Real-world evidence (RWE) is the clinical evidence about the usage and potential benefits or risks of a product derived from the analysis of real-world data (RWD). RWD and RWE are playing an increasing role in health care decisions. They can be used to support regulatory decision-making, clinical trial designs, guideline development, and healthcare policy-making.
 
Understanding the intent of the use of RWE in the design and conduct of an RWE study is critical for several reasons:
 
[1] Regulatory Requirements: The regulatory standards for evidence used to support different types of decisions can vary substantially. For instance, the data standards required by the FDA for a new drug approval (which often rely on randomized clinical trials) are much more stringent than the standards for post-marketing surveillance or comparative effectiveness research. Understanding the end use of the evidence helps in designing a study that