Health (Page 10)

Heidi Overton has been nominated to lead the Food and Drug Administration at a moment of significant opportunity, but also big challenges. Innovation in medicine has never moved faster, providing more ways for Americans to improve their health and for the United States to advance the health of its population. But the institutions that carry that progress forward are under strain.

Overton has a chance to help bridge that divide — and the experience to do it. She has the opportunity to demonstrate these goals at Thursday’s confirmation hearing in front of the Senate health committee.

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Dr. Oz praised GLP-1s from Eli Lilly’s lab, where he said MFN agreements have drastically cut the price of obesity drugs. Problem is, at least Lilly’s GLP-1s are exempt from the MFN agreements (see below). But who am I to correct people? I incorrectly attributed an AEI post to another conservative think tank in this newsletter last week. Send news tips and correction requests to John.Wilkerson@statnews.com or John_Wilkerson.07 on Signal.

Novo’s (potentially) big win

STAT has written about how Eli Lilly and Novo Nordisk benefited from their deals with the Trump administration to cut obesity drug costs for seniors. It turns out, Novo might have gotten an additional sweetener.

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Good morning. As an avid Pilates goer, I appreciated this story.

The need-to-know this morning

• Roche reported a positive Phase 2 trial of enicepatide (previously known as CT-388) in patients with type 2 diabetes and obesity, with the drug helping patients lose weight and lower blood sugar. The company already has two Phase 3 weight loss studies ongoing and is planning on starting another pivotal trial next year looking at blood sugar control and cardiovascular outcomes.
• Celldex Therapeutics said its experimental treatment for chronic hives achieved the goals of two Phase 3 clinical trials. 
• Ionis Pharma said an experimental therapy for a rare form of ALS prolonged survival and slowed the progression of the disease in a Phase 3 clinical trial. The RNA-based drug, called ulefnersen, is licensed to the Japanese pharma company Otsuka. 

Viking drug shows potential for less frequent maintenance dosing

Viking Therapeutics said this morning that in a Phase 2 trial, its injectable obesity candidate, initially tested as a weekly treatment, still looked to be efficacious after transitioning to every-other-week or monthly dosing.

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Top of the morning to you. Gray skies are hovering over the Pharmalot campus right now, but our spirits remain sunny, nonetheless. Why? We will trot out a bit of insight from the Morning Mayor, who would say “Every new day should be unwrapped like a precious gift.” To celebrate the notion, we are brewing still more cups of stimulation and invite you to join us. Remember, a prescription is not required, nor is a copay. Our choice today is butter pecan. Meanwhile, here are a few items of interest. Hope you have a smashing day, and of course, do stay in touch. …

Western pharmaceutical companies are urging the Trump administration to allow them to keep doing deals with Chinese companies for promising drug candidates, despite a 2025 law restricting U.S. investment in Chinese technology, The Wall Street Journal reports. An industry trade group has been lobbying to make sure the new rules the Treasury Department is writing, as part of that law, maintain their ability to do deals in China. The law, called the Comprehensive Outbound Investment National Security Act or COINS, requires the federal government to screen deals with certain countries involving sensitive technology. So far, the Treasury Department has been reluctant to fully incorporate pharmaceuticals.

The U.S. Food and Drug Administration posted documents ahead of an advisory committee meeting on Wednesday that suggest the agency may take a favorable view of a Grail blood test aimed at detecting many different types of cancer early, when they can be treated more effectively, STAT writes. The test is already available as a laboratory-based diagnostic, but FDA approval would be expected to help lead to reimbursement from Medicare and other insurance payers. Investors are upbeat, but the documents include an anomaly that might not signal an open-and-shut positive outcome for the company. 

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Good morning health tech readers!

My kids have been in school for one week and everyone is already sick. But the newsletter marches on!

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Teen social media bans are hot these days, with new regulations installed or soon to come in Australia, Indonesia, and the European Union. But how will we know these rules are working? And what does “working” even mean?

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CLEVELAND — Amy Acton couldn’t speak. 

Supporters of Acton, the Democratic nominee for governor of Ohio, were waiting for her remarks along the banks of the Black River, once known as the “river of fish tumors” because of the catfish deformed by steel mill slag. Now the river was being rehabilitated. Acton, a physician who vaulted to national fame when she led Ohio’s aggressive response to Covid-19 as the state’s director of public health back in 2020, was running with the promise to do the same for state politics. 

