Real-world data: towards achieving the achievable in cancer care

Abstract

The use of data from the real world to address clinical and policy-relevant questions that cannot be answered using data from clinical trials is garnering increased interest. Indeed, data from cancer registries and linked treatment records can provide unique insights into patients, treatments and outcomes in routine oncology practice. In this Review , we explore the quality of real-world data (RWD), provide a framework for the use of RWD and draw attention to the methodological pitfalls inherent to using RWD in studies of comparative effectiveness. Randomized controlled trials and RWD remain complementary forms of medical evidence; studies using RWD should not be used as substitutes for clinical trials. The comparison of outcomes between nonrandomized groups of patients who have received different treatments in routine practice remains problematic. Accordingly , comparative effectiveness studies need to be designed and interpreted very carefully. With due diligence, RWD can be used to identify and close gaps in health care, offering the potential for short-term improvement in health-care systems by enabling them to achieve the achievable.

Assessing the efficacy‐effectiveness gap for cancer therapies: A comparison of overall survival and toxicity between clinical trial and population‐based, real‐world data for contemporary parenteral cancer therapeutics

“…RWE has the potential to serve many purposes with increasing interest in the use of real-world data to broader applications outside of informing day-today clinical decision making. 60 For example, efforts to use more representative data for regulatory decisions currently are underway, notably with the recently established framework for the US FDA's Real-World Evidence Program. 61,62 This program aims to use RWE to either support the approvals of new therapies or satisfy Cancer April 15, 2020 postapproval study requirements through postmarketing surveillance.…”

Effectiveness of image‐guided radiotherapy for locally advanced lung cancer patients treated with definitive concurrent chemoradiotherapy

“…Furthermore, although this RCT 8 failed to show superiority in its primary endpoint (recurrence-free survival, HR 0.81 P = 0.33), post hoc analyses did show a benefit to IGRT in biochemical progression-free, clinical progression-free interval, and rectal toxicity. From the viewpoint of evidence-based medicine, our finding was not a high level evidence 15,26 and should be cautiously interpreted due to the nonrandomized study design. 15 Although OS was obviously the most important endpoint in cancer treatment, it may not be the most relevant endpoint for IGRT, and our study lacked the data on local control or toxicity which might be better endpoints for IGRT.…”

“…In the primary analysis (PA), we adopted the propensityscore (PS) method to balance the measured potential confounders. [13][14][15] We evaluated the probability of receiving IGRT (vs. non-IGRT) with a logistic regression model based on all the above covariates, used the logit of the probability as the PS, and then assessed the balance of covariates between groups via the standardized difference (SDif). 16,17 We compared the hazard ratio (HR) of death between the IGRT and non-IGRT groups during the entire follow-up period via a PS weighting approach with overlap weights as suggested in studies.…”

Radiomics and imaging biomarkers in observational clinical studies with retrospective data

“…En este sentido, tanto los estudios prospectivos, tipo Ensayos Clínicos, como los retrospectivos, especialmente los realizados con casos del mundo real (RWD), presentan sus ventajas pero también sus inconvenientes (6). La principal ventaja de los estudios prospectivos experimentales es el control y la homogeneidad de la muestra, con criterios de inclusión y de exclusión bien definidos desde el principio.…”