Mapping the divide: Early development of global cancer drugs and its mismatch with cancer burden

The misalignment between cancer burden and drug innovation poses significant challenges for global health. However, the global scope, temporal trends, and underlying factors contributing to this mismatch remain underexplored. We aim to evaluate the extent, trends, and driving forces behind the disparity between early drug development and cancer burden. This retrospective cohort study encompasses a total of 9473 early phase cancer drug trials from 1990 to 2023. Concentration curves and concentration indices (CIs) were employed to measure and track the mismatch over time. Residual analysis derived from a regression model identified cancers that may be either overlooked or overly focused upon. Decomposition of CIs was applied to ascertain the contributors to this mismatch and its evolution. The CI between early drug development and cancer burden exhibited an upward trend over time, rising from 0.105 (95 % CI: 0.015 to 0.225) in the 1990s to 0.208 (95 % CI: 0.092 to 0.326) in the 2020s. Early drug development activities had disproportionately favored high-burden cancers. Demand-side factors, such as disease burden (average contribution: 53.35 %) and market size (average contribution: 25.16 %), were the primary drivers of both the mismatch and its growth. The primary drivers of early drug development are medical and market demands, which lead to disproportionate focus on more prevalent or commercially attractive cancers. To address these disparities, targeted initiatives and policy reforms are necessary to ensure that drug development aligns more closely with global health needs, especially for cancers that receive insufficient attention. • The misalignment between cancer burden and drug innovation continues to challenge global health. • Concentration indices rose from 0.105 (1990s) to 0.208 (2020s), showing focus on high-burden cancers. • Demand-side factors, including disease burden (53.35 %) and market size (25.16 %), drive this mismatch and its escalation. • Hodgkin lymphoma, thyroid cancer, and testicular cancers remain persistent underdevelopment. • Alignment requires targeted funding, enhanced industry-academia collaboration, and improved technology transfer.