Assessing the value of cancer treatments from real world data-Issues, empirical examples and lessons learnt

There is an increasing demand for real world evidence. The shift towards relative effectiveness assessment increasingly based on real world data is a natural consequence of the shift towards new adaptive pathways for development and introduction of new medicines in cancer care. The increasing number of alternative treatment options will further increase the need for outcomes data to help optimize the clinical pathways and resource allocation. In this article the authors explore the opportunities and challenges of real world evidence based on three case studies in cancer care. The central theme is to identify what knowledge gaps can be filled by real world data. Three areas of utility are identified: (1) to validate surrogate endpoints impact against hard endpoints and outcomes over time; (2) to valuate new treatments outside the strict protocol of clinical trials and (3) to optimize the value of new treatments based on regional variations in uptake. The authors also reflect upon how to increase the availability of real-world evidence and ensure sustainable access to needed data. European collaboration could be part of the solution.