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.
Glasmacher et al. (Sat,) studied this question.