Tuesday, November 14, 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.

Is FDA lowering its standards?

At last night’s STAT Plus event in Washington D.C, a fiery Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, pushed back against Ed Silverman — our Pharmalot columnist — when he suggested FDA was lowering its approval standards in order to speed up drug approvals. (It wasn’t entirely Ed’s fault, this was the theme of the event, after all.)

“I’m irritated by this,” said Woodcock, referring to the widely held narrative that the FDA was dumbing down its regulatory standards.

She insisted FDA was doing no such thing. The decision to approve Sarepta’s Duchenne muscular dystrophy drug? The right one, even if there was a “difference of opinion” within the agency, she said. About Addyi, the female sexual dysfunction drug which critics say doesn’t work, Woodcock argued otherwise, insisting the agency actually listened to women. “There’s no female sexual dysfunction? That’s B.S. Women just don’t complain as much.” On cancer drugs, Woodcock said approvals based on tumor response, and not a proven survival benefit, were perfectly appropriate — and the right thing to do — when patients have no other options.

The qualities of a blockbuster drug

Is there a secret sauce that renders a drug blockbuster-worthy? A new Trinity Partners report takes a look at the factors that contribute to a medication’s commercial success, peeling apart the performance of the 41 drugs approved in 2014. Some top line findings:

Across the board, commercial performance (thank goodness) correlated pretty substantially with therapeutic efficacy. 

The immunotherapy drugs approved in 2014 performed well, largely because of their efficacy and general lack of generic competition. Keytruda was the top-ranker, with Opdivo coming in third, when evaluated for therapeutic value, commercial success, and R&D expense. 

But anti-infectives didn’t do so hot — not because they don’t work, but because the reimbursement structure just isn’t equipped to support these costly, if effective, new drugs. 

And while not all orphan drugs were big successes, some certainly did well — particularly those targeting hemophilia. But mass-market drugs did not perform well, largely because of the breadth of lower-cost competition currently available. 

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Pharma might get another friend in Trump world

President Trump is nominating a former Eli Lilly executive to succeed Dr. Tom Price at Health and Human Services, promising his pick "will be a star for better healthcare and lower drug prices!" That last part might ring as odd in light of Lilly's recent record on the subject of pricing. Humalog, Lilly's banner insulin product, has more than doubled in cost since 2011, a trend that has sparked state investigations and a class-action lawsuit.

It's hard to say how Azar's Lilly pedigree might inform his HHS leadership — that's sure to be a topic at his Senate hearing — but it could be a bad omen for PBMs.

Lilly is among the raft of drug makers to blame rising out-of-pocket costs on the industry's middlemen, saying last year that its own price hikes largely were to pay ever-increasing rebates to the likes of CVS and Express Scripts. If Azar believes PBMs are making too much money with too little scrutiny, as many in Washington do, he could soon be in a position to make their lives more difficult.

A probiotic for immunotherapy

The microbiome seems to yield substantial power over cancer drugs — with some bacteria rendering treatments useless, while other, “good” bugs buoy their efficacy. A new partnership between MD Anderson, the Parker Institute, and Cambridge-based Seres Therapeutics is now working on a bacterial cocktail that might tweak the microbiome so as to improve how immunotherapies work.

Read more on STAT Plus.

CRISPR goes to Washington

A trio of CRISPR experts, including Editas Medicine CEO Katrine Bosley, are due to speak before a Senate committee this morning about all the wondrous benefits and staggering risks that come with gene editing.

It starts at 10 a.m. ET, and you can stream it here. STAT's Ike Swetlitz will be reporting from the hearing, so check back later for a full run-down.

More reads

  • Pfizer provides some clues to its succession plan. (Wall Street Journal)
  • Scientists used CRISPR to edit out PCSK9 in mice. (Reuters)
  • Novartis co-opts Homology’s gene-editing tech for R&D pipeline. (Endpoints)

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Thanks for reading! Until tomorrow,

Damian & Meghana

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