FDA Strengthens Requirements for Confirmatory Trials, Emphasizing Early Planning and Due Diligence

Key Insights

• The FDA is reinforcing the necessity of early planning and thorough due diligence in confirmatory trials for drugs and biologics, impacting accelerated approvals.
• New draft guidance from the FDA reflects strategies promoted by the Oncology Center of Excellence (OCE) for efficient drug development.
• A single randomized study may now be sufficient for both accelerated approval and subsequent verification of clinical benefit under the updated guidelines.

The U.S. Food and Drug Administration (FDA) is intensifying its scrutiny of confirmatory trials, emphasizing the critical need for advance planning and due diligence in these studies. This shift, detailed in a new draft guidance, impacts the approval pathway for drugs and biologics, particularly those receiving accelerated approval based on preliminary evidence.

The draft guidance incorporates strategies championed by the FDA's Oncology Center of Excellence (OCE). These strategies include early planning of confirmatory trials and the potential use of a single, well-designed randomized study to support both the initial accelerated approval and the later verification of clinical benefit. This approach aims to streamline the drug development process while ensuring that approved therapies demonstrate tangible benefits for patients.

The FDA's move reflects a broader effort to optimize the efficiency and rigor of drug development. By emphasizing early planning and a more streamlined approach to confirmatory trials, the agency seeks to reduce the time and resources required to bring new therapies to market, without compromising the standards for safety and efficacy. The guidance is broadly applicable to drugs and biologics, marking a significant step in the FDA's approach to drug regulation.