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.

Keywords: Europe; cancer; clinicians; oncology; randomized controlled trials; real-world data; real-world evidence; survey.

FIGURE 1

Overview of the number of survey participants per country.

FIGURE 2

Breakdown of the respondents by (A) oncological specialty; (B) experience, expressed in years active as a clinician; (C) type of anticancer treatments they administer to their patients; and (D) type of hospital or institution that employs them. The aggregate numbers reported for (A,C,D) exceed the total number of participants because multiple response options could be selected for these questions.

FIGURE 3

Participants’ views of the evidentiary criteria employed by regulatory authorities in their country or region for allowing new anticancer treatments to enter the market. The percentages shown do not add up to exactly 100% due to rounding.

FIGURE 4

Participants’ level of (dis)agreement with specific statements. The percentages shown for each statement may not add up to exactly 100% due to rounding. The numbers in brackets indicate the absolute number of respondents corresponding with each percentage.

FIGURE 5

Participants’ understanding of what real-world data are, categorized according to the classification system of Makady et al. (2017).

FIGURE 6

Connotation that the term “real-world evidence” had for participants. The numbers in brackets indicate the absolute number of respondents corresponding with each percentage.

FIGURE 7

Participants’ perceptions of the strength of the real-world evidence that can be produced by (A) different study designs, and (B) different sources of real-world data. The percentages shown for each item may not add up to exactly 100% due to rounding. The numbers in brackets indicate the absolute number of respondents corresponding with each percentage.

FIGURE 8

Participants’ perceptions of challenges associated with the collection and use of real-world data. The percentages shown for each challenge may not add up to exactly 100% due to rounding. The numbers in brackets indicate the absolute number of respondents corresponding with each percentage.

FIGURE 9

The role that participants thought real-world evidence should play in the decision-making process of (A) regulators and (B) payers, and (C) the role it currently plays in their own decision-making, as compared with evidence derived from randomized controlled trials. The percentages shown for each item may not add up to exactly 100% due to rounding. The numbers in brackets indicate the absolute number of respondents corresponding with each percentage.

FIGURE 10

Types of studies that participants thought should be performed when regulators grant marketing authorizations to anticancer treatments on the condition that uncertainties regarding their effectiveness are addressed in the post-approval setting. The percentages shown do not add up to exactly 100% due to rounding.

FIGURE 11

Participants’ views on the suitability of real-world data for addressing different types of uncertainties relating to the adoption of new anticancer therapies into clinical practice. The percentages shown for each uncertainty may not add up to exactly 100% due to rounding. The numbers in brackets indicate the absolute number of respondents corresponding with each percentage.

FIGURE 12

Participants’ reported experience with being involved in studies relying on the analysis of real-world data.

FIGURE 13

Participants’ support for sharing anonymized or pseudonymized electronic health data of their patients with external parties for research purposes, if (A) these data would be transferred to a central, internationally accessible repository, or (B) if no such transfer would occur.

FIGURE 14

(A) Participants’ projections of how the role of real-world evidence in the development of anticancer treatments will evolve over time, and (B) their perceptions of a hypothetical growth in its future influence.