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The Readout Damian Garde & Meghana Keshavan

Democratizing data with All of Us

For four years, All for Us, the government's $1.5 billion federal initiative, has been collecting health data from 1 million Americans of diverse backgrounds. It’s been a gold mine for insights about medicine — but has also presented a daunting technological challenge given the vast amount of data involved.

As STAT’s Katie Palmer writes, All of Us has worked with the Broad Institute, Vanderbilt University, and Verily to build a tool called Researcher Workbench, a cloud-based analytics platform to provide the technical infrastructure to make sense of these huge datasets. It contains, on the back end, an open-source system called Terra, which is in essence a massive sandbox allowing scientists to play around with gobs of diverse data.  

But as more researchers sign on to use the data, and more data become available, Terra is being put to the test.

Read more.

Another controversial Sarepta decision nears

For the third time, the Food and Drug Administration is nearing an approval-decision deadline for a new medicine from Sarepta Therapeutics to treat patients with Duchenne muscular dystrophy.

As with the previous two Sarepta reviews, the FDA’s decision promises to be controversial because it will be based on the drug’s ability to produce a tiny amount of the muscle protein dystrophin missing in these patients, even though there is no established proof that more dystrophin improves muscle function or slows the progression of the disease. 

The FDA has until Thursday to announce its approval decision for the Sarepta drug, called Amondys 45. If approved, Amondys 45 — joining already-approved Exondys 51 and Vyondys 53 — would extend Sarepta’s commercial reach to approximately 30% of all Duchenne patients. Sarepta revenue this year is expected to grow nearly 30% to $686 million, according to consensus analyst estimates. 

There’s been no hint from FDA that it won’t follow precedent and approve Amondys 45 based on data showing single-digit percent increases in dystrophin production. However, the risk of a rejection increased last January when Sarepta’s experimental gene therapy failed to improve the muscle function of Duchenne patients despite producing large quantities of dystrophin protein.

Most vaccine trials aren’t sharing ethnic or racial data

Covid-19 laid bare the racial and ethnic disparities in the U.S. health care system — which has, in turn, become a key priority for vaccine developers. But a dive into a decade’s worth of vaccine clinical trials shows that Black Americans, American Indians, and people aged 65 and older were underrepresented in trials, STAT’s Nicholas St. Fleur writes. Adult women, by contrast, were overrepresented, according to a new study in JAMA Network Open

And, shockingly, more than 40% of the 230 studies examined by researchers didn’t record participants’ race; 65% didn’t report their ethnicity. 

"This is a massive gap in information, and if we want to improve enrollment in clinical trials and we want to see diversity in clinical trials, we need the data," one of the paper’s authors told STAT. "It’s amazing we don’t have that data." 

Read more.

Vaccine prices vary wildly from country to country

Although nearly 60 million doses of the Covid-19 have been administered in the U.S, most other countries around the globe are lagging. Supplies are an issue, but so is the cost to governments, as NPR points out.

For example, the cost of a single dose of the Oxford-AstraZeneca vaccine is reported to cost just $2.72 in India, $4 in the U.S., and $8.50 in Uganda. But a dose of the vaccine from Sinopharm, which was developed in China, ranges in cost from $18.50 in Senegal to $44 in China to $72.50 elsewhere. 

"These [smaller] countries don't have the purchasing power or the ability to strike bilateral deals," one policy adviser with Doctors Without Borders said. "Obviously, manufacturers are less interested in delivering relatively small quantities to smaller countries than they are delivering larger quantities to countries with larger populations and also bigger purchasing power."

More reads

  • No SPAC slack as more shell companies targeting biotech hit Wall Street. (FierceBiotech)
  • Biotech is acting ‘more and more like’ Bitcoin, Evercore says (Bloomberg)

Thanks for reading! More tomorrow,

Damian

Monday, February 22, 2021

STAT

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