The crowd, attending a Labor Day festival in the industrial city of Lorain, had spent the afternoon grazing on boba tea and pastelitos from food trucks while a live band covered ’90s hits like “Return of the Mack.” They didn’t yet know that Acton wasn’t going to make it onstage after all. Earlier that day, at a different campaign event about 90 miles east in Canfield, a man carrying two handguns, brass knuckles, and a Taser had barreled toward Acton while yelling her name, knocking over an elderly couple in the crowd. (The man, Patrick Havas, has since pleaded not guilty to charges including assault.)

Of all the high-profile electoral contests this November, the heated race for Ohio governor could be the most direct test of where public opinion in the heartland currently stands on health care, trust in science, and the lasting legacy of the Covid pandemic. Acton’s Republican opponent, Vivek Ramaswamy, is the billionaire founder and former CEO of biotech company Roivant who built his political profile as a 2024 presidential candidate in part on accusing the government of overreach in the pandemic — claims he’s made central to his current gubernatorial campaign as well.

STAT visited events for each candidate on the same day in August to get a sense of how Acton and Ramaswamy are tapping into American health anxieties, and how their messages are resonating with Ohioans. Acton stands by her decisions during the pandemic, but is betting that people in Ohio are ready to move on and instead focus on fixing problems like health care affordability and access. (Affordable Care Act enrollment in Ohio dropped by one-third in the past year following the expiration of federal subsidies, the most of any state.) 

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WASHINGTON — Brian Blase, president of Paragon Health Institute, has become a trusted adviser to Republicans in Congress, who passed sweeping changes to federal funding for health care last year.

The Medicaid program is expected to pay out some $1 trillion less than projected over the coming decade, thanks to policies Blase supported. The changes are causing health providers to scramble and rethink how they offer care. 

The cuts to the Medicaid program through the One Big Beautiful Bill Act were used to offset the cost of tax cuts — a change that’s expected to lead to 7 million people losing Medicaid coverage, according to preliminary estimates from the nonpartisan Congressional Budget Office.

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In December 2025, as social media’s potential impact on youth mental health faced growing scrutiny, Australia became the first country to ban social media for people under 16, with Indonesia quickly following. The European Union is taking a slightly different approach, banning social media for children under 13 and personal accounts for people under 15 after pressure from several E.U. nations. 

Elsewhere, big tech companies are being challenged in the courts. Meta recently agreed to pay up to $18 billion to resolve claims in the U.S. that Facebook and Instagram harmed children. Meta will also introduce “safety by design” features for young people, such as concealing the number of “likes” on posts. But will these features meaningfully change young people’s behavior or experiences online? Will the bans have their intended effect?  

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For much of my career in cancer care, I have seen a contradiction play out in the exam rooms of my colleagues and across the country. Cancer patients ask how to manage pain, fatigue, anxiety, immune suppression, and the daily toll of the disease. Clinicians often prescribe only medication, even though they know other evidence-based treatments could help: acupuncture for chemotherapy-induced nausea; exercise and nutrition counseling to improve clinical outcomes; and mindfulness-based programs to ease distress, sleep problems, and even immune dysfunction.

When doctors and patients do discuss these treatments, too often the conversation ends with a shrug: “Insurance doesn’t cover that.”

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Tensions over increased White House control of federal biomedical research funding escalated in recent days, with several members of Congress voicing strong opposition after news leaked about a plan to create a panel of political appointees to sign off on all National Institutes of Health grants.  

In what has been reported to be a contentious meeting at the White House on Friday, Russell Vought, the head of the White House Office of Management and Budget, pushed for an executive order creating a commission that would review all NIH grants, according to the Washington Post. President Trump, reportedly angry that NIH Director Jay Bhattacharya had refused to cut funding for Harvard researchers, directed Vought to draft the order.  

Politico reported Vought and Bhattacharya would serve on the proposed board, which would be required to reach unanimous decisions on grants. 

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Shares in Grail, the company developing a blood test aimed at detecting many different types of cancer early, when they can be treated more effectively, surged 33% Monday after the Food and Drug Administration posted documents ahead of an advisory committee meeting that suggest the agency may take a favorable view of the test.

