Old dog with new tricks: An introduction to real-world evidence for pharmacists

Abstract

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Integration of real-world evidence from different data sources in health technology assessment

“…RWE is derived from RWD and characterized by the routine use of health technologies in real life outside of the clinical trial setting [15]. RWD are collected from different sources [2,3,5,11,16] and can be quantitative or qualitative. They contain information pertaining to a patient's medical history, demographics, clinical outcomes, lab data, imaging information, resource use and costs, health behaviours and/or patient experiences [2,4,11].…”

“…RCTs are still the gold standard of evidence and are preferred to assess innovative technologies’ safety, efficacy, and effectiveness before launch. Apparently, despite low external validity because of the lack of generalizability, RCTs have a high level of internal validity due to the often-lower risk of bias [ 2 , 7 , 11 , 16 ]. However, sometimes randomization is unfeasible because of ethical issues and technical challenges (e.g., rare diseases and medical device technologies) [ 9 , 11 , 16 ].…”

“…Despite pragmatic trials, single-arm trials, and indirect comparison evidence as some sources of evidence for HTA, stakeholders look to other sources of evidence like RWE. This type of evidence aims to fill the evidence gaps and inherent shortcomings of RCTs as well as minimize the uncertainty in decision-making processes [ 2 , 5 , 8 , 11 , 12 , 14 , 16 , 20 ].…”

“…The other major challenges include transparency, data quality, security and privacy, and data linking. These challenges and barriers apply to many data sources but with varying levels of importance depending on the type and complexity of data sources that need to be considered [ 1 , 2 , 4 , 9 , 11 , 14 , 19 , 24 , 25 ].…”

“…The potential value of Real-World Evidence (RWE) has garnered a great deal of global attention from a range of stakeholders, such as decision-makers, researchers, practitioners, patients, and manufacturers [1][2][3][4]. In the decision-making sphere for health technologies, regulatory agencies such as the Food and Drug Administration and European Medicines Agency have historically used RWE to evaluate post-marketing safety and are now working towards expanding its use to support the approval process in limited circumstances [5].…”