Monday report

David Ermer–Congress, FDA, Government Contracting, Medical / Rx research, U.S. Healthcare–

From Washington, DC

  • Fierce Healthcare reports,
    • “The Department of Health and Human Services said Monday it has launched “a major initiative” to overhaul the nation’s organ transplant system.
    • “The announcement cites an investigation conducted earlier this year by the department’s Health Resources and Services Administration (HRSA) “that revealed disturbing practices by a major organ procurement organization.”
    • “The news also came a day after a New York Times report that vividly described the case of a patient taken off life support and declared dead, but whose heart and respiratory system were found to still be active after surgeons had made an incision into her chest to secure her organs for donation.”

From the Food and Drug Administration front,

  • BioPharma Dive poses and answers five questions about Sarepta, the FDA and a Duchenne gene therapy crisis.
  • STAT News adds,
    • “Sarepta Therapeutics said Monday evening it will pause all shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, acceding to a request made by the Food and Drug Administration after several patient deaths.
    • “It’s a surprising reversal by Sarepta. The company had initially rejected the agency’s request, which was issued Friday. 
    • “Shipments will halt by close of business Tuesday evening, the company said.
    • “The decision wasn’t entirely in Sarepta’s control. On Monday, Children’s Hospital Los Angeles (CHLA) said it was pausing use of Elevidys while the drug is in regulatory limbo. “Patient safety is paramount at CHLA and the hospital has communicated its decision with affected patient families while it awaits any further determination by the FDA,” the hospital told STAT.” * * *
    • “Sarepta did not indicate how long the Elevidys suspension might last. Ritu Baral, a biotech analyst at Cowen, estimated 3-6 months, during which time the company will test a proposed immune-suppressing protocol designed to mitigate the serious liver toxicity caused by the gene therapy, and share those data with the FDA.” 
       
  • Fierce Pharma tells us,
    • “FDA Commissioner Marty Makary, M.D., has made his pick for the next director of the FDA’s Center for Drug Evaluation and Research (CDER) in veteran biotech executive George Tidmarsh, M.D., Ph.D.
    • “Tidmarsh takes over CDER after a lengthy career in biotech and academia that saw the development of seven FDA-approved drugs, the formation of several biopharma companies and the establishment of Stanford University’s Master of Translational Research and Applied Medicine (M-TRAM) program.
    • “Some of the biopharma companies that Tidmarsh had a hand in founding and leading include Metronome Therapeutics, Revelation Biosciences and Horizon Therapeutics, which has since been picked up by Amgen for $27.8 billion. He’s been stationed at his alma mater at Stanford University’s School of Medicine for the last 15 years, most recently serving as an adjunct professor of pediatrics and neonatology.”

From the public health and medical research front,

  • The American Medical Association lets us know what doctors wish their patients knew about bronchitis.
  • The New York Times reports,
    • “[U]sing genetic data from five million people, an international group of hundreds of researchers reports that it has developed an obesity risk score, known also as a polygenic risk score. It combines thousands of gene variants to estimate individuals’ predicted body mass indexes, which continue to be used by doctors to anticipate weight-related health dangers.
    • “The researchers showed that the scores can predict which young children are at risk of obesity as adults. And, in another test, they found that overweight and obese adults with high-risk scores quickly regain any weight that they lose with lifestyle programs.
    • “Their paper was published on Monday in the journal Nature Medicine.”
  • Per Health Day,
    • “Doctors should ask diabetic women at every visit about their intention to have a child, to make sure they get the appropriate care prior to conception, new guidelines say.
    • “This will help avoid miscarriages and birth defects among women who have diabetes before pregnancy, the authors write in the Journal of Clinical Endocrinology & Metabolism.
    • “We developed these guidelines as diabetes rates are rising among women of reproductive age and very few women with diabetes receive proper preconception care,” lead author Dr. Jennifer Wyckoff, an endocrinologist at the University of Michigan in Ann Arbor, said in a news release.”
  • and
    • “Hispanic people are more likely to develop peripheral neuropathy than white people, and it’s not clear why, a new study has found.
    • “Hispanic folks were 32% more likely than white people to have this nerve disorder even after accounting for known health, lifestyle and social risk factors, researchers reported July 16 in the journal Neurology.
    • “It is likely that unknown social risk factors exist for this population,” lead researcher Evan Lee Reynolds, an assistant professor at Michigan State University in East Lansing, said in a news release. “Identifying additional risk factors for Hispanic people should be the focus of future studies.”
  • The Washington Post reports,
    • “Children with twice the level of the antimicrobial chemical triclosan in their urine were 23 percent more likely to report eczema symptoms than those with lower concentrations, according to research published in the journal Environmental Health Perspectives.
    • “Triclosan was commonly found in antibacterial soaps and body washes before 2016, when the Food and Drug Administration restricted its use in consumer wash products after finding that it was no more effective than standard soap and water. However, according to the Cleveland Clinic, triclosan can still be found in some toothpastes, deodorants, cosmetics and detergents.”
  • Per BioPharma Dive,
    • “A potential multibillion-dollar drug is advancing to late-stage testing now that its developer, Alkermes, has in hand positive results from a smaller study focused on a certain kind of narcolepsy.
    • “According to Alkermes, the study found all three doses of its drug under evaluation were significantly better than a placebo at improving scores on a test that places participants in a quiet, dark, peaceful room and monitors how awake and alert they are. Alkermes described the results as clinically meaningful and said all the drug-treated groups achieved “normative wakefulness” — in this case, taking more than 20 minutes to fall asleep.
    • “All doses of the drug, previously dubbed ALKS 2680 and now named alixorexton, were also generally well tolerated. Alkermes said there were no so-called serious treatment-emergent adverse events. Nor were there any treatment-related safety signals seen in participants’ vital signs, or on liver, kidney and eye exams.”
  • Per Fierce Pharma,
    • “AstraZeneca is raising its defenses against Johnson & Johnson in a fierce competition in EGFR-mutated non-small cell lung cancer.
    • “Following an FDA approval last year based on a tumor progression benefit, AZ said Monday that its Tagrisso combination with chemotherapy extended patients’ lives compared with Tagrisso alone in first-line advanced EGFR-mutated NSCLC.
    • “The readout came from the final overall survival analysis of the phase 3 Flaura2 trial, which had previously shown that the Tagrisso-chemo combo reduced the risk of disease progression or death by 38% versus Tagrisso monotherapy. The latest update found that the combo’s overall survival improvement was statistically significant and clinically meaningful, AZ said.”

From the healthcare business front,

  • Fierce Healthcare reports,
    • “UnitedHealthcare is rolling out a new benefit to encourage preventive cancer screenings.
    • “The insurer’s new breast and colon cancer detection benefit will be available for eligible commercial plans starting Jan. 1, 2026. Through the program, UHC will cover members’ first diagnostic imaging tests for breast cancer and diagnostic tests for colon cancer.
    • “This closes a care gap, UHC said, as follow-up testing may not be classified as a preventive service, which can lead to costs the member does not expect. Rhonda Randall, D.O., chief medical officer of UHC’s commercial business, told Fierce Healthcare in interview that concerns about costs can lead patients to skip key preventive services.
    • “We wanted to do remove that real or perceived barrier for beneficiaries,” Randall said.
    • “For example, about half of routine colonoscopies identify polyps that lead to follow-up testing, and 11% of mammogram screenings lead to additional imaging tests, both of which can draw higher costs if the resulting diagnostics are not covered as preventive.
    • “Under the new benefit, both the initial preventive screenings and follow-up testing will be covered at in-network providers, the insurer said.”
  • Axio tells us, “Hospitals are steadily buying small physician practices and, in the process, driving up the price of care, a new National Bureau of Economic Research study shows.”
  • MedCity News relates
    • CMS proposed a 2.4% Medicare payment increase for hospitals’ outpatient departments and ambulatory surgery centers in 2026, along with a new two-tiered physician reimbursement structure based on participation in value-based care models. Provider groups are welcoming the short-term boosts, but they fear the changes don’t go far enough to address their long-term financial pressures.
  • Beckers Hospital Review offers nine statistics from Press Ganey’s 2025 patient experience report.
    • “Press Ganey found higher scores in patient experience across all care settings in 2025, and a few metrics distinguish top-quartile facilities from the rest, according to a July 21 report. 
    • “Experience isn’t just a reflection of care — it’s the new frontier of care delivery,” Patrick Ryan, chairman and CEO of Press Ganey, said in a July 21 news release. “The systems that will lead in the next decade are those that approach trust, communication, and consistency not as soft skills, but as hard strategy. Our data makes it clear: when organizations embed experience into the core of how they deliver safe, high-quality care, they earn lasting loyalty and drive better outcomes cross the board.”
  • Per Fierce Pharma,
    • “Since starting operations in North Carolina 30 years ago, Biogen has spent approximately $10 billion to bolster its manufacturing presence in the Tar Heel state.
    • “But that’s not enough for the Massachusetts-based biotech, which says it will spend $2 billion more over the next three years, adding factories and upgrades at its two sprawling campuses in the Research Triangle Park (RTP).
    • “The investment pledge comes as biopharma companies respond to tariff threats from President Donald Trump by touting manufacturing expansions in the U.S.
    • “Biogen said the investment will fuel the advancement of its “late-stage clinical pipeline,” and will include expansion of its antisense oligonucleotide (ASO) capabilities and infrastructure. ASOs are snippets of RNA and DNA used in the development and production of gene therapies and cancer treatments.”