhRMA is about to lose its lead messenger.

Rodger Currie, PhRMA's top lobbyist, is leaving the organization at the end of August, STAT's Washington correspondent Nicholas Florko reports. The news was announced internally by the association’s president, Steve Ubl, in an internal email sent late yesterday afternoon. Currie has chosen to “pursue new opportunities.”

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Will Canada run out of drugs?

Should you ever read your partner's diary? And what does Pepe the Frog know about genomics?

We discuss all that and more on the latest episode of “The Readout LOUD,” STAT’s biotech podcast. First, STAT's Nicholas Florko joins us to explain what President Trump's drug importation plan means and, perhaps more important, what it does not. Then we pick apart the surprising union of Pfizer and Mylan, two companies with strikingly divergent cultures. Finally, computer scientist Jeremy Blackburn joins us to discuss a fascinating new study about how racists are using 23andMe and Ancestry on the Internet.

You can listen to the episode here. To listen to future episodes, be sure to sign up on iTunes, Stitcher, Spotify, or wherever you get your podcasts.

The complicated work of putting a valuation on life

It’s pretty uncontroversial to say that the value of a given drug depends on just how much it helps people. A lifesaving therapy is worth one price; a moderately effective treatment is worth another. But the business of actually assigning numbers to that idea — and thus deciding whether a drug is cost-effective — injects intense human emotion into a quantitative conversation.

That all came to a head at the most recent meeting of the Institute for Clinical and Economic Review, a nonprofit that tasks itself with determining what a drug is actually worth. As STAT’s Kate Sheridan reports, ICER applied its rubric to treatments for Duchenne muscular dystrophy. But for the patient advocates in the room — most of whom were parents of children with DMD — simply asking the question sounded like putting a dollar value on human life.

“It’s an extremely uncomfortable position,” said Mindy Leffler, a DMD parent who spoke during the meeting. “How am I supposed to make some comment on the value of my son’s life?”

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The quest for better lab mice could come down to the gut

Mice are indispensable contributors to the cause of scientific advancement, but they’re not always the most trustworthy guides. Countless new therapies have cured murine disease only to hopelessly fail when tried on actual humans.

That might be in part because lab mice lack the microbiome diversity of everyday creatures. As STAT’s Shraddha Chakradhar reports, a group of scientists crafted a way to breed what they call “wildling mice,” test subjects who retain their genetic engineering but inherit a naturalistic microbiome from outside-the-lab surrogates.

Scientists exposed them to a pair of therapies that had previously showed promise in mice but proved useless for humans and found that the hybrid creatures appeared more predictive than their forebears. 

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Vertex will have an interesting 2020

Vertex Pharmaceuticals just beat Wall Street’s quarterly sales estimates and is on pace to turn its franchise of cystic fibrosis medicines into a $10 billion enterprise. But nothing lasts forever, and so investors have begun to ask: What will Vertex look like after its CF business has peaked?

We’ll start to get some answers next year. In 2020, the world will see mid-stage data on VX-814, a treatment for the rare lung disease AAT deficiency, and early results on VX-147, meant to treat a rare kidney disease abbreviated FSGS. Next year could also bring initial results from Vertex’s genome editing therapy for sickle cell disease and beta thalassemia, and 2020 could finally be the year Vertex decides which of its in-development pain treatments will advance to late-stage trials.

Vertex has spent the last few years keeping its promises in CF and maintaining irons in the fire for the future. A year from now, under new CEO Reshma Kewalramani, the company will start to learn which might actually pan out. 

More reads

  • The untold story of the ‘circle of trust’ behind the world’s first gene-edited babies. (Science)
  • Vas Narasimhan of Novartis: ‘We are not at all prepared for a pandemic.’ (New York Times)
  • Federal judge allows pharma to proceed with lawsuit against California transparency law. (STAT Plus)
  • Blue-blooded crabs at heart of pharma dispute on drug testing. (Reuters)

Thanks for reading! Until next week,


Friday, August 2, 2019


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