US
FDA
approval
drugs
benefits
cancer
Click for Source
Exposure to US Cancer Drugs With Lack of Confirmed Benefit After US Food and Drug Administration Accelerated Approval
Summary
- Among 5 oncology indications, 26.1% of eligible treatment initiations involved an Accelerated Approval (AA) indication that was later withdrawn due to lack of benefit.
- There is an inherent trade-off between expediting access to promising cancer drugs and the potential withdrawal of some indications.
- It is crucial to balance early access against the risk of exposing the population to cancer therapies that show no benefit over standard care.
- Limitations of the study include the inability to assess population-level exposure due to insufficient samples and follow-up for analysis in only 5 withdrawn AA indications.
- The Accelerated Approval Integrity Act proposed in March 2022 aims for earlier access and quicker FDA responses to negative confirmatory trial data to minimize exposure to ineffective AA therapies.
Between 2009 and 2022, the US Food and Drug Administration (FDA) approved 48 drugs for 66 oncology-related indications under the Accelerated Approval (AA) program. Indications granted AA based on surrogate end points are subsequently required to confirm clinical benefit. Since 2009, 15 indications (23%) have been withdrawn due to lack of benefit over standard of care.
We estimate the proportion of patients who received treatment for 5 oncology-related indications later withdrawn after failure to confirm efficacy under the AA program...
US, FDA, approval, drugs, benefits, cancer
