FDA Withdrawals and Oncology Drug Use
The U.S. Food and Drug Administration’s (FDA) accelerated approval programme allows certain oncology drugs to enter the market based on surrogate measures of efficacy. However, if post-approval confirmatory trials fail to demonstrate clinical benefit, the FDA removes these indications from the drug’s labelling. Despite this, such medications may still be prescribed off-label. A recent Review published in JAMA Oncology examines how oncologists adapt prescribing patterns following the withdrawal of an accelerated approval indication.
A research team led by Dr. Catherine S. Hwang and colleagues analysed changes in oncology drug use after the removal of accelerated approval indications. They focused on four drug-indication pairs:
- Atezolizumab for breast cancer
- Atezolizumab for urothelial cancer
- Idelalisib for follicular lymphoma
- Romidepsin for peripheral T-cell lymphoma
Using data from the Optum Clinformatics DataMart Database, researchers identified patients who had been diagnosed with these conditions and followed their medication usage patterns. The study assessed changes in drug utilisation before and after the publication of negative trial results or FDA safety warnings.
The study included 762,752 patients and found significant shifts in prescribing patterns following negative trial results. Before the release of these findings, the monthly prevalence of medication use had increased substantially. However, once negative results were publicised, usage dropped significantly.
For instance, atezolizumab use in breast cancer patients increased by 16 cases per 1 million patients before trial results but decreased by 28 per 1 million afterwards. Similar declines were observed for idelalisib and romidepsin. Interestingly, romidepsin continued to be prescribed for peripheral T-cell lymphoma at a stable rate, likely due to its demonstrated efficacy in certain subpopulations.
These findings suggest that oncologists rapidly integrate new clinical evidence into practice, adjusting treatment plans in response to evolving data. The decline in off-label prescribing post-trial suggests that either clinicians are proactively limiting use or payers are restricting coverage of ineffective treatments.
Some drugs remained in use for extended periods before their indications were officially withdrawn, exposing patients to potentially ineffective or unsafe therapies.
However, the study also highlights concerns about delays in regulatory action. Some drugs remained in use for extended periods before their indications were officially withdrawn, exposing patients to potentially ineffective or unsafe therapies. The researchers emphasise the need for timely post-approval studies and swift regulatory enforcement to protect patients from unnecessary risks.
This study underscores the importance of ongoing vigilance in oncology drug regulation. Oncologists should remain aware of evolving drug efficacy data and advocate for policies ensuring the timely removal of ineffective therapies from clinical use. As the accelerated approval pathway continues to play a role in oncology, balancing rapid access to promising treatments with rigorous post-market evaluation will be critical.
Paper: Hwang CS. Changes in Oncology Medication Use After Withdrawal of Accelerated Approval. JAMA Oncol. Published online April 03, 2025. doi:10.1001/jamaoncol.2025.0359. Access online here.
