Identifying Factors Associated with the Acquisition of Multiple Indications for Anticancer Drugs.

With the declining success rate of new drug development, lifecycle management strategies have become increasingly important. However, approximately 25-40% of approved drugs remain limited to a single indication. In this study, we compared drugs that obtained multiple indications with those confined to a single indication, aiming to identify factors associated with the acquisition of multiple indications. We identified anticancer drugs initially approved by US Food and Drug administration (FDA) between 2015 and 2020. This retrospective observational study includes 48 anticancer drugs for solid tumors. For each drug, data were collected on approved indications, sponsoring company characteristics, and clinical efficacy outcomes, including objective response rate (ORR) and progression-free survival (PFS). The drugs were categorized based on the number of approved indications, and comparative analyses were conducted. No clear association was observed between multiple indication acquisition and factors such as mechanism of action (MoA) or company size at the initial approval. Since additional indications may be obtained through various approaches-including expansion to new tumor types or different lines of therapy-no consistent relationship was observed between specific strategies and the acquisition of multiple indications. In contrast, drugs demonstrating higher clinical efficacy and those initially approved for non-rare cancers were more likely to achieve multiple indications. The factors associated with additional indications varied depending on the approach, and only a limited set of factors consistently correlated with multiple indication acquisition. Notably, clinical efficacy and the tumor type at initial approval appeared to be associated with acquisition of multiple indications. These findings provide important insights into research and development strategies and may inform decision-making in pharmaceutical lifecycle management.