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How insurers are handling Leqembi coverage, what whole genome sequencing revealed about sick babies, & why the 988 crisis line needs help

July 12, 2023
Reporter, Morning Rounds Writer
Good morning. Today we learn about how that new Alzheimer's drug might be covered and monitored, why Takeda withdrew its dengue vaccine from FDA consideration, and where the 988 crisis line could use help.

Health policy

Now that the new Alzheimer's drug is approved, how are insurers handling it?

Last week the FDA gave full, traditional approval to Leqembi, a new treatment developed by Eisai and Biogen to slow the progression of Alzheimer's disease. The drug has modest benefits but potentially serious side effects for some, including brain swelling and bleeding, so insurers are finalizing their plans to cover the drug as well as associated scans and diagnostic tests. 

Medicare told STAT's Rachel Cohrs it will cover most patients eligible for Leqembi, plus brain scans and genetic testing to screen for and monitor potential side effects. Medicare now covers one amyloid PET scan per lifetime, but a new proposal is coming "soon," an agency spokesperson said. As a condition of coverage, Medicare has already rolled out a new patient registry to collect more information from physicians prescribing Leqembi. "I am frankly surprised that they're up and ready," Lon Schneider, director of the California Alzheimer's Disease Center, said. Read more.


public health

Takeda withdraws dengue vaccine from FDA process

Takeda is voluntarily withdrawing its application to the FDA for approval of its dengue vaccine, throwing into question whether it will be licensed in the U.S. Already approved in the E.U., the U.K., Brazil, Argentina, Indonesia, and Thailand, the vaccine protects against a disease nicknamed breakbone fever because of the painful infection it triggers. One other vaccine is licensed in the U.S., GSK's Dengvaxia, but its use is restricted to children age 6 to 16 living in dengue-endemic areas — principally Puerto Rico — who've previously had a laboratory-confirmed case of dengue.

Dengue is a challenging virus because infection with one of its four serotypes does not protect against infection by another. In some cases a previous exposure through infection or vaccination can lead to more serious disease later. Takeda said its decision was based on the FDA's request for additional data. STAT's Helen Branswell has more.  


genetics

Study of DNA sequencing in sick babies resurfaces challenges of interpretation

Researchers are testing DNA sequencing in the tiniest of patients, certain the testing has a role to play in diagnosing and treating seriously ill babies in the first year of life. But a new study in JAMA underscores how reading the genome is one challenge, but interpreting it is another. In the trial, 400 hospitalized infants had their entire genomes sequenced; they were also tested with a targeted gene panel. 

Whole-genome sequencing, while slower, outperformed the targeted panels in pinpointing the cause of an infant's symptoms, with a 49% diagnosis rate compared to 27% for the narrower panel. For a fifth of participants, that led to changes in their treatment. But the study also found that labs often interpreted genetic information differently in 43% of cases. "You think, 'Well, if you sequence it, you must know.' And that's not the case," study co-author Jill Maron said. STAT's Jonathan Wosen explains.



Closer Look

One year in, the 988 crisis line also needs help

CallCenter_Illustration_MollyFerguson_070523Molly Ferguson for STAT

In the year since the new, easy-to-remember 988 number went live, call center staff have worked hard to keep up with increasing calls while also building new infrastructure to grow. There's more to do, STAT's Theresa Gaffney reports.

  • Staffing: "You have absolutely no idea if people are going to sit idly by or they're going to be overtaxed and know how to support both of those scenarios," said Eileen Davis at Call2Talk in Massachusetts.
  • Funding: "We can handle these huge national disasters in crisis," said Rachelle Pellissier at Crisis Support Services of Nevada, "[but] dealing with not having the right funding or funding withheld or the loss of funding? Now there is some frickin' stress."
  • Responding: "There are instances where, unfortunately, the call with 911 ends with them saying, 'unless you get more information [from the caller], we can't really help you,'" said Danielle Silverstein at the Long Island Crisis Center.

health 

Cancer in kids is rare, but rates have ticked up

Cancer in children is sometimes called a rare disease because it is so uncommon. It's still the leading cause of death from disease under age 20, so any increase attracts attention. A new CDC report in the Journal of the National Cancer Institute says cases rose from 13,327 in 2003 to 14,381 in 2019, peaking at 15,624 in 2015. The reasons why could be more electronic pathology reporting to cancer registries or changing risk factors, such as assisted reproductive technology and maternal age.

"It's a small increase of 0.5% per year on average," David Siegel, a pediatric oncologist and epidemiologist at the CDC, told STAT's Angus Chen. "The main message is that pediatric cancer is very rare. As a parent, I take comfort knowing that these changes are relatively small and it's unlikely for any child to develop these cancers, but it's important to keep track of cancer trends going forward."


Global health

Opinion: New sickle cell therapies must go where they're needed the most

A milestone for sickle cell disease treatment is nearly here, with two gene-based treatments expected to win regulatory approval this year. Yet for people enduring the debilitating blood disorder in sub-Saharan Africa, it will be a hollow victory. More than 60% of the 120 million people worldwide with this disease live in Africa, where 1,000 babies are born every day with two copies of the sickle hemoglobin gene that causes the disorder.

For them, the gene therapy's expected multimillion-dollar price tag will be far out of reach. Prices of this magnitude are unsustainable if gene therapy is to move beyond treating just a handful of patients, Jayasree Iyer, CEO of the Access to Medicine Foundation, writes in a STAT First Opinion. "Rolling out a new generation of sickle cell cures without a strategy to get them to those populations with the greatest need is not acceptable." Read more.


On this week's episode of the "First Opinion Podcast," STAT's Torie Bosch chats about a new gene therapy with Hawken Miller, a journalist and a content strategist for Cure Duchenne, an organization started by Miller's mother when he was diagnosed with Duchenne at a young age. Listen here.


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What we're reading

  • The U.S. scientist at the heart of Covid-19 lab leak conspiracies is still trying to save the world from the next pandemic, Time
  • What does 'brain dead' really mean? The battle over how science defines the end of life, Nature
  • 'We hope to break the logjam': Economists pitch ideas for minimizing future drug shortages, STAT
  • Sex, drugs and spreadsheets: Dr. Glazer treats Wall Street's addiction surge, Wall Street Journal
  • Eisai executive who helped lead development of Alzheimer's drug to retire, STAT

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