Real-world evidence (RWE) refers to the clinical evidence regarding the usage and potential benefits or risks of a product derived from analysis of real-world data (RWD). These data are routinely collected from a variety of sources, such as electronic health records, claims and billing data, patient registries, and data gathered through personal devices. RWE is increasingly being used in clinical trials and marketing authorisations.
 
The ethical imperative of incorporating RWE into clinical trials intended to support marketing authorizations arises from several factors:
 
[1] Patient-centric Approach: RWE provides a more holistic and accurate view of the patient experience as it considers a wide range of factors such as comorbidities, patient behaviour, social determinants of health, and the full array of interventions. This allows for a patient-centric approach in medical research, which respects the dignity, rights, and welfare of individuals.
 
[2] Generalizability: RWE is collected from diverse patient populations and settings and can therefore inform on how a drug or intervention works in the ‘real world,’ beyond the controlled environment of randomized controlled trials (RCTs). This increases the generalizability of research findings and supports the ethical principle of justice, ensuring that the benefits of research are applicable and beneficial to all groups.
 
[3] Safety and Effectiveness: RWE can provide additional information on a drug’s safety and effectiveness in broader populations and over longer periods, beyond what can be feasibly assessed in RCTs. This can provide early signals of potential harm and help prevent unnecessary patient exposure to harmful interventions.
 
[4] Efficiency: Incorporating RWE can make research more efficient, reducing the time and cost of bringing a new product to market. This is ethically significant as it can accelerate patient access to beneficial new treatments.
 
[5] Informed Decision Making: RWE helps physicians, patients, and policy-makers make informed decisions. Better evidence can improve the quality of care, patient outcomes, and health system efficiency.
 
[6] Inclusivity: Traditional clinical trials often exclude certain patient groups, such as the elderly or those with multiple health conditions. RWE includes data from these often under-represented groups, ensuring that the findings are applicable to them.
 
The importance of RWE lies in its ability to provide an enhanced understanding of a drug’s safety, effectiveness, and value in routine clinical practice. It can capture the wide heterogeneity of patients, healthcare providers, and treatment settings, providing a much-needed complement to traditional RCTs. By integrating RWE into the process of marketing authorizations, regulators can make more informed decisions, ensuring that only safe and effective drugs reach the market, ultimately improving patient health outcomes.