Assessing the value of cancer treatments from real world data—Issues, empirical examples and lessons learnt. (March 2017)

• Record Type: Journal Article
• Authors: Moen, F.; Svensson, J.; Steen Carlsson, K.
• Is Part Of: Journal of cancer policy. Volume 11(2017)
• Page Start: 32
• Page End: 37
• Publication Date: 2017-03
• Subjects: Chronic myeloid leukemia -- Prostate cancer -- Regional variations -- Real world evidence -- Real world data -- Value of medicines -- Sustainability; Cancer -- Government policy -- Periodicals; Cancer -- Patients -- Services for -- Periodicals; Medical Oncology -- Periodicals; Public Health -- Periodicals; Cancer; Periodicals; 362.196994
• DOI: 10.1016/j.jcpo.2016.11.001
• Languages: English
• ISSNs: 2213-5383

Abstract

Highlights: Real world data is increasingly important as the pathways for introduction of new medicines in cancer care changes and the number of treatment options including combination therapies multiply. There are a range of issues relevant to estimating the value of a medicine can only be answered by non-intervention, observational studies, preferably in several settings. 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. To be useful, studies of real world evidence must have well-defined patients and preferably include a control group. As the stratification of disease and treatment continues, larger cohorts of patients are needed to supply sufficient data. European cooperation can help generate needed data. Abstract: 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.