Use of real-world evidence in meta-analyses and cost-effectiveness models

Abstract

Real-world evidence (RWE) provides external validity, supplementing and enhancing the randomized controlled trial data with valuable information on patient behaviors and outcomes, turning efficacy and safety results into real-world effectiveness and risks, but the use of RWE is associated with challenges. The objectives of this communication were to (1) summarize all guidance on how to conduct an RWE meta-analysis (MA) and how to develop an RWE cost-effectiveness model, (2) to describe our experience, challenges faced and solutions identified, (3) to provide recommendations on how to conduct such analyses. No formal guidelines on how to conduct an RWE MA or to develop an RWE costeffectiveness model were identified. Using the context of non-vitamin K antagonist oral anticoagulants in stroke prevention in atrial fibrillation, we conducted an RWE MA, after having identified sources of uncertainty. We then implemented the results in an RWE cost-effectiveness model, defined as a model where all inputs come from RWE, including all parameters related to treatment effect. Based on challenges faced, our first recommendation relates to the necessity of conducting sensitivity analyses, either based on clinical or methodological considerations. Our second recommendation is the need for extensive collaboration with a wide range of experts, during the development of the analyses protocols, the implementation of the analyses and the interpretation of the results. RWE may address a number of gaps related to the treatment effect, and RWE economic evaluations for the treatment effect can provide extremely valuable insights into real-world economic value of interventions. As the increased recognition of the value of RWE could influence health technology assessment decision, and current practices, this communication supports the urgent need of more formal guidelines.

Real-World Data of Anticoagulant Treatment in Non-valvular Atrial Fibrillation

“…Although clinical trials are the gold standard to assess the efficacy and safety of a drug, RWD data may be used to reflect the broader picture of various clinical settings, provide supplementary insight, and identify gaps (10). RWD studies could help evaluate the effectiveness of healthcare interventions (15) and cost-effectiveness studies, helping policy-makers to make decisions on the optimal allocation of resources (20). The present study was conducted with EHRs associated with a public general practice setting that covers 92% coverage of the population living in the area.…”

Clinical and economic analysis of Gastrodin injection for dizziness or vertigo: a retrospective cohort study based on electronic health records in China

“…The data of this study was from real-world, not from controlled clinical trials. Real-world data is different from controlled clinical trial data, real-world evidence provides external validity and can provide evidence for the effectiveness and economic effects [ 52 ]. Real-world evidence can be used to complement data from controlled clinical trials to enable the generalization of clinical findings to a more inclusive and larger population, it can provide instructive information for clinical practice [ 53 ].…”

Effectiveness and Economic Evaluation of Polyene Phosphatidyl Choline in Patients With Liver Diseases Based on Real-World Research

“…The lower ratio indicates the lower cost of adding one therapeutic effect, indicating greater practical significance of the regimen ( Boersma et al, 2010 ). In addition, in order to better understand the uncertainty of this study, sensitivity analysis was used to confirm the stability of economic analysis result; we adjusted the economic evaluation data to a certain extent (medication costs were reduced by 10%; examination costs were increased by 5%; total efficiency decreased by 10%) to see if the results remained the same ( Bowrin et al, 2020 ).…”