Latest From Ramsey Baghdadi
Artificial intelligence model’s comments signal the likely start of computer-generated comments to the US FDA’s public notices.
ICER Hemophilia Gene Therapy Reviews Show Clear ‘Net Health Benefit’ But Caution On Patient, Payer Fronts Given Upfront Costs
Restrictive interpretations of FDA labels for BioMarin and CSL Behring products by payers could signal slow launch of gene therapies seeking to supplant existing treatments.
Bryan, who oversees reviews of gene and cell therapies in US FDA’s biologics center, cautioned against falling in love with a biomarker when it is showing no ‘clinical effect.’
A quickened shift toward gene therapies for small populations may be the result of the new Medicare drug pricing law given protections afforded to those types of products.
Five-year plan includes multi-phased approach to ALS at the same time that Amylyx’s AMX0035 is pending review with a late September decision deadline; action plan’s mid-term priorities for FY 2023-2024 include a cell and gene therapies safety project.
Committee Chair Maria Cantwell (D-Wash.) solicits advice from witnesses for additional authorities to conduct oversight of PBMs and pushes for a GAO study.