Califf just squeaked in as FDA commissioner. Now comes the hard part

The next year at the FDA is going to make Robert Califf’s marathon confirmation process look easy, STAT’s Nicholas Florko predicts. The longtime Duke cardiologist, narrowly confirmed by the Senate yesterday, inherits an agency deep in deliberations over a slew of controversial applications. Califf likely won’t weigh in on the minutiae of those decisions — in keeping with standard practice — but these decisions will have huge consequences for his tenure at the FDA. He will have to explain the decisions to the public, and he will take the blame for any misstep the agency makes. From pediatric Covid vaccines to vaping to ALS drugs, Nick lays out what’s at stake in six approval decisions. Read more.

Covid vaccine in pregnancy helps protect babies

Covid-19 vaccination is recommended during pregnancy, but a new CDC report says it can also help protect babies from hospitalization for up to six months. Results from the study, which looked at two-dose regimens of mRNA vaccines while the Delta and Omicron variants were widespread, fit with previous research showing that other vaccinations pass on antibodies against disease to infants. The new study found that 84% of babies who ended up in the hospital for Covid infections were born to mothers who had not been vaccinated. Another CDC report released yesterday said that at the Omicron variant’s peak, hospitalization rates among children and adolescents were four times as high as rates during Delta’s peak, with higher rates among children too young to be vaccinated.

When newly public companies trade ‘below cash,’ are they in trouble?

One in four biotechs that went public in 2020 are trading below cash, according to a new STAT analysis of data from the financial database provider Sentieo. The indicator — which means a company has more cash on hand than its overall public valuation — seems like further, glaring evidence that too many biotechs raced to market in 2020, a favorite hypothesis among investors whose life science portfolios have plummeted this year. “For a company to trade below cash is like the investing equivalent of having a very bad credit score. It is a sign that investors believe you are headed towards trouble and are at a risk of being unable to create any value for your investors,” investor Brad Loncar told STAT’s Kate Sheridan. But it’s not always so clear cut, others warn. Read more in STAT+.

Closer look: A family carried a rare mutation that should have been lethal. What kept them alive?

(Alex Hogan/STAT)

How could an entire family live with a disease-causing gene that proved universally fatal in lab animals? The genetic mutation caused a congenital heart defect that Cecilia Lo of the University of Pittsburgh said means, “If you have it, you’re pretty much dead.” But a family living about an hour away from her lab was very much alive. Lo wondered if, against all odds, they’d inherited not just one but two ultra-rare mutations, the second shielding them from the deadly effects of the first. It seemed stunningly improbable — but that is, in fact, what she and her colleagues showed in a paper published yesterday in Cell Reports Medicine. STAT’s Eric Boodman tells the story of the family’s rare legacy. “I was like, ‘I don’t know what we all did in a past life. But we got cursed with something,” Nicole Burns said. Read more.

Dropping race-based equation reclassifies 5 million people with kidney disease, study estimates

Race-based algorithms to measure health are much-maligned as both outdated and wrong because they rely on social constructs that do not reflect genetic difference. Measuring kidney function has been a flashpoint because Black Americans are four times more likely than white Americans to have kidney failure but are less likely to receive kidney transplants. Last fall experts advanced a new approach to diagnose kidney function that excludes race, one that two studies also published last fall said were more accurate in Black patients. Yesterday a study in JAMA Network Open comparing old and new guidelines estimates that using the new equations would move more than 5 million U.S. adults into a new kidney function classification, mostly in the moderate stages. About 1 million Black people moved into a more severe category, and about 4.5 million non-Black people moved into a less severe category.

Opinion: Barriers to gender-affirming care need to fall

Medicine gets it wrong both when it requires a diagnosis of gender dysphoria — distress over assigned gender — before delivering gender-affirming care and when it assumes psychological distress defines being transgender (and that gender-affirming care can fix it), researchers assert in a commentary posted yesterday in Cell Reports Medicine. They recommend discontinuing the use of the gender dysphoria diagnosis as an inclusion criterion for accessing gender-affirming care, arguing it will focus clinical attention on a patient’s readiness for medical intervention, rather than their ability to validate their gender identity. “A model that instead centers on patient readiness could allow transgender individuals to both seek desired gender-affirming care as well as confront mental health issues, related or unrelated to their being transgender,” the researchers write.

What to read around the web today

A woman is cured of HIV using a novel treatment. New York Times
Clinicians are leaving their jobs at mental health centers amid rising demand. Boston Globe
Pharma companies rarely default on debt, but it remains a risk factor for investors. STAT+
Their bionic eyes are now obsolete and unsupported. IEEE Spectrum
The seven habits of Covid-resilient nations. The Atlantic