New Cancer Drug Approvals: Less Than Half Of Important Clinical Trial Uncertainties Reported By The FDA To...

Area of Science:

• Oncology
• Drug Safety
• Regulatory Science

Background:

• Uncertainties regarding the benefits and harms of newly approved drugs are common.
• The Food and Drug Administration (FDA) drug label is the primary communication channel for this information to physicians.
• Physicians may not always consult the drug label directly, but other information sources often incorporate label data.

Purpose of the Study:

• To assess the extent to which the FDA communicates uncertainties about new cancer drugs in their approved labeling.
• To determine if uncertainties highlighted as important in the FDA's Benefit-Risk Framework are adequately conveyed.

Main Methods:

• Systematic search of FDA review documents for new cancer drugs approved between 2019 and 2022.
• Identification and categorization of clinical trial uncertainties by FDA reviewers.
• Analysis of the FDA-approved drug labels to determine the reporting of these identified uncertainties.

Main Results:

• FDA reviewers identified 213 clinical trial uncertainties for 52 new cancer drugs, with 50% deemed important to the approval decision.
• Information on only 26% of all identified uncertainties was present in the physician labels.
• Crucially, only 48% of uncertainties considered important to the FDA's approval decision were reported in the drug labels.

Conclusions:

• The FDA's communication of drug uncertainties in approved labeling is incomplete, particularly for uncertainties critical to the approval decision.
• Enhanced communication of drug evidence uncertainties in labels is essential for informed physician decision-making regarding safe and effective drug use.
• There is a significant gap between identified drug uncertainties and their reporting in FDA-approved prescribing information.