FDA Panel Says Innovative Sickle Cell Gene Therapy Is Safe Enough For Use

The New York Times: Panel Tells F.D.A. That CRISPR Sickle Cell Cure Is Safe Enough For Patients
A panel of experts said on Tuesday that a groundbreaking treatment for sickle cell disease was safe enough for clinical use, setting the stage for likely federal approval by Dec. 8 of a powerful potential cure for an illness that afflicts more than 100,000 Americans. The Food and Drug Administration had previously found that the treatment, known as exa-cel and jointly developed by Vertex Pharmaceuticals of Boston and CRISPR Therapeutics of Switzerland, was effective. The panel’s conclusion on Tuesday about exa-cel’s safety sends it to the F.D.A. for a decision on greenlighting it for broad patient use. (Kolata, 10/31)

The Boston Globe: Patients With Sickle Cell Disease Urge FDA To Greenlight Landmark Gene-Editing Treatment
About a dozen people who have sickle cell disease or relatives with the debilitating blood disorder implored the Food and Drug Administration on Tuesday to approve a revolutionary gene editing treatment that those who received it in a clinical trial said transformed their lives. ...The FDA sought feedback about the scientific methods that Vertex and CRISPR used to evaluate the risk of inadvertently changing patients’ DNA beyond the targeted disease — so-called off-target editing. (Saltzman and Weisman, 10/30)

On the opioid crisis —

Reuters: Optum, Express Scripts Want Opioid Special Master Out After Reply-All Email Mishap
Pharmacy benefit managers OptumRx and Express Scripts have asked a federal appeals court to disqualify a long-serving special master in national opioid litigation from working on any cases against them, after he accidentally hit "reply all" on an email that they say revealed him to biased. In a petition filed Monday with the 6th U.S. Circuit Court of Appeals, the companies said the August email, in which Special Master David Cohen wrote that pharmacy benefit managers (PBMs) "knew a lot" about illicit opioid prescriptions, created an appearance of impartiality that disqualified him. (Pierson, 10/31)

KFF Health News: Gubernatorial Candidates Quarrel Over Glory For Winning Opioid Settlements
Opioid settlement cash is not inherently political. It’s not the result of a law passed by Congress nor an edit to the state budget. It’s not taxpayer money. Rather, it’s coming from health care companies that were sued for fueling the opioid crisis with prescription painkillers. But like most dollars meant to address public health crises, settlement cash has nonetheless turned into a political issue. (Pattani, 11/1)

In other developments —

NBC News: By Repeatedly Denying Claims, Medicare Advantage Plans Threaten Rural Hospitals And Patients, Say CEOs
Because the plans routinely deny coverage for necessary care, they are threatening the existence of struggling rural hospitals nationwide, CEOs of facilities in six states told NBC News. While the number of older Americans who rely on Medicare Advantage in rural areas continues to rise, these denials force the hospitals to eat the increasing costs of care, causing some to close operations and leave residents without access to treatment. (Morgenson, 10/31)

Politico: ‘Fundamentally Flawed’: VA Providers Bemoan New Electronic Health Records System
It’s been six months since the VA paused its rollout of a new electronic health records system but the physicians using it are still filing complaints and raising concerns about patient safety, according to internal messages obtained by POLITICO. Messages this month from clinicians at Mann-Grandstaff, a Veterans Affairs facility in Spokane, Wash., show deep frustration with the software from Oracle Cerner, the electronic health records vendor, stemming from outages and errors that prevent staff from filling prescriptions and referring patients to other providers. The messages also show providers struggling to access key patient data needed for patient safety. (Leonard, 10/31)

Politico: Reed: Tuberville Military Holds May Have Contributed To Marine Commandant’s Hospitalization
A top Senate Democrat said that the Marine Corps commandant’s recent medical emergency may be due in part to the fallout from Sen. Tommy Tuberville’s hold on top military promotions, which has forced several top officers to hold down multiple jobs. Senate Armed Services Chair Jack Reed (D-R.I.) leveled the accusation a day after the service disclosed that Gen. Eric Smith, a member of the Joint Chiefs of Staff, was hospitalized on Sunday. There was no immediate word of when Smith would be released or return to work. (Gould and O'Brien, 10/31)

The Hill: Sen. Tillis Tests Positive For COVID
Sen. Thom Tillis (R-N.C.) said Tuesday he tested positive for COVID-19 and would miss votes this week in the Senate. His office confirmed to The Hill he plans to return to Washington next week. (Fortinsky, 10/31)

Also —

Reuters: U.S. FDA Approves Amgen's Biosimilar Version Of J&J's Psoriasis Drug
The U.S. Food and Drug Administration on Tuesday approved Amgen's (AMGN.O) biosimilar version of Johnson & Johnson's (JNJ.N) blockbuster psoriasis treatment, Stelara, for multiple inflammatory diseases. Despite the FDA approval, Amgen's treatment is expected to be launched in 2025 as part of a legal settlement between the two companies earlier this year to delay the entry of the therapy. (10/31)