The FDA is advancing its effort to reduce unnecessary animal testing for cancer therapies and other treatments. In draft guidance released May 29, the agency recommended that companies conducting non-clinical safety assessments of certain biologics use toxicology studies in a single rodent species, provided sponsors can demonstrate that these therapies act similarly in humans and rodents. For other molecules, sponsors may instead conduct pharmacology studies showing the asset binds its intended target and triggers intended effects.
New risk assessment framework
The FDA outlined a “Weight of Evidence risk assessment” approach to establish non-clinical safety of therapeutic candidates without animal studies. These submissions can include pharmacodynamic and pharmacokinetic data, along with literature-based analysis of safety issues associated with the disease target. According to the draft document, “by reducing animal testing and incorporating an integrated knowledge-based risk assessment, this guidance is anticipated to facilitate greater efficiencies in product development without compromising patients’ safety.”
Complementary advances in cell therapy development
The FDA is also opening pathways to speed cell and gene therapy development. New draft guidance allows sponsors to leverage what the agency called “prior knowledge” or “generally accepted scientific knowledge” for their submission packages—including manufacturing information, non-clinical data, or clinical evidence. Companies may also use “platform knowledge” gleaned from developing similar technologies or products, including mechanisms of action, delivery modes, and manufacturing processes. The agency noted that companies should justify why selected prior knowledge applies to their particular product in development.
This article is an AI-assisted summary. All facts and figures are drawn from the original report: https://www.biospace.com/fda/fda-policy-tracker-2026-priority-vouchers-questioned-prvs-return