Objective  To evaluate the efficacy, safety and economy of cyclin-dependent kinase 4/6 (CDK4/6) inhibitors for the first-line treatment of hormone receptors positive (HR+), human epidermal growth factor receptor 2 negative (HER2-) advanced breast cancer (ABC) by rapid health technology assessment, and to provide evidence for clinicians and policymakers.

Methods  PubMed, Cochrane Library, Embase, CNKI, WanFang Data, VIP databases and the official website of health technology assessment (HTA) agency were electronically searched to collect clinical evidence and literature of CDK4/6 inhibitors in the treatment of HR+/HER2-ABC from the inception to December 31, 2023. Two reviewers independently identified studies, extracted data, assessed the quality of included studies, and descriptively analyzed and summarised the results.

Results  A total of 33 articles were included, including 9 systematic reviews/Meta-analyses, 15 pharmacoeconomic studies and 9 HTA reports. In terms of efficacy, compared with endocrine therapy alone, the addition of CDK4/6 inhibitors significantly improved progression-free survival (PFS) and overall survival (OS) in patients with HR+/HER2-ABC (P<0.05), but there was no significant difference in efficacy among palbociclib, abemaciclib and ribociclib (P>0.05). In terms of safety, more adverse events were observed in patients treated with CDK4/6 inhibitors when compared with endocrine therapy (P<0.05). There was a difference in the incidence of adverse effects between the different CDK4/6 inhibitors, with palbociclib having higher incidence of haematological adverse effects (P<0.05), and abemaciclib being more likely to cause gastrointestinal adverse reactions such as diarrhoea (P<0.05). The economic evaluation results were variable due to differences in healthcare costs, analysis perspectives, willingness-to-pay thresholds, and study duration in different countries.

Conclusion  CDK4/6 inhibitors have similar efficacy in the first-line treatment of HR+/HER2-ABC patients, but there are some differences in aspects such as safety and economy.

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