The meeting, scheduled for Wednesday, will bring together key experts to discuss the test, known as Galleri. The FDA is not obliged to follow the advice of these panels, but it often does.

Investors had already been bidding up Grail’s stock in anticipation of the meeting. The test is already available as a laboratory-based diagnostic, but FDA approval would be expected to help lead to reimbursement from Medicare and other insurance payors. 

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Hi! Today we have some consolidation in radiopharma, an early ADHD signal for orexin agonists, and a call from Reed Jobs to double NIH funding.

I like that Jobs wants biology to be treated as a space race. I also want more racing to space. 

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Several hospitals in Texas owned by the giant for-profit chain HCA Healthcare are suing Independence Blue Cross, a large Blue Cross Blue Shield plan based in Philadelphia, for more than $345,000 in unpaid medical claims that involved care from roughly four years ago.

The dispute highlights the contentiousness that still exists over prior authorization and the varying standards for determining whether care is medically necessary. It also provides a snapshot into the high prices hospitals charge Blues plans.

Prior authorizations, which are requests to insurance companies to preapprove care, exist to reduce wasteful and unnecessary care. But providers, patients, and policymakers have condemned insurers, saying they are using those policies to cut costs and increase profit. Some have called for a ban of the practice. In response, health insurers have made some voluntary pledges to cut down.

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Hello there and welcome to a new week. STAT reporter Andrew Joseph here in London filling in for Mr. Pharmalot for the day. Before we get to the headlines, an extra dose of encouragement to check out all the great pieces that STAT has to offer today, from Reed Jobs opining on the NIH budget to our new trust-in-science reporter Nick Florko outlining his own experience grappling with, as he puts it, “how frustrating it is to realize that modern medicine does not have all the answers.” Now to those headlines. … 

Novo Nordisk is seeking to assure investors that it can regain its momentum in the coming years, outlining plans to expand its pipeline and find new products that it could sell more like consumer goods than traditional medicines, building off what’s occurring with its obesity treatments, STAT shares. At the company’s capital markets day in London, CEO Mike Doustdar said Novo would launch at least five multi-blockbusters by 2030 and deliver revenue growth on par with other major pharma firms. He also presented a strategy that involved investing more in disease areas outside diabetes and obesity and said the company “will be more active within business development,” an acknowledgment of investors’ concerns that Novo was too reliant on its landmark GLP-1 drugs and needed to develop other products.

Drugs called orexin agonists have been hailed for their ability to treat rare sleep disorders, but with new data, Alkermes is showing for the first time that the new class of therapies may benefit people with ADHD, STAT writes. In a randomized Phase 1 study, the company’s new drug, called ALKS 7290, was well tolerated and showed a signal of efficacy. Participants in the study started with a median score of 39 on a diagnostic known as the Adult ADHD Investigator Symptom Rating Scale, indicating that they had moderate to severe symptoms. After two weeks, those taking a high dose of 50 milligrams of ALKS 7290 experienced a 19-point reduction on the scale, indicating that their symptoms had become mild.

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I asked and you delivered … some great reading recommendations, including “Legacy” by Uche Blackstock and “Healing” by Theresa Brown. I’m adding both to my list, along with “The Emperor of All Maladies” (which has four new final chapters!). My own suggestion in return: I recently finished “The Spirit Catches You and You Fall Down,” a deep dive on a Laotian family trying to navigate the American medical system.

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Drugs called orexin agonists have been hailed for their ability to treat rare sleep disorders, but with new data, Alkermes is showing for the first time that the new class of therapies may benefit people with ADHD.

In a randomized Phase 1 study, the company’s new drug, called ALKS 7290, was well tolerated and showed a signal of efficacy, Alkermes said Monday.

Participants in the study started with a median score of 39 on a diagnostic known as the Adult ADHD Investigator Symptom Rating Scale, indicating that they had moderate to severe symptoms. After two weeks, those taking a high dose of 50 milligrams of ALKS 7290 experienced a 19-point reduction on the scale, indicating that their symptoms had become mild.

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LONDON — Novo Nordisk on Monday sought to assure investors that it would regain its momentum in the coming years, outlining plans to expand its pipeline and find new products that it could sell more like consumer goods than traditional medicines, building off what’s occurring with its obesity treatments.

