Policy

In this episode of Denatured, you’ll listen to Oxana Iliach, senior director of regulatory strategy at Certara and Vera Pomerantseva, director of product management for risk-based quality management at eClinical Solutions. We speak about how the FDA’s latest decision to have one, rather than two pivotal studies, for new drug applications raises the bar for data collection and risk-based management.

The senator, who has long advocated for expanding access to experimental therapies, reportedly called the FDA’s request for a sham surgery–controlled Phase 3 trial for uniQure’s Huntington’s disease gene therapy “bureaucratic idiocy.”

Single-trial approvals are raising the bar on trial design and execution. The new paradigm is pushing sponsors to plan earlier, step up their data and risk‑based quality management and use modeling and AI to generate one compelling, regulator‑ready evidence package.

Industry groups have identified upfront costs as a barrier to streamlining U.S. drugmaking. The nonprofit API Innovation Center has a proposed answer for how to tilt finances in favor of investments in continuous manufacturing.

Adam Urato, who is currently a vaccine advisor to the CDC, is closely associated with acting CDER director Tracy Beth Høeg and is a fellow skeptic of the use of selective serotonin reuptake inhibitors during pregnancy.

One of the two new members of the CDC’s Advisory Committee on Immunization Practices questioned the safety of COVID-19 vaccines before the Texas Senate in 2021.

Aside from creating a toxic work environment, CBER Director Vinay Prasad has also been accused of berating his staff and retaliating against reviewers who questioned his decisions.

Without naming a specific product, Commissioner Marty Makary referred to an investigational therapy, delivered surgically into the brain, that the FDA was “pressured” to approve even after finding no clinical benefit to patients.

Last week, the FDA made its one pivotal trial policy official, sparking myriad questions from industry leaders, including around specific evidence required for the single study and why it hasn’t been implemented across all therapeutic areas before now.

The rescheduling of the Advisory Committee on Immunization Practices comes amid fresh leadership upheaval at the CDC as NIH director Jay Bhattacharya replaces Jim O’Neill as acting head of the agency.