Uncertainties in New Cancer Drug Approvals

A study published in Health Affairs has found that the FDA's benefit-risk framework for cancer drugs has identified significant uncertainties in new cancer drug approvals. The researchers, led by the London School of Economics and Political Science, analyzed 52 new cancer drug approvals between 2019 and 2022 and found that the FDA had identified 213 clinical trial uncertainties, with 50% of them considered important to the approval decision. However, only 26% of all uncertainties and 48% of the important uncertainties were reported in the FDA-approved prescribing information.

Key Takeaways:

• The FDA's benefit-risk framework for cancer drugs has identified significant uncertainties in new cancer drug approvals, affecting 50% of the drugs approved between 2019 and 2022.
• FDA reviewers identified 213 clinical trial uncertainties, with 50% of them considered important to the approval decision.
• Only 26% of all uncertainties and 48% of the important uncertainties were reported in the FDA-approved prescribing information.
• The study suggests that communicating uncertainties about the evidence of drugs in the label is essential for informing physicians about drugs' safe and effective use.
• The researchers used FDA review documents for uncertainties identified by FDA reviewers with new cancer drugs.
• Notably, physicians might not regularly consult the drug label for prescribing decisions, but other information sources used by physicians either index or incorporate information from the label.

Statistics:

• 52 new cancer drugs were approved by the FDA between 2019 and 2022.
• 213 clinical trial uncertainties were identified by FDA reviewers, with 50% considered important to the approval decision.
• Only 26% of all uncertainties and 48% of the important uncertainties were reported in the FDA-approved prescribing information.
• The study analyzed FDA review documents from 2019-22.
• The study was conducted by researchers from the London School of Economics and Political Science, including Avi Cherla, Steven Woloshin, Anita K. Wagner, Olivier J. Wouters, Elias Mossialos, Huseyin Naci, and Courtney Davis.

Sources:

• New Cancer Drug Approvals: Less Than Half of Important Clinical Trial Uncertainties Reported By the Fda To Clinicians, 2019-22. Health Affairs, 2025;44(7):830-838.
• London School of Economics and Political Science.