At the company’s capital markets day, taking place here all day Monday, CEO Mike Doustdar said Novo would launch at least five multi-blockbusters by 2030 and deliver revenue growth on par with other major pharma firms. He presented a strategy that involved investing more in disease areas outside diabetes and obesity and said the company “will be more active within business development,” an acknowledgment of investors’ concerns that Novo was too reliant on its landmark GLP-1 drugs and needed to develop other products.

Novo shares dropped 5% during Doustdar’s opening remarks, a sign that investors wanted more specifics. 

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In late August, the Food and Drug Administration approved a new therapeutic for pancreatic cancer, daraxonrasib, developed by Revolution Medicines. This drug is for previously treated patients and represents a revolutionary new therapeutic to target KRAS inside pancreatic cancers, the most commonly mutated oncogene found in the disease. Having this mutation is a very bad sign for a pancreatic cancer patient, often indicating that their case is terminal.

Medicine has known about KRAS for decades, but because of its physical shape, it’s been nearly impossible to treat. Until daraxonrasib proved for the first time that KRAS could be treated, bound, and stopped.

While pancreatic cancer remains a devastating diagnosis, this therapy nearly doubled the survival rate in a clinical trial — an incredible result for those patients and their families. Time is priceless.

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WASHINGTON — Zach Lahn, a farmer and Republican candidate for Iowa governor, says he won’t forget these moments: visiting a bait-and-tackle shop, bream fishing, and campaigning alongside health secretary Robert F. Kennedy Jr.

“RFK Jr.’s story is one that I have long admired,” Lahn wrote on his official campaign page in August, following Kennedy’s visit to Iowa. 

Kennedy, in turn, wrote that Lahn was a “superstar.” 

Lahn could use the help. He’s trailing the Democratic candidate in recent polls, in a state that voted for President Trump by more than 10 percentage points in 2024. 

While many GOP candidates are struggling, Lahn’s race is a particularly sharp test of Kennedy’s pull with voters, especially those who are displeased with the president, whose disapproval rating has risen sharply. 

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Public trust in science, and in health institutions in particular, has been in decline for years. 

On one hand, disinformation and cultural shifts have led people to lose faith in medicine and institutions that are critical for individual and public health. On the other hand, it is not hard to find examples of institutions that have lost the public’s trust for good reason. For nearly 11 years here at STAT, our journalists have been doggedly reporting on misdeeds, scandals, and crimes perpetrated by some of the biggest companies and names in the U.S. health care system. 

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When health secretary Robert F. Kennedy Jr. talks about the chronic disease epidemic ravaging America’s young, I am one of the people he is speaking about. At 34 years old, I am overweight and already suffering from high cholesterol and high blood pressure. I have ADHD so bad I often forget to brush my teeth in the morning. And when a doctor — who I waited several months to see in hopes he’d figure out why I’m incessantly tired — recently asked what conditions my very high dose of an SSRI was treating, my response was simply “all of the above.”

I tell you all of this as STAT’s inaugural reporter on trust in science, because I know what it means to be searching for an explanation to what ails you, and a way to fix it. I also know all too well how frustrating it is to realize that modern medicine does not have all the answers. On bad days, when I’m especially anxious or I step on the scale after a good week of dieting to see no results, Kennedy’s claim that America’s medical system has turned into “a sick care system” that rewards doctors, hospitals, and drug companies resonates more than I’d like to admit. At my most desperate, I’ve tried the keto diet, injected a compounded peptide, and taken fistfuls of dietary supplements. It’s this worldview that will inform my work in this new role. 

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Addiction clinics around the country are seeing a growing number of cases related to gas station products made from the kratom leaf and its derivatives.

At the telehealth addiction clinic Boulder Care, for example, people who report using kratom products constituted 3% of all new patients in September 2025. By July 2026, that number was 8%, and in August, it jumped to 14%. 

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If you are a patient looking to join a clinical trial, a clinician advising one, or a researcher trying to understand the trials that have been conducted for a particular medication, you may have encountered ClinicalTrials.gov. The website serves as a resource for clinicians and patients interested in trial participation as well as for researchers who want to understand the body of evidence for different conditions, assess signs of publication bias, and explore trial composition and representativeness. Since late 2007, federal law has required companies and researchers to register most Phase 2 to Phase 4 drug trials. Over 600,000 studies are listed on the site, which draws more than 3 million visitors each month and can be easily filtered based on patient age, sex, diagnosis, and ZIP code.

However, pregnant patients can’t easily identify studies for which they are eligible. ClinicalTrials.gov lacks structured data on whether pregnant or lactating people were included or can join a trial.

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ITM Isotope Technologies Munich SE and Telix Pharmaceuticals, two leading companies in the burgeoning radiopharmaceuticals field, are merging in a deal worth at least $1.65 billion.

Under the deal, Telix will acquire all shares of the privately held company ITM. Telix could also pay another $700 million to shareholders, if ITM’s lead drug meets regulatory and sales goals.

The merger creates another well-resourced company in the radiopharmaceutical field, which is currently dominated by Novartis. These medicines work by delivering radioactive isotopes directly to cancer cells. Novartis supercharged the field with the launch of its two such treatments in 2018 and 2022. 

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The Jehovah’s Witnesses have partly eased their long-standing prohibition on the medical use of donated blood, allowing members to receive products derived from blood but maintaining a ban on transfusions of whole blood.

The policy, announced Friday, is the second revision this year in what has long been one of the most controversial and distinctive teachings of the religious organization.

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WASHINGTON — President Trump made an announcement Friday to tout one of his initiatives to lower drug prices. It left many questions unanswered.

All 50 states, Washington, D.C., and Puerto Rico will participate in the GENEROUS model, which is intended to give lower drug prices to Medicaid programs, Trump said.

As part of broad drug pricing deals with the Trump administration, more than two dozen pharma companies agreed to sell drugs to state Medicaid programs at prices available in peer countries under GENEROUS. The deals are a major component of President Trump’s plan to lower drug prices, and have become key to his messaging on affordability. 

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And so, another working week will soon come to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans. Our agenda is still taking shape, but we plan on promenading with the official mascots, catching up on our reading, escorting Mrs. Pharmalot to a speakeasy and, if time permits, holding another listening party, where the rotation will likely feature this, this, this, this and this. And what about you? Now that autumn is upon us, this is a fine time to jump in your jalopy to visit orchards and patches. If the weather fails to cooperate, you could binge-watch a few series on the telly or shop for warmer garb. Maybe a new sweater or two is needed. Or stop by the eatery that always appeared inviting. Well, whatever you do, have a grand time. But be safe. Enjoy, and see you on Tuesday, since we will be off again to observe ancient rituals and a pinch hitter will be in our stead on Monday. …

The U.S. is working on rules for pharmaceutical companies investing in China that would likely preserve their ability to strike most licensing deals for Chinese drugs, Reuters reports. This would mark a departure for the Trump administration, which has sought to tighten business ties with China for other industries under new national security legislation, and would be looser than restrictions sought ‌by some lawmakers. The rules, being drafted by the U.S. Treasury Department, are likely to allow U.S. drugmakers to invest in promising new drugs being developed by Chinese companies as long as they are not related to pathogens or biotechnology that could be weaponized.

An analysis of U.S. Food and Drug Administration data found hundreds of post-market studies listed as delayed and, in some cases, the work was delayed by more than a decade or the manufacturer was still developing a plan for the study, KFF Health News confides. Relying on post-approval studies to resolve questions risks exposing patients to products that do more harm than good. Whoever is paying the bills — patients, insurance companies, employers, or government health programs such as Medicare and Medicaid — can end up wasting money and rewarding manufacturers for useless or risky products.

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Today, we discuss the first gene therapy approval for Sanfilippo syndrome, learn of a rare gene variant linked to dramatically heightened lung cancer risk in non-smokers, and look ahead toward the confirmation hearing for the nominee to lead the FDA.

About the Sanfilipo approval: I first wrote about the effort to develop a therapy a decade ago. I get melancholy sometimes when I think of how many kids have declined or died before their families can get cutting-edge therapies approved for children who are not their own. But science takes time, and biotech can produce a better tomorrow, no?

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As I was writing this newsletter, I was listening to Rebecca Black’s recent album — who you might recognize from her 15-year-old song, Friday. Happy Friday, everyone!

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I was 8 years old, sitting at dinner with my grandparents, watching Gong Gong’s face turn bright red after a few sips of yellow wine. He loved making me laugh, and though he enjoyed his drinks, I think he liked my reaction even more.

Over a decade later, I would recognize the same alcohol flush reaction at parties and happy hours in some of my East Asian friends, and in myself. A few of these friends took histamine blockers like Pepcid AC before going out, hoping to hide the anticipated redness. Once, I watched someone keep drinking through the flush to prove they had a higher tolerance than what their body was communicating.

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There are more men sitting in my plastic surgery waiting room now than ever before. If I had to choose one word to describe the men I see today, it would be “influenced.”

Procedures on men in the U.S. have grown by 4%, a rate that makes men the fastest growing population in aesthetics. In 2024, men underwent 1.6 million minimally invasive cosmetic procedures and almost 100,000 surgical procedures in the United States.

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HEIBAN DISTRICT, Sudan — For more than a decade, medical missionary Tom Catena has run the largest hospital in the Nuba Mountains, a rugged territory the size of Austria, most recently in the midst of a brutal civil war.

There is no oncologist on staff at Mother of Mercy, but as the war destroys much of the country’s health care system, patients like Fatouma Mahdi Ahmed Saeed, who has cervical cancer, keep turning up at his door.

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Across medicine, doctors are increasingly using artificial intelligence models to predict patient risk, from sepsis to falls to death. Often embedded directly into electronic health records, these predictions can be easy to incorporate into care — and their performance can be easily taken for granted. 

James Deardorff, a geriatrician and assistant professor in the division of geriatrics at the University of California San Francisco, has developed several models that aim to make predictions for older adults, from mortality to the need for nursing home care. In geriatrics, he says, it’s important for clinicians to be aware of both the performance of an algorithm — including in subgroups like older patients — and how to responsibly use its output.

This month, Deardorff penned a commentary on a large analysis of Epic’s proprietary end-of-life prediction model published in JAMA Network Open, highlighting how a model can contribute to a poor outcome even if it’s accurate. If a patient’s one-year mortality prediction is used to prompt an open-ended conversation about the goals of care, he and his co-author wrote, there are few downsides. But if it’s used to inform a higher-stakes decision like transplant priority, the impacts could be profound. 

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Health secretary Robert F. Kennedy Jr. announced Thursday a new effort to improve autism diagnoses, arguing that the current model is outdated, too time-intensive, and overreliant on professionals with limited availability. 

“We are not satisfied simply for describing autism and managing its consequences. We are asking what drives the different forms of autism, and why developmental trajectories differ, why some children regress, and which interventions work for them,” Kennedy said during his keynote address at a conference for Children’s Health Defense, an anti-vaccine organization he previously led. 

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For years, researchers have known that, among people diagnosed with lung cancer, the prevalence of nonsmokers has been increasing. But they have been trying to determine what factors — whether genetic, environmental, or something else — might put these “never-smokers” at heightened risk. 

Now, researchers have identified a very rare genetic variant that is associated with 25-fold higher odds of lung cancer, they reported in the journal Science on Thursday. In the U.S., the variant is far more common among people in Southern Appalachia than in other regions. 

Experts said that the variant likely only plays a role in a small portion of never-smoker lung cancer cases. But the research provides further evidence that these types of tumors can be tied to distinct risk factors, which may in turn require thinking differently about screening programs and treatment strategies. More broadly, the new study highlights the types of narrow but crucial discoveries that can be made by turning to large datasets replete with health information from millions of people. In this case, the investigators relied on genetic data from 23andMe. 

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The Food and Drug Administration on Thursday approved a new gene therapy for Sanfilippo syndrome type A, an ultra-rare disease sometimes known as childhood Alzheimer’s.

The therapy, called Fayuvi, was developed by Ultragenyx. It is the first drug specifically approved to treat Sanfilippo. Ultragenyx did not immediately release a price.

“It’s hard to overstate what this approval would mean for everybody who is living with this really horrific disease and watching their children suffer and pass away early, and all the rest that it brings,” Cara O’Neill, chief science officer of the Cure Sanfilippo Foundation, said in an interview earlier this week. “This, gosh, would just finally mean when patients and families receive this shocking diagnosis, they wouldn’t be told to … take their kids home and love them. They would be given hope and an action plan for treatment.”

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