Availability of emergency authorization route in US depends upon lack of 'adequate, approved and available alternatives,' but as more vaccines make their way through the BLA process sponsors may find the EUA door slowly closing.

Authorized for mild-to-moderate COVID-19 in patients 12 and older at high risk for severe disease, Paxlovid (nirmatrelvir/ritonavir) jumped ahead of Merck & Co./Ridgeback’s molnupiravir in the EUA queue for oral antivirals, with the latter possibly hung up by efficacy and safety concerns.

Move toward decentralized trials means sponsors may be working with naïve clinical investigators who lack experience, Greenleaf’s Cynthia Schnedar says; DoJ’s Gustav Eyler urges drug sponsors to look for oddities in clinical trial data and to voluntarily report potential misconduct.

At his Senate confirmation hearing, Robert Califf said he wants to establish a system, built on electronic medical records, that can be used to more quickly confirm the benefits and risks of accelerated approval drugs.

Office of Tissues and Advanced Therapies seeks to optimize its organizational structure and adopt more group-level communications, instead of one-on-one interactions with sponsors, to keep up with the massive growth in cell and gene therapies, director Wilson Bryan says.

US FDA’ s latest ‘n of 1’ guidance describes clinical considerations for INDs to support dosing and monitoring of an individual with a life-threatening genetic disease who is eligible to receive an ASO; recommendations apply only when a few patients may be candidates for treatment.