Latest From Ramsey Baghdadi
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.
The upcoming review of two novel gene therapies from bluebird bio marks will be the first time the US FDA’s CTGTAC has met for a genetic product review in five years. That is a surprisingly long gap – and one that probably will not be repeated.
‘Project Optimus’ is continuing to press forward on reframing the approach to dosing of anticancer drugs. The initiative is very personal to OCE Director Richard Pazdur.