Real world data: an opportunity to supplement existing evidence for the use of long-established medicines in health care decision making

Abstract

Evidence from medication use in the real world setting can help to extrapolate and/or augment data obtained in randomized controlled trials and establishes a broad picture of a medication’s place in everyday clinical practice. By supplementing and complementing safety and efficacy data obtained in a narrowly defined (and often optimized) patient population in the clinical trial setting, real world evidence (RWE) may provide stakeholders with valuable information about the safety and effectiveness of a medication in large, heterogeneous populations. RWE is emerging as a credible information source; however, there is scope for enhancements to real world data (RWD) sources by understanding their complexities and applying the most appropriate analytical tools in order to extract relevant information. In addition to providing information for clinicians, RWE has the potential to meet the burden of evidence for regulatory considerations and may be used in approval of new indications for medications. Further understanding of RWD collection and analysis is needed if RWE is to achieve its full potential.

Efficacy and Safety of Ixekizumab Through 5 Years in Moderate-to-Severe Psoriasis: Long-Term Results from the UNCOVER-1 and UNCOVER-2 Phase-3 Randomized Controlled Trials

“…Currently, to our knowledge, there are no head-to-head clinical trials between ixekizumab and secukinumab; however, head-to-head analyses of real-world data sources have shown better drug survival over a long time period for ixekizumab [48,49]. Continued study of real-world data sets is needed to further elucidate the comparative effectiveness of these medicines in more clinically relevant populations that are not restricted to the enrollment criteria of clinical trials [50,51]. It is not known why there are such differences in effectiveness between these two molecules that target the same cytokine; however, the higher binding affinity of ixekizumab for IL-17A [49,52] and a more consistent dosing regimen over time may contribute to these differences.…”

Assessing the efficacy‐effectiveness gap for cancer therapies: A comparison of overall survival and toxicity between clinical trial and population‐based, real‐world data for contemporary parenteral cancer therapeutics

“…Furthermore, RWE may be able to support data in circumstances in which the generation of RCT data either is insufficient or unlikely, such as in the case of rare disease . Interest in the use of RWE to support health policy decisions, such as for drug reimbursement decisions, also has been generated . In particular, there is interest in the use of this evidence to provide ongoing re‐evaluation of technologies to ensure that effectiveness and value are preserved after regulatory approval and adoption …”

“…62 Interest in the use of RWE to support health policy decisions, such as for drug reimbursement decisions, also has been generated. 64 In particular, there is interest in the use of this evidence to provide ongoing re-evaluation of technologies to ensure that effectiveness and value are preserved after regulatory approval and adoption. 65 However, RWE is not without its limitations.…”

Health Care and Pharmaceuticals 2020: Key Factors, Expected Trends and Way Ahead

“…All drug launches happen after randomized controlled trials (RCTs) have establish efficacy and safety in an appropriately sized population. Real World Evidence (RWE) is derived from the analysis of data collected from healthcare settings, outside the ambit of the strict patient selection criteria of RCTs, and helps to understand observations and events in different kinds 15 The US FDA aims to publish draft guidance for the use of RWE by October 2021, In Asia, (including India), there is growing awareness among regulatory authorities to structure the inclusion of real world evidence into assessing long term efficacy and safety of approved drugs in different patient groups and its benefit in clinical and economical decision making. Though RWE generation is focused mainly on post launch assessment of drugs, soon earlier stages of the clinical drug development pipeline may also start to use RWE to support critical decisions.…”