Harnessing real-world data in cancer care to improve efficiency, value, and affordability in healthcare interventions

As treatment expenses and cancer rates increase among an aging population, the shortcomings of conventional randomized controlled trials (RCTs) are becoming evident. They are costly, slow, and often not representative of the real patient population. Real-world data (RWD) could improve the efficiency and affordability of cancer treatment.
The first part examines efficiency, value, and affordability in oncology, offering seven recommendations to enhance efficiency. It demonstrates that "value" and "affordability" lack uniform definitions. Different stakeholders—clinicians, payers, industry, and health scientists—prioritize varying aspects like survival benefit, safety, quality of life, or cost when evaluating cancer treatments. This requires standardized frameworks and dialogue between stakeholders.
The practical part demonstrates concrete applications of RWD in oncology:
- By evaluating gravimetric measurement data from chemotherapy preparation, significant dosage deviations were detected in 2.25% of medications, which would have gone undetected in traditional preparation with a syringe.
- To improve data interoperability, a standardized coding system for therapy regimens was developed and implemented in a prototype database. This enables cross-institutional comparisons.
- Despite testing costs, KRAS testing for metastatic colorectal cancer saves €780 per patient per cycle by avoiding ineffective EGFR inhibitor therapies in mutation carriers.
The paper synthesizes findings and recommends integrating RWD into clinical and policy decisions. RWD does not replace RTCs, but instead expands the evidence base to include aspects of real-world effectiveness, safety, and cost-effectiveness. For successful implementation, obstacles like data quality, interoperability, and cultural change must be overcome. Cross-sector collaboration between regulators, clinicians, payers, scientists, and industry is essential.