FDA Unveils Framework To Fast-Track Rare Disease Gene Therapy Approvals
The proposed system would create a standardized process for authorizing cutting-edge treatments where there is a plausible reason to think they might work, the AP reported.
AP:
Rare Diseases: FDA Proposes New System To Therapy Approvals
Federal health officials on Monday laid out a proposal to spur development of customized treatments for patients with hard-to-treat diseases, including for rare genetic conditions that the pharmaceutical industry has long considered unprofitable. The preliminary Food and Drug Administration guidelines, if implemented, would create a new pathway for bespoke therapies that have only been tested in a handful of patients due to the challenges of conducting larger studies. The FDA announcement specifically mentions gene editing, although agency officials said the new approach could also be used by other drugs and therapies. (Perrone, 2/23)
More news from the Trump administration —
The Marshall Project:
Federal Prisons Prohibit Gender-Affirming Care For Transgender People
The federal prison system will stop providing gender-affirming medical or social transition care to almost any transgender people, under a new policy released by the Bureau of Prisons Thursday. (Schwartzapfel, 2/23)
Becker's Hospital Review:
AHA Urges HHS To Align AI Rules With Existing Healthcare Regulations
The American Hospital Association is calling on federal health officials to reduce regulatory barriers and ensure clinician oversight as artificial intelligence becomes more integrated into clinical care. In a Feb. 23 letter to the Department of Health and Human Services, the AHA outlined recommendations in response to the agency’s request for information on accelerating AI adoption in healthcare. (Diaz, 2/23)
The New York Times:
Trump, Bruised And Unpopular, Turns To State Of The Union For A Reset
This year, a substantial number of Democrats are planning to boycott the speech and attend an alternative event, a rally called the “People’s State of the Union,” which will take place on the National Mall near the Capitol. Representative Hakeem Jeffries, Democrat of New York and the minority leader, has encouraged members to either sit silently through the speech or boycott it altogether, rather than attend and create distractions in the House chamber. Such protests potentially risk alienating swing voters ahead of the midterms. (Broadwater, 2/24)
In news about the Epstein files —
AP:
Medical Influencer Attia Quits CBS News After Name In Epstein Files
Dr. Peter Attia, a medical influencer whose emails with the late sex offender Jeffrey Epstein were revealed in the latest U.S. Justice Department release of files, has resigned a post with CBS News. Attia, podcast host and author of “Outlive: The Science & Art of Longevity,” was one of a group of people named last month by CBS News editor-in-chief Bari Weiss as a contributor to network programming. He was the subject of a “60 Minutes” profile that ran on the network last October. But shortly after the appointment, Attia’s name surfaced in hundreds of Epstein documents. (2/23)
Stat:
Epstein Blood Samples Ignited A 2013 Furor In Harvard Genome Lab
Among several fridges inside the Harvard Medical School lab of renowned geneticist George Church is one devoted to housing tubes of human blood and spit destined to have their cellular contents cracked open, the DNA letters read out and posted to the wilds of the open internet. Eventually. (Molteni, 2/24)
