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

Moen F, Svensson J & Steen Carlsson K

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.

Three areas are identified where RWE can be of particular value: (1) to validate surrogate endpoints impact against hard endpoints and outcomes over time; (2) to evaluate 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 on the need for larger cohorts of patients as the stratification of disease and treatment continues.

The special issue of Journal of Cancer Policy Vol 11 (March 2017) can be found here and all papers are freely available from Friday 27th January, for eight weeks.


Journal of Cancer Policy 2017; 11: 32–37
DOI: http://dx.doi.org/10.1016/j.jcpo.2016.11.001