FEBRUARY 2024LIFE SCIENCES REVIEW9IMPACT OF REAL-WORLD EVIDENCE AND DIGITAL INNOVATION IN HEALTHCAREOur CPhI Pharma Awards 2021's winning entry (Winner of Excellence in Pharma: Digital Innovation; Finalist in Excellence in Pharma: Regulatory Procedures and Compliance) recognized a set of projects over a three-year period. RWE and RCTsRCTs are rigorous in assessing a causal effect between treatment options and outcomes. There are several elements their designs: (1) random allocation to intervention groups; (2) patients and trialists should remain unaware of which treatment was given until the study is completed-although such double blind studies are not always feasible or appropriate; (3) all intervention groups are treated identically except for the experimental treatment; (4) patients are normally analyzed within the group to which they were allocated, irrespective of whether they experienced the intended intervention (intention to treat analysis); (5) the analysis is focused on estimating the size of the difference in predefined outcomes between intervention groups. However, despite being the gold standard in terms of evidence hierarchy, a lack of patient diversity among certain groups that are underrepresented is commonplace in RCTs, and FDA Oncology Center of Excellence's Project Equity is timely. RWD can be fragmented siloed and generally not integrated across different data-capture systems via interoperability. According to the United States (US) Food and Drug Administration (FDA), such data can come from a wide variety of sources, such as electronic health records, claims and billing activities, product and disease registries, patient-generated data including in home-use settings, and data gathered from other sources that can inform on health status, such as mobile devices. Bring evidence together systematically and holistically can be valuable. Patient-CentricityTo analyze the uses of medications, especially for patients who are chronically ill, there are three concepts that are important, i.e., adherence, compliance and persistence (Table 1). AnRWE study design generally consists of a patient-specific index event, which can be either a particular diagnosis or therapy via their diagnosis or drug codes, or a combination of both, per patient. A pre-index period, which can be arbitrarily from 90 days to a year, potentially captures the patient's co morbid conditions. Subsequently, a post-index period, which can be arbitrarily from a year or longer, follows up on the patient's medication use, initial fill, refill/refills patterns, non-invasive/invasive procedures, as well as healthcare resource utilization patterns such as hospitalization, as well as emergency department, intensive care unit, and/or outpatient clinic visits. Understanding of adherence is particularly important in the era of COVID-19 since "social determinants of health are critical elements that can impact not just the likelihood of having an NCD or becoming infected with COVID-19, but also access to healthcare, and a patient's adherence and persistence with their treatments."AI, ML and DLThe use of AI, ML and DLis beneficial to patients, for example, for biomarker validation to improve diagnostic accuracy. There are various techniques to optimally combine linear or nonlinear biomarkers to improve diagnostic accuracy, and data science methods, e.g., classification and regression trees and cluster analysis, as well as classical logistic regression, may be applied. In summary, RCTs, RWE and digital innovation can jointly be highly useful to advance medical sciences, understand disease burdens, and optimize treatments precisely, timely and effectively. Beyond detections and diagnoses, RWD of high quality and representativeness may help comprehensively map patient journeys. Such information can help evaluate diagnoses, therapies, and prognoses holistically and comprehensively. Furthermore, beyond single-pill approaches, integrated care and cutting-edge technologies rely on advances in outcomes research and data science. RCTs, RWE and digital innovation can jointly be highly useful to advance medical sciences, understand disease burdens, and optimize treatments precisely, timely and effectively
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