Weekend Update

David Ermer–Congress, FDA, Generative AI, Medical / Rx research, OPM, U.S. Healthcare–

From Washington, DC

  • Tomorrow, the Senate will take a final vote on H.R. 6938, Commerce, Justice, Science; Energy and Water Development; and Interior and Environment Appropriations Act, 2026, which the House of Representatives already has passed. When as expected, the Senate passes this bill, Congress will have passed half of the twelve appropriations bills in regular order.
  • Roll Call offers more insights into the state of the appropriations process.
    • “The House, meanwhile, looks to move forward with a bundle of two more spending bills, including funding for the State Department, the Treasury and an assortment of related agencies including the White House itself.”
    • While the Senate has scheduled a recess for next week, “[t]he House is expected to be in session next week except for Jan. 19, which is Martin Luther King Jr. Day.”
  • Per a Senate news release,
    • “On Thursday, January 15, [at 10 am ET] the Senate Health, Education, Labor, and Pensions (HELP) Committee will vote on several bipartisan bills to improve American families’ health.
    • “The bills under consideration include:
      • “S. 1157, Women and Lung Cancer Research and Preventive Services Act
      • “S. 921, Tyler’s Law
      • “S. 2169, Rural Hospital Cybersecurity Enhancement Act
      • “S. 272, Protect Infant Formula from Contamination Act.” * * *
    • “Click here to watch live.”
  • Per a House news release,
    • “Ways and Means Committee Chairman Jason Smith (MO-08) and Energy and Commerce Committee Chairman Brett Guthrie (KY-02) announced the details for an upcoming hearing with five of the biggest health insurance company CEOs to answer questions on making health care more affordable for all Americans.” * * *
    • “The date of the hearing will be January 22, 2025, with the panel appearing before the House Committee on Energy and Commerce Subcommittee on Health in the morning, and the House Committee on Ways and Means in the afternoon. 
    • “Company CEOs in attendance will be UnitedHealth Group, CVS Health, Elevance Health, The Cigna Group, and Ascendiun (the parent company of Blue Shield of California).”
  • Govexec tells us,
    • “The Trump administration is bringing back an awards program for senior executives after canceling it for fiscal 2025. 
    • “In a Jan. 5 memo, Office of Personnel Management Director Scott Kupor asked agencies to nominate up to nine percent of their highest-ranking career employees for the fiscal 2026 presidential rank awards. The program was established in 1978 to honor civil servants who have exhibited consistent achievement.”
  • BioPharma Dive reports,
    • Johnson & Johnson on Thursday [January 8] became the latest pharmaceutical company to agree to “most favored nation” pricing in return for a reprieve on pharmaceutical tariffs. The company didn’t provide many specifics in its announcement. But it’ll participate in a government online channel for drug purchasing; enable U.S. patients to access drugs at comparable prices to what’s paid in foreign countries; and give the same prices to Medicaid, which already has best-price guarantees. J&J will also build a cell therapy plant in Pennsylvania and a facility in North Carolina as part of its $55 billion commitment to boost U.S. manufacturing capacity.

Frrom the Food and Drug Administration front,

  • Per an FDA news release,
    • “The U.S. Food and Drug Administration today announced it is sharing information about the agency’s flexible approach to overseeing chemistry, manufacturing and control (CMC) requirements for cell and gene therapies (CGT). The agency’s more flexible approach has been, and is expected to continue to be, helpful in expediting product development and will help guide the FDA’s evaluation of development strategies in preparation for a Biologics License Application (BLA) submission.  
    • “Regulatory flexibility must be tailored for cell and gene therapies,” said FDA Commissioner Marty Makary, M.D., M.P.H. “These are common-sense reforms that will address the unique characteristics of cell and gene therapies and foster more innovation.”
  • The Washington Post explains the problem that the FDA seeks to resolve.
    • “Genetic therapies could be used to treat hundreds of diseases. The path to patients is tricky.”
  • STAT News notes,
    • “Stoke Therapeutics and the Food and Drug Administration were unable to reach agreement on an expedited submission for the company’s severe epilepsy treatment, the company said Sunday. 
    • “Following a meeting in December, the FDA did not shut the door on Stoke’s request to submit zorevunersen, a treatment for Dravet syndrome, later this year, rather than wait for the completion of an ongoing Phase 3 study in the middle of 2027, Stoke CEO Ian Smith told STAT in an interview.
    • “Instead, regulators asked the company to submit more information, and further discussions are planned. Stoke expects to make a decision on a regulatory path for zorevunersen by the middle of the year.” 

From the public health and medical / Rx research front,

  • Medscape relates,
    • “Different subgroups of patients with adult-onset diabetes showed different rates of comorbidities and mortality outcomes. Patients with severe insulin-resistant diabetes (SIRD), a high-risk subtype, were most likely to have kidney, liver, and heart diseases despite having only mild hyperglycemia, and some of these conditions were present even before diabetes was diagnosed.”
  • Healio points out,
    • “Between 50% to 75% of children struggle with adhering to health care regimens.
    • “Rebecca Liu, a senior at UCLA, described her idea for a game that could educate children about their health and improve adherence.”
  • The Wall Street Journal considers,
    • “What Time Should You Wake Up? Probably Not 5 a.m.
    • “Sleep experts warn against rising too early if you’re not naturally a morning person, and provide tips for a better night’s sleep.” * * *
    • “Your ideal rise time is linked to your chronotype, your genetic predisposition to waking at a certain time. (If you need help understanding yours, take Breus’s quiz, which requests your name and email address.)”
  • MedPage Today reports,
    • “An immuno-oncology (IO) regimen for intermediate-stage hepatocellular carcinoma (HCC) significantly extended treatment benefit versus transarterial chemoembolization (TACE), according to an interim analysis of a randomized trial.
    • “Time to failure of treatment strategy (TTFS) increased from 9.5 months with TACE to 14.6 months with the combination of atezolizumab (Tecentriq) and bevacizumab (Avastin). Treatment-related adverse events (TRAEs), including grade ≥3 TRAEs, occurred more often with the systemic therapy, but no unexpected or excess fatal events occurred.
    • “The results provided justification to continue enrollment and follow-up to a second planned interim analysis later this year, reported Peter R. Galle, MD, of University Medical Center Mainz in Germany, at the American Society of Clinical Oncology Gastrointestinal Cancers Symposium.”

From the U.S. healthcare business and artificial intelligence front,

  • Modern Healthcare reports,
    • “The 44th annual J.P. Morgan Healthcare Conference kicks off Monday, drawing more than 8,000 healthcare executives and investors to San Francisco. 
    • “At a time of widespread uncertainty for the industry’s profit potential, companies will unpack their latest financial results while touting capital projects, technology advancements and merger and acquisition plans to private equity firms, venture capitalists and other potential investors. More than 500 public and private companies are expected to discuss a range of topics including artificial intelligence, biotechnology, personalized medicine, digital health, healthcare funding, regulation and glucagon-like peptide-1 drugs that treat diabetes and obesity.”
  • The Wall Street Journal adds,
    • “When Health Secretary Robert F. Kennedy Jr. released his MAHA Report in May, a who’s who of the wellness world convened at the White House for the occasion. There was the influential physician Mark Hyman, who co-founded the direct-to-consumer testing company Function Health, recently valued at $2.5 billion. Also in attendance: Alex Clark, the host of the popular Turning Point USA podcast “Culture Apothecary.” Longevity influencer Gary Brecka, who’d recently had Kennedy over to get intravenous drips and use Brecka’s hyperbaric chamber, was present, along with the “medfluencers” Dr. Will Cole and Dr. Paul Saladino.
    • “All of them support Kennedy’s ascent to the nation’s top health job. And all of them stand to gain from the spotlight he’s placed on alternative health. That’s because each of them has ties to the business of supplements, a $70 billion, lightly regulated U.S. market that could benefit from his support. On Brecka’s podcast, recorded after their wellness treatments, Kennedy vowed to end “the war on vitamins.”
  • Fierce Pharma lets us know,
    • As Novartis turns the calendar on a new year, the Swiss drugmaker is elaborating further on plans for the $23 billion U.S. investment it unveiled last April.
    • Next on the docket will be a new, 35,000-square-foot radioligand therapy (RLT) facility in Winter Park, Florida, Novartis reported Friday. 
    • Joining established plants in Novartis’ American RLT network in Indiana, New Jersey and California, the new facility will boost the company’s radiopharmaceutical manufacturing to “optimize the delivery” of the cutting-edge cancer treatments to patients across the southeastern United States, Novartis said in a Jan. 9 press release. The upcoming site is expected to come online by 2029, the company added. 
  • Buiness Insider relates,
    • “Anthropic is rolling out a major expansion of its healthcare and life-sciences offerings, as AI companies race to embed large language models more deeply into regulated medical workflows.
    • “The company on Sunday announced Claude for Healthcare, a product that allows healthcare providers, insurers, and consumers to use Claude for medical purposes through HIPAA-ready infrastructure. 
    • “The launch builds on Anthropic’s earlier release of Claude for Life Sciences, which focused on research and drug discovery, and reflects the company’s broader effort to position its AI models as practical tools for regulated industries.”

Notable Obituary

  • The New York Times reports,
    • “Dr. Joel Habener, an American endocrinologist who discovered GLP-1, the protein fragment that became the basis of Ozempic, Wegovy and other blockbuster weight-loss and diabetes drugs that are transforming 21st-century medicine, died on Dec. 28 in Newton, Mass. He was 88.
    • “His death, in a retirement community, was confirmed by his brother, Stephen, who said the cause was a heart attack.” * * *
    • “Dr. Habener’s discovery of GLP-1, in 1987, came about almost by accident. “It was a eureka moment,” he said in a 2023 interview, “which rarely happens in science.”
    • “He was a researcher at the time at Massachusetts General Hospital, where he arrived in 1971 and remained until his retirement in 2023.”

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