Reimagining Clinical Studies To Support Accelerated Approval For Anticancer Products

By Jason Birri, Hema Balasubramanian, and Anna Hertzberg

The demand for speed in drug development has always existed. Balancing the urgency for new therapies with thorough clinical evaluation is crucial. This places health authorities, like the U.S. Food and Drug Administration (FDA), in a challenging position, where they must protect the public from unsafe or ineffective drugs without unduly delaying vital therapies.

Oncology drug development has historically prioritized the swift delivery of promising therapies to patients with limited options. However, recent advancements, especially in targeted therapies, are shifting this paradigm. As safer and more effective targeted therapies emerge, the benefit-risk landscape for new oncology drugs evolves.

A significant turning point was the launch of FDA's Project Optimus, which focuses on optimizing dose selection to enhance safety and tolerability. The FDA has also shifted its attention to improving the Accelerated Approval (AA) pathway for anticancer products. Through AA, conditional product approval is based on limited clinical data with confirmatory studies using traditional endpoints. However, the number of drugs approved through AA has increased, leading to scrutiny and controversies.

To address concerns, the FDA's Office of Oncologic Drugs and the Oncology Center of Excellence have conducted multiple reviews. These assessments prompted a need for more frequent surveillance and a reevaluation of the clinical studies required to support AA for anticancer products.