Challenges and Potential Solutions to Advance Global Cancer Drug Development

Abstract

Despite recent advancements in oncology drug development, patient access to innovative cancer therapies remains inadequate. There is an urgent need for more patient-centric approaches, with meaningful patient input from trial design through to health technology assessment (HTA) consultation. Multi-stakeholder consensus calls for better representation of the diversity of the target population and integration of patients' preferences in clinical cancer research by systematically collecting patient-reported outcomes using standardized methods, and acknowledging trade-offs between survival and long-term wellbeing. Furthermore, the generation of insufficiently robust data for regulatory and HTA decision-making continue to delay patient access to innovation. This could be mitigated through smarter study designs, including smaller, fit-for-purpose randomized studies and prospectively designed trials. Finally, concerted efforts are required to develop and validate novel intermediate/surrogate endpoints that enable earlier assessment of treatment outcomes to facilitate timely, evidence-based decisions that improve the patient experience across the cancer care continuum.