Many cancer patients may be treated with drugs that were granted accelerated approval but do not provide a clinical benefit, according to research published in JAMA Oncology.

Researchers quantified the use of drugs that were granted accelerated approval for 5 oncology indications, but those approvals were later withdrawn due to negative trial results. 

In a cohort of 4342 cancer patients, about 26% of all treatments they received involved the drugs for which accelerated approval was withdrawn. 

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The cohort included patients with advanced or recurrent triple-negative breast cancer (TNBC), bladder cancer, hepatocellular carcinoma (HCC), gastric cancer, or small-cell lung cancer (SCLC) who were evaluated between May 18, 2016, and March 8, 2022. 

The patients were eligible if they received at least 1 line of systemic therapy during this period. In total, the patients received 6560 lines of therapy.  

The researchers estimated the prevalence of treatment with the drugs that were temporarily granted accelerated approval for the 5 oncology indications. The accelerated approvals were for:

  • Atezolizumab as first-line treatment for TNBC
  • Pembrolizumab as third-line treatment for PD-L1-positive gastric cancer
  • Atezolizumab as second-line or later treatment for bladder cancer
  • Nivolumab as second-line or later treatment for HCC
  • Nivolumab as second-line or later treatment for SCLC. 

The median time from accelerated approval to withdrawal was 46 months overall (range, 12-58 months). Between accelerated approval and withdrawal, 26.1% of all cancer treatments involved 1 of the 5 treatments that had accelerated approval withdrawn. 

The prevalence of treatment with the withdrawn drugs was higher between the time of accelerated approval and the time of negative trial publication (35.5%) than between the negative trial publication and ultimate withdrawal (15.7%). 

Prevalence of Treatment With Drugs That Were Withdrawn After Accelerated Approval


Overall Cohort

Atezolizumab for TNBC

Pembrolizumab for Gastric Cancer

Atezolizumab for Bladder Cancer

Nivolumab for HCC

Nivolumab for SCLC

Prevalence of Use from Approval to Withdrawal







Prevalence of Use from Approval to Negative Trial







Prevalence of Use from Negative Trial to Withdrawal







“An expected tradeoff exists between expediting access to promising cancer drugs and withdrawal of some indications,” the researchers wrote. “Given the growth of withdrawals due to negative confirmatory trials and emerging evidence on the high spending associated with AA [accelerated approval] drugs, it is critical to balance early access against population-level exposure to cancer therapies with no benefit over standard of care.” 

Disclosures: Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of disclosures.


Parikh RB, Hubbard RA, Wang E, et al. Exposure to US cancer drugs with lackof confirmed benefit after US Food and Drug Administration accelerated approval. JAMA Oncol. Published online February 23, 2023. doi:10.1001/jamaoncol.2022.7770

This article originally appeared on Cancer Therapy Advisor