FDA Draft Guidance Provides Recommendations on Cancer Trial Endpoints

The most reliable method for demonstrating the efficacy of a cancer drug or biologic is to show a statistically significant improvement in a clinically meaningful endpoint in blinded, randomized, controlled trials, but there are single-arm study approaches that might be successful in certain settings, explains a new FDA draft guidance.

The draft guidance is the first in a series of guidance documents the agency is drafting to provide recommendations on clinical trial endpoints for the approval of cancer drugs and biologics. The initial draft guidance provides background and discusses general regulatory principles for oncology endpoints. Each subsequent guidance in the series will focus on endpoints for specific cancer types, the agency said.

In addition to addressing single-arm studies, the draft guidance contains a detailed comparison of important cancer approval endpoints. The document analyzes the advantages and disadvantages of each endpoint and provides recommendations on the types of endpoints that might be most effective in certain clinical situations.

To view the guidance, "Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics," go to http://www.fda.gov/cber/gdlns/clintrialend.pdf (http://www.fda.gov/cber/gdlns/clintrialend.pdf).