The Readout Damian Garde

Good morning! We’re taking the rest of the week off in the name of July 4. The Readout will be back in your inbox first thing Monday.

Real-world evidence partisans now have data to fight about

For years, biopharmaceutical futurists have pointed to a day when so-called real-world evidence, culled from sources like health records and insurance claims, could replace the costly gold standard that is the randomized clinical trial, an idea that has raised the hackles of more than a few skeptics. Now, thanks to the FDA and a company called Aetion, the two sides have a pilot study to squabble over.

As STAT’s Matthew Herper reports, Aetion put its computational power to the test by trying to replicate a study comparing two diabetes treatments. The result, according to company co-founder Dr. Sebastian Schneeweiss of Brigham and Woman’s Hospital, was “quite an achievement,” one that “elevated the conversation from ‘real-world evidence is all bad’ to 'let’s have a differentiated conversation about this.’”

But outside experts took issue issue with the idea, questioning whether Aetion’s approach could ever be useful in reality. One even called the inaugural attempt “dangerous.”

Read more.

TILs are once step closer to CAR-T's glory

Iovance Biotherapeutics got welcome news from the FDA. An ongoing clinical trial can be used to seek regulatory approval for a bespoke cell therapy to treat women with advanced cervical cancer, the biotech said yesterday.

The Iovance treatment is custom-built around immune cells called tumor infiltrating lymphocytes, or TILs. They are different from the more well-known and personalized T cell therapies called CAR-Ts that are approved to treat certain types of blood cancers. 

At the ASCO meeting in June, Iovance reported deep and durable tumor responses from a mid-stage clinical trial. Iovance suggested enrolling more cervical cancer patients into the study to collect enough data to seek approval. The FDA agreed, which means Iovance could be ready to file in the second half of 2020.

The need-to-know on biotech’s future in sickle cell

For decades, thousands of patients with sickle cell disease have had few options to relieve the excruciating pain brought on by the inherited blood disorder. But that paradigm could radically shift in the near future, with new drugs and gene therapies rapidly approaching the market.

And we’re going to talk about it with a physician expert. Our July STAT Expert Advantage conference call will dig into the current standard of care and how investigational therapies from Global Blood Therapeutics, GlycoMimetics, and Bluebird Bio could alter the treatment landscape.

It’s on July 23, and you can sign up right here.

When pharma CEOs go on a ‘listening tour’

There was a time when Novo Nordisk, despite its billions in annual revenue, slipped beneath the radar when the subject was anti-pharma scorn. Then came escalating angst over the rising cost of insulin, which happens to be a banner Novo product, and now CEO Lars Fruergaard Jorgensen has embarked on a self-described listening tour to address the outrage.

Speaking to STAT’s Ed Silverman, Jorgensen described the effort as a sign of his “desperation.” He’s read countless stories about patients struggling to pay for their insulin and listened to politicians shame his company as heartless profiteers. “And I compare that with the reality, where we have seen over the last five years that our prices have gone down,” Jorgensen said. “Our net price — what we get — is decreasing rapidly.”

The disconnect, he acknowledged, is complicated. And it’s not merely a matter of lowering list prices.

Read more.

More reads

  • Health tech companies often flop. But this researcher’s track record suggests there’s a strategy for success. (STAT)
  • 5 names to know in digital therapeutics: the people behind apps that work like medicines. (STAT Plus)
  • Amarin raises sales guidance for heart drug Vascepa, hires more sales reps for larger marketing push. (STAT)
  • Elizabeth Warren blasts former FDA commissioner for joining Pfizer’s board. (STAT)

Thanks for reading! Until next week,


Wednesday, July 3, 2019


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