How do cancer clinicians perceive real-world data and the evidence derived therefrom? Findings from an international survey of the European Organisation for Research and Treatment of Cancer

Abstract

Background: The role of real-world evidence (RWE) in the development of anticancer therapies has been gradually growing over time. Regulators, payers and health technology assessment agencies, spurred by the rise of the precision medicine model, are increasingly incorporating RWE into their decision-making regarding the authorization and reimbursement of novel antineoplastic treatments. However, it remains unclear how this trend is viewed by clinicians in the field. This study aimed to investigate the opinions of these stakeholders with respect to RWE and its suitability for informing regulatory, reimbursement-related and clinical decisions in oncology.Methods: An online survey was disseminated to clinicians belonging to the network of the European Organisation for Research and Treatment of Cancer between May and July 2021.Results: In total, 557 clinicians across 30 different countries participated in the survey, representing 13 distinct cancer domains. Despite seeing the methodological challenges associated with its interpretation as difficult to overcome, the respondents mostly (75.0%) perceived RWE positively, and believed such evidence could be relatively strong, depending on the designs and data sources of the studies from which it is produced. Few (4.6%) saw a future expansion of its influence on decision-makers as a negative evolution. Furthermore, nearly all (94.0%) participants were open to the idea of sharing anonymized or pseudonymized electronic health data of their patients with external parties for research purposes. Nevertheless, most clinicians (77.0%) still considered randomized controlled trials (RCTs) to be the gold standard for generating clinical evidence in oncology, and a plurality (49.2%) thought that RWE cannot fully address the knowledge gaps that remain after a new antitumor intervention has entered the market. Moreover, a majority of respondents (50.7%) expressed that they relied more heavily on RCT-derived evidence than on RWE for their own decision-making.Conclusion: While cancer clinicians have positive opinions about RWE and want to contribute to its generation, they also continue to hold RCTs in high regard as sources of actionable evidence.

Real-world data in oncology: a questionnaire-based analysis of the academic research landscape examining the policies and experiences of the cancer cooperative groups

“…We have previously investigated how European and Israeli cancer clinicians perceive RWD and RWE by surveying members of the EORTC network. 38 Although this past survey and the present one were not designed with the same objectives in mind, some of their findings align closely with each other. For example, the participants that filled in our earlier questionnaire viewed the methodological complexities of RWD research as more challenging to address than its technical difficulties, a sentiment that the cooperative groups seemed to confirm based on their prior involvement in RWD studies.…”

“…In the current study, responders preferred to characterize RWD as data gathered without influencing the conditions under which they are generated, just like in our previous survey. 38 This discrepancy between different stakeholders’ understanding of RWD again demonstrates the need for formulating a standardized and internationally accepted definition for this concept. Establishing such a uniform definition would likely stimulate and facilitate discussion and collaboration in the area of RWD research.…”

Cytochrome P450 inhibitor/inducer treatment patterns among patients in the United States with advanced ovarian cancer who were prescribed or were eligible for poly(adenosine diphosphate [ADP]-ribose) polymerase inhibitors in the first-line maintenance setting

“…Moreover, DDIs for these PARPis are poorly understood in both clinical and real-world settings. In addition, although observational cohort studies provide the potential to look across the continuum of care and not just data from hospital settings, using claims and EHR data has the potential for misclassification bias due to coding inaccuracies and missingness in diagnosis, procedure, drug codes, and physician notation ( Saesen, 2022 ). Furthermore, there was a high level of missingness in the enriched oncology real-world database, limiting the data available on some clinical characteristics (eg, BRCA mutation status and ECOG performance status), and responses to 1L therapy were not in the scope of the study.…”

Positive correlation between persistence of medical nutrition therapy and overall survival in patients with head and neck cancer

“…Research based on real-world evidence (RWE) has received increasing attention over the past decade and plays a major role in healthcare decisions, including anti-cancer treatments and MNT [19,20]. Provided that the quality of the data is robust and transparent, the results of RWE-based research are valuable complements to randomized controlled trials for evaluating treatment effectiveness.…”