Interpretation and Impact of Real-World Clinical Data for the Practicing Clinician

Abstract

Real-world studies have become increasingly important in providing evidence of treatment effectiveness in clinical practice. While randomized clinical trials (RCTs) are the “gold standard” for evaluating the safety and efficacy of new therapeutic agents, necessarily strict inclusion and exclusion criteria mean that trial populations are often not representative of the patient populations encountered in clinical practice. Real-world studies may use information from electronic health and claims databases, which provide large datasets from diverse patient populations, and/or may be observational, collecting prospective or retrospective data over a long period of time. They can therefore provide information on the long-term safety, particularly pertaining to rare events, and effectiveness of drugs in large heterogeneous populations, as well as information on utilization patterns and health and economic outcomes. This review focuses on how evidence from real-world studies can be utilized to complement data from RCTs to gain a more complete picture of the advantages and disadvantages of medications as they are used in practice.Funding: Sanofi US, Inc.

Allergen immunotherapy: what is the added value of real-world evidence from retrospective claims database studies?

“…RWE studies may use information from sources such as electronic hospital records, disease registries, and prescription databases, which provide large datasets from diverse patient populations, or may be observational, and collect prospective or retrospective data over a long period of time. They can therefore provide information on the long-term safety and effectiveness of drugs in large heterogeneous populations, together with data on drug utilization patterns and health economic outcomes [14]. In terms of AIT, real-world studies offer the ability to assess evolving risk-benefit profiles, including the effectiveness of AIT beyond 2 years post-treatment and the long-term preventive effects of AIT on asthma progression and new asthma onset [15].…”

“…Evidence from RWE studies complements data from RCTs and is increasingly recognized by regulatory bodies as a valuable source of information to support decision-making, monitoring of postmarketing safety and life cycle product development, and to assist clinical trial design [14]. Real-world data form a key component of health-care technology assessments conducted by bodies such as the UK National Institute for Health and Care Excellence (NICE) to guide clinical decision-making and are also increasingly utilized by payers and other key stakeholders to inform decisions regarding formulary placement and the allocation of health-care resources [14].…”

“…RWE is emerging as an important means to better understand the utility of treatment interventions in broader, more representative patient populations [14]. Because RWE studies are performed in actual clinical practice settings, they are better able to assess the effectiveness and safety of medications in the way they are used by patients and clinicians in routine practice [14], and by taking into account the relationships between patient characteristics, lifestyle factors, treatment compliance and regimens, comorbidities, quality of life, and clinical outcomes. Therefore, RWE can aid patient selection, guide treatment decisions, and instill confidence in prescribing AIT, as well as improve patient communication.…”

Challenges in the clinical implementation of precision medicine companion diagnostics

“…This review assesses the clinical diagnostic testing landscape supporting PM and the challenges to clinical implementation of CDx tests based on real-world evidence derived from patient data collected during routine clinical health care delivery. The validity of tracking such observational data sets to find associations with health outcomes has been demonstrated and acknowledged in several studies [15][16][17]. The authors review published literature and insights in the 'Pharma Precision Medicine Readiness Report 2019,' a longitudinal analysis of the commercial and organizational readiness of biopharmaceutical companies and other stakeholders within PM regarding CDx testing (available at https://www.diaceu tics.com/pmreport19/) [13], published by Diaceutics, a data analytics and implementation service provider in diagnostic commercialization.…”

A registry-based study evaluating overall survival and treatment duration in Swedish patients with metastatic castration-resistant prostate cancer treated with enzalutamide

“…medical visits, laboratory monitoring and criteria for drug stopping or adjustment) among patients with diverse characteristics, and reflect local and national clinical guidelines [7,8]. Recommendations for the timing of drug initiation are similar, regardless of whether the setting is clinical practice or clinical research [9]; however, treatment duration might differ in the real world, as patients are likely to be older and/or less healthy than those eligible for RCT inclusion [7,10].…”