OBJECTIVES: Nanotechnology enabled drug-delivery facilitates highly targeted and therefore potentially more effective treatment options. This innovation has the potential to significantly improve the cost-effectiveness of existing valued interventions, especially in Oncology. However, despite decades of commercialisation attempts, nanotherapeutics struggle with demonstrating pharmacoeconomic value. This research will investigate the sources of challenges associated with generating cost-effectiveness evidence for nanomedicine applications in cancer treatment.

METHODS: A targeted search of the PubMed database was conducted using search terms for nanomedicine, oncology, and pharmacoeconomic studies. Articles were included according to pre-defined criteria; publications between 2007-2024, review articles and publications that included cost-effectiveness analysis (CEA) studies were prioritised. Identified studies on CEAs of oncological nanomedicines were included and data was extracted on the gaps in evidence generation.

RESULTS: The search yielded 13 publications with assessments of pharmacoeconomic valuation of nanomedicines in oncology. Out of the extracted articles, 9/13 discussed CEAs of oncological nanomedicines, and 1/13 had a systematic review of CEAs (n=9). The overlapping theme of the extracted sources was that few CEAs of oncological nanomedicines are available, and those that are, contain methodological gaps.

CONCLUSIONS: Key challenges in generating cost-effectiveness evidence for oncological nanomedicines originate from one of two causes: 1) lack of internationally standardised safety and efficacy guidance leads to poor clinical throughput that limits opportunities for conducting CEAs 2) No comprehensive cost-taxonomy in nanomedicine CEAs leads to ineffective capture of generated value and impedes effective comparison of results. For the purposes of commercialisation, the quality of cost-effectiveness evidence is insufficient, as payers frequently site this as the main weakness in nanomedicine applications. This is particularly important, as other value attributes, like innovation, are insufficient to compensate and make a strong case for reimbursement.