Assessing the value of cancer treatments from real world data—Issues, empirical examples and lessons learnt
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- 作者：F. Moen ; fredrik.moen@ihe.se ; J. Svensson ; K. Steen Carlsson
- 关键词：Chronic myeloid leukemia ; Prostate cancer ; Regional variations ; Real world evidence ; Real world data ; Value of medicines ; Sustainability
- 刊名：Journal of Cancer Policy
- 出版年：2017
- 出版时间：March 2017
- 年：2017
- 卷：11
- 期：Complete
- 页码：32-37
- 全文大小：331 K
- 卷排序：11
文摘
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.