A panel of experts agreed that exa-cel, a sickle cell disease gene therapy, was safe enough for clinical use, setting the stage for a potential FDA approval; the United States saw an increase in infant mortality rates for the first time in more than 2 decades; the new antiburnout campaign from the CDC asks leaders to better support health care workers.
A panel of experts announced Tuesday that exagamglogene autotemcel (exa-cel), a sickle cell disease gene therapy from Vertex Pharmaceuticals and CRISPR Therapeutics, was safe enough for clinical use, according to the New York Times. This recommendation sends it to the FDA for a decision on whether to approve it for broad patient use, with a decision likely by December 8. Although Vertex has not yet said what it will charge for exa-cel, it, and treatments like it, are expected to cost millions of dollars per patient. If approved, exa-cel would be the first medicine to treat a genetic disease using the CRISPR gene-editing technique. The therapy’s approval could also be the first of a series of new options as the FDA will decide on a second potential gene therapy cure for the disease by December 20.
In 2022, the United States saw an increase in infant mortality rates for the first time in more than 2 decades, according to CNN. The National Center for Health Statistics (NCHS) report released Wednesday said that the overall infant mortality rate, as well as the neonatal infant mortality rate, rose by 3% from 2021 to 2022; the postneonatal mortality rate rose by 4%. Also, NCHS reported that infant deaths due to maternal complications and bacterial sepsis shot up by 8% and 14%, respectively. Danielle Ely, an NCHS health statistician and one of the report’s authors, explained to CNN that infant mortality is often a useful marker for public health experts to understand a country’s overall health system. Consequently, the increase in the US infant mortality rate could be an outlier or a sign of an underlying health care issue; next year’s data will help NCHS know for certain.
The new antiburnout campaign from the CDC and the National Institute for Occupational Safety and Health asks leaders to better support health care workers, according to STAT. The campaign offers tools to help leaders improve their respective work environments, including a worker well-being questionnaire, online workshops on related topics, and a guide asking leaders to share their mental health struggles to encourage staff to do the same. Fixing the problem of health care worker burnout requires larger systemic change, however. Experts identified some pain points that need to be addressed to reduce health care workers’ moral distress, including prior authorization, complicated electronic health record systems, and the structure of reimbursement and insurance plans.
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