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Thursday, July 13, 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.

CAR-T's coronation

It took about seven hours, but a panel of expert FDA advisers gave a unanimous thumbs up to Novartis' pioneering CAR-T therapy, setting the stage for a landmark approval.

Despite the relative ho-humminess of the panel — which heavily focused on nitty-gritty safety issues before ending in a bout of verbal high fives — Novartis' success is a fairly big deal. From the FDA's perspective, CAR-T represents an entirely novel treatment modality, and the first approval will usher in a new era, with all the attendant promise, worry, hype, and hand-wringing.

If you're particularly CAR-T inclined (and perhaps a tad sadistic), you can relive the panel discussion through the our live blog archive. Have thoughts on the next event we should cover live? Drop us a line.

The FDA just got less likely to grind to a halt

The House yesterday easily approved the five-year reauthorization of the agreements that spell out how much drug and device manufacturers pay to help keep the lights on at FDA, STAT's Erin Mershon reports. Next, the package heads to the Senate, which will have to approve it in the next three weeks or risk the FDA sending out its first layoff notices on Aug. 1. 

Though the package has broad, bipartisan support in the Senate, the next vote may not be as straightforward.  

Partisan politics surrounding Republican efforts to repeal and replace Obamacare could make Democrats reluctant to fast-track another health care priority. It's also likely that some senators will want to offer amendments that aim to bring down the high cost of prescription drugs or make it easier for patients with life-threatening illnesses try experimental treatments. 

Read more on STAT Plus.

Sponsor content by The 2017 STAT Wunderkinds

Seeking young scientists with big futures

STAT's on a mission to find the brightest young minds working in science today. The 2017 STAT Wunderkinds is an editorial award that will celebrate these brilliant and innovative young scientists for their work in academia, industry, and clinical settings. Don't miss out on your opportunity to celebrate the future of science nominate your Wunderkind today!

Does a drug have to cost the same for every patient?

Do you gamble on the masses or do you focus on a small but lucrative group of customers? That’s central to the debate around indication-based pricing, which is the topic of discussion in a new New England Journal of Medicine article — and a smart piece in the Wall Street Journal

WSJ takes a look at the anti-inflammatory drug Ilaris, from Novartis. A study may have broken open the market for Ilaris — expanding the indication from rare inflammatory diseases to the a much broader cardiovascular market. But if it’s positioned as a drug for a large population, the $16,000 price tag won’t cut it. So should Novartis lower the cost, and risk its lucrative rare disease revenue stream? Or might it be possible to price the drug differently for different indications?

The NEJM piece takes a look at that concept. Its conclusion? It's a tantalizing prospect for drug makers, but perhaps a raw deal for patients.

Or, as the authors write: “…simple economics makes it clear that relative to uniform pricing, indication-based pricing results in higher prices for patients who benefit the most, higher utilization by patients who benefit the least, higher overall spending, and higher manufacturer profits.”

Validating the big data approach to drug development

In silico drug development just got a healthy bit of validation: UCSF researchers used some nifty computational methodology to probe several oncologic, genomic, and pharmacologic databases to spit out some promising drugs. 

They found, for instance, that pyrvinium pamoate, a long-approved drug used typically in treating parasites, could be effective in treating liver cancer. So far, they're seeing efficacy in mice.

Read more on STAT Plus.

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.

gene therapy, (n.): A once-definite phrase that has since nebulously expanded to cover, it seems, all of biotech? That includes CAR-T, modified viruses, and hey, why not old-fashioned protein therapies?

"First gene therapy — ‘a true living drug’ — on the cusp of FDA approval." — The Washington Post

More reads

  • Is AstraZeneca's CEO about to quit and join Teva? A pair of Israeli newspapers say yes. (Haaretz)
  • Buried in Trump’s executive order on drug pricing is a windfall clause for pharma. (Kaiser Health News)
  • Allergan CEO comes out against aesthetic treatments for teens in debate-sparking blog post. (FiercePharma)
  • FDA gets good marks on compassionate use, except for... (STAT Plus)

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

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Thanks for reading! Until tomorrow,

Damian & Meghana

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