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An impossible choice in sickle cell disease: a cure or fertility

December 6, 2023
Reporter, Morning Rounds Writer
Good morning. Today Megan Molteni tells us how infertility worries will likely temper sickle cell patients' embrace of new gene therapies.

special report

New gene therapies for sickle cell disease come with an impossible choice: a cure or fertility

SickleCellFertility_Illustration_MollyFerguson_120423Molly Ferguson for STAT

For people with sickle cell disease, a chance at freedom from a debilitating genetic disease can mean giving up the chance to have biological children without assistance from reproductive technology. A new genetic therapy, called Casgevy and likely to be approved by the FDA this week, deploys CRISPR to edit the DNA of blood-forming cells so they no longer twist into a sickle shape and wreak havoc, inflicting extreme pain and damaging organs. Most of the 100,000 or so Americans with sickle cell are Black; they have endured a severe shortage of qualified disease specialists and insensitive care in emergency rooms and urgent care settings.

To prepare for Casgevy or an older treatment that depends on a bone marrow transplant from a genetically matched donor, patients must have chemotherapy to wipe out their own faulty cells. That can decimate fertility, too. "No one should have to give up their ability to create a life, if that's what they want, in order to save their lives," patient Marie Tornyenu told STAT's Megan Molteni. Read more.


drug pricing

A new CVS approach to paying for drugs won't lower prices for patients, experts say

CVS Health is promising to simplify how its pharmacies get paid for drugs. But that doesn't mean the drugs will get cheaper, experts told STAT's Tara Bannow. The country's biggest pharmacy chain is switching to a system where pharmacy benefit managers, employers, and other insurers pay for drugs based on the cost of the drug plus a set markup and dispensing fee. Sounds like the model popularized by Mark Cuban of Shark Tank fame, right? 

Not really. The CVS CostVantage program appears designed to pad the company's own bottom line rather than making drugs cheaper for patients and employers, experts said. CVS owns the country's largest pharmacy benefit manager, Caremark, noted Karen Van Nuys of the USC Schaeffer Center for Health Policy and Economics. "It's squeezing the balloon in one place and it gets bigger in another," Van Nuys said. "When you're an integrated entity, you can pick where you want to realize profit." Tara explains.


politics

Senate panel looks to Medicare policy to fix drug shortages

Drug shortages are plaguing the country, with particularly acute problems for people being treated for cancer. Yesterday members of the Senate Finance Committee took their first steps today toward using Medicare payment policy to fix drug shortages. Economic factors — the complicated way Medicare pays hospitals or the dominance of group purchasing organizations that buy hospital drugs —  are implicated in the problem because they force generic drug makers to operate on razor-thin profit margins. That means less-than- proper maintenance of facilities, leading to shutdowns.

Witnesses for the Senate Finance hearing emphasized that solutions should focus on encouraging hospitals to buy from suppliers that maintain resilient supply chains. The committee didn't coalesce around a solution, but several lawmakers from both parties asked about a Medicare policy that would increase payments to hospitals that buy from suppliers that focus on the strength of their supply chains. STAT's John Wilkerson has more.



closer look

Opinion: Here's a plan to get weight-loss drugs to everyone who needs them (and save Medicare, too)

VJHT65DGJGQOZOEYG2IXXP4A2ICydni Elledge/New York Times 

Data on the new class of weight-loss drugs has shown they work well not just for obesity and diabetes, but also for heart disease. But their price has also been jaw-dropping, especially because people often regain weight soon after they stop taking them. Still, Jeremy Shane, former general manager of WebMD/Medscape Editorial writes in a STAT First Opinion, if the goal in the U.S. is to help people be healthier and save Medicare's solvency, too, these drugs are our best bet. 

Providing them will be costly, he says, but so is the status quo. He argues that most of the drugs' value will be realized only if people actually get to Medicare age with less metabolic disease. "It is possible to design pricing tied to patient outcomes with payments to pharmas spread out over many years, even after patent expirations," he says. Read more on how that could play out.


health

For better or for worse: Couples share high blood pressure

If your spouse has high blood pressure, you're more likely to have it, too. A study out today in the Journal of the American Heart Association found this phenomenon is true not just in the U.S. and England, but also in China and India. The prevalence of hypertension among married or partnered heterosexual couples over age 50 was highest in England (47%), then the U.S. (38%), followed by China (21%) and India (20%). While high blood pressure is more common in the U.S. and England, the blood pressure link between couples was stronger in China and India.

The researchers raised the hope that couples might be helped together. Blood pressure can be modified with more physical activity, less stress, and a healthier diet. Two caveats: Only heterosexual couples were included and only one instance per person of a high blood pressure reading (over 140/90) or report of high blood pressure was collected.


reproductive health

Bariatric surgery affects how much weight is gained in pregnancy, study says

With obesity drugs drawing so much attention these days, it's easy to forget about bariatric surgery. But a new study in JAMA Network Open explored weight gain in pregnancy after weight-loss surgery. Looking at more than 12,000 pregnancies in Sweden, the researchers concluded that women with a history of bariatric surgery gained less weight during pregnancy than similar women who hadn't had the surgery. 

Also important was the fact that mothers in the surgery group who were classified as being of normal weight status at the start of pregnancy were at higher risk of not gaining enough weight during pregnancy, and more likely to have babies who were small for gestational age. "Clinical attention to women with history of bariatric surgery and a normal weight status in early pregnancy might be warranted," the researchers write.


On this week's First Opinion podcast, First Opinion Editor Torie Bosch talks with U.S. Rep. Raul Ruiz (D-Calif.) about the country's shortage of infectious disease physicians and what it's like to be a doctor in Congress. Listen here.


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