Real-world data and Randomized Controlled Trials
Randomized controlled trials (RCT) are the gold standard used by researchers to explore and market new drugs and interventions. Nevertheless, establishing efficacy or if the treatment works in ideal conditions is not enough (Dang & Vallish, 2016). With the increasing advancements in medicine and technology, patients, providers, and sponsors opt for robust information about the effectiveness of any new treatment or if it benefits people in real-world settings (Singal et al., 2014).
To answer the demands for real-world evidence, more and more health care providers and regulatory bodies have started to integrate real-world data (RWD) in research and practice. Real-world data can generate medical insights from additional sources, such as electronic health records, medical surveys, and administrative claims, instead of randomized controlled trials.
Real-world data: Definition
Real-world data is defined as data obtained from a heterogeneous population in real-world settings, with sources varying from administrative claims to health surveys. Although health care providers worldwide recognize the importance of real-world data, we should note that there’s no consensus about its definition. Interestingly, in a recent study, which included 53 documents and 20 interviews, 38 definitions of real-world data were identified (Makady et al., 2017). While most of the definitions categorized real-world data as information obtained from non-randomized controlled trials, many experts were unable to provide a clear institutional definition.
Therefore, to clarify the concept of real-world data, previous definitions given by major initiatives and healthcare bodies can be utilized and modified. According to the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), real-world data is “Data used for decision-making that are not collected in conventional RCTs.” The Association of the British Pharmaceutical Industry (ABPI) states, “For the purposes of this guidance, “RWD” will refer to data obtained by any non-interventional methodology that describe what is happening in normal clinical practice.” On the other hand, the official definition given by RAND goes, “RWD” is an umbrella term for different types of health care data that are not collected in conventional RCTs. RWD in the health care sector come from various sources and include patient data, data from clinicians, hospital data, data from payers, and social data.” Last but not least, the Innovative Medicines Initiative (IMI)-GetReal defines real-world data as “An umbrella term for data regarding the effects of health interventions (e.g., benefit, risk, and resource use) that are not collected in the context of conventional RCTs. Instead, RWD is collected both prospectively and retrospectively from observations of routine clinical practice. Data collected include, but are not limited to, clinical and economic outcomes, patient-reported outcomes, and health-related quality of life. RWD can be obtained from many sources including patient registries, electronic medical records, and observational studies.” (Makady et al., 2017).