The process of accelerated approval by the US Food and Drug Administration (FDA) has been pivotal in expediting promising drugs to market, particularly in critical areas such as oncology. However, concerns have been raised regarding the efficacy and clinical benefit of drugs approved through this pathway. A recent study, published in JAMA, examined the outcomes of cancer drugs granted accelerated approval between 2013 and 2023, focusing on the demonstration of clinical benefit through confirmatory trials.
The study analysed 129 accelerated approvals for cancer indications granted between 2013 and 2023. Two main analyses were conducted: the first assessed the clinical benefit of drugs with at least 5 years of follow-up, while the second focused on the evidence supporting conversion decisions to regular approval. The evaluation included criteria such as overall survival, quality of life, and the characteristics of confirmatory trials.
Among the 46 indications with at least 5 years of follow-up, only 63% were converted to regular approval, with the majority failing to demonstrate a benefit in overall survival or quality of life.
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Pivotal trials for these indications often relied on surrogate measures such as tumour response rate and progression-free survival, raising concerns about the accuracy of these predictors in assessing clinical benefit.
Of the indications converted to regular approval, approximately two-thirds showed improvements in overall survival or quality of life, while one-third failed to demonstrate significant improvement in these outcomes.
A notable trend observed was the increasing use of surrogate measures like response rate to support conversion from accelerated to regular approval, despite concerns about their ability to accurately predict clinical benefit.
The study highlights the need for careful consideration of the clinical benefit of drugs granted accelerated approval, especially in oncology. Patients and healthcare providers should be aware of the limitations associated with drugs approved through this pathway, particularly those that do not demonstrate significant improvements in patient-centred outcomes. The study authors stressed that regulatory agencies should prioritise the collection of robust clinical data, including overall survival and quality of life, to ensure that drugs granted accelerated approval offer meaningful benefits to patients.
The findings of this study underscore the importance of evaluating the clinical benefit of cancer drugs approved through accelerated pathways. By critically assessing the evidence supporting these approvals, stakeholders can make more informed decisions regarding the use of these therapies in clinical practice. Ultimately, ensuring that drugs provide tangible benefits to patients should remain the cornerstone of drug approval processes.
