Real World Evidence In Europe - The Results Of An Expert Survey

Gill, JL; Albanell, Joan; Avouac, Bernard; Dank, M.; Duncombe, Robert; Fink-Wagner, Antje; Hutton, J; Jahnz-Różyk, Karina; Kossler, Ingrid; Podrazilova, Katerina; Schramm, W; Spandonaro, Federico; Carneiro, António Vaz; Wartenberg, Markus; Kanavos, Panos

doi:10.1016/j.jval.2017.08.1554

Cited by 2 publications

(1 citation statement)

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“…They did not consider RWE to be more or less useful to either stakeholder group, at least in comparison to evidence produced by RCTs. The experts who filled in the survey of Gill et al (2017) also doubted that the results of RWD studies would ever be weighted similarly to those of RCTs in evaluations of product dossiers. However, they were largely of the opinion that RWE was more likely to be applied for supporting reimbursement-related decisions than for influencing the conditions under which marketing authorizations are granted.…”

Section: Discussionmentioning

confidence: 99%

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

et al. 2022

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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.

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Section: Discussionmentioning

confidence: 99%

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

et al. 2022

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Assessing the Value of Integrated Evidence Approaches in Drug Development

DiMasi,

Olson,

Smith

et al. 2024

Preprint

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Background The use of Integrated Evidence Packages (IEPs) by the pharmaceutical industry has expanded in recent years with the aim of optimizing healthcare and patient outcomes. The evidence base of IEPs goes beyond traditional randomized controlled trials (RCTs) to provide holistic evidence suitable for all stakeholders. However, this approach to drug development is not systematically adopted by all sponsors because of perceived uncertainty in its investment value. Methods We introduce the concept of value drivers to which we apply an expected net present value (eNPV) model of the cash flows for drug development and commercialization. The approach is outlined for two, typical, hypothetical lifecycle management IEPs. The measure of IEP value is defined as the increment in eNPV that occurs when IE programs are employed in comparison to when they are not. Results We found substantial value for IEPs. One example incorporated a plan to conduct an observational study that could be used as a basis for approval in lieu of a classical phase II trial for a supplemental indication. In the other example, increased adoption of the new treatment leads to a highly positive increment in eNPV based on the critical evidence generated in a phase IIIb study. Conclusions Use of value drivers and eNPV-based value models when planning for IEPs can provide objective guidance for project teams. The value can be estimated through formal economic analysis that considers planned timelines, R&D costs, estimates of the likelihood of regulatory approval, patient access and clinical adoption if development is successful.

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Real World Evidence In Europe - The Results Of An Expert Survey

Cited by 2 publications

References 0 publications

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

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

Assessing the Value of Integrated Evidence Approaches in Drug Development

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