Pertuzumab-based regimens and FOLFIRINOX are cost-effective for specific cancers while neoadjuvant pembrolizumab is notNew data shows cost-effectiveness of specific cancer drug regimens

Neoadjuvant drug therapy has become an important component of multidisciplinary cancer treatment, but newer targeted, immunotherapy-based, and multi-agent regimens are often associated with higher costs and uncertainty regarding long-term economic value. This systematic review evaluates the economic evidence for neoadjuvant drug regimens in cancer and summarizes key cost-effectiveness findings across tumor types and healthcare settings. We systematically searched PubMed, Embase, and the Cochrane Central Register of Controlled Trials (CENTRAL) from database inception to June 1, 2026. Eligible studies were full economic evaluations comparing neoadjuvant drug regimens or assessing the addition of a specific drug component to a baseline neoadjuvant regimen in patients with cancer. Studies comparing neoadjuvant drug regimens with non-drug-based or non-neoadjuvant strategies were excluded. Study selection and data extraction were performed independently by two reviewers. Reporting completeness was assessed using the CHEERS 2022 checklist, potential sources of bias were assessed using the ECOBIAS checklist, and findings were narratively synthesized. A total of 2,572 records were identified, and 7 studies met the inclusion criteria. The included studies were published between 2015 and 2024 and involved breast cancer, muscle-invasive bladder cancer, and borderline resectable or locally advanced pancreatic cancer. Five studies focused on breast cancer, mainly HER2-positive disease. Most studies used model-based approaches and quality-adjusted life-years as the primary outcome. Pertuzumab-based neoadjuvant regimens for HER2-positive breast cancer and FOLFIRINOX for pancreatic cancer were generally reported to be cost-effective in their respective healthcare settings. In contrast, neoadjuvant pembrolizumab for muscle-invasive bladder cancer and AC-TH compared with TCH for breast cancer were not cost-effective under base-case assumptions. Key drivers included pathological complete response, recurrence or progression risk, drug costs, utility values, adverse event costs, time horizon, and assumptions linking short-term efficacy to long-term outcomes. Current economic evidence remains limited and concentrated mainly in HER2-positive breast cancer. Cost-effectiveness conclusions depend strongly on drug prices, downstream treatment costs, and model-based extrapolation of early efficacy endpoints. Future studies should incorporate long-term follow-up and real-world evidence. https://www.crd.york.ac.uk/PROSPERO/view/CRD420261462564, Identifier CRD420261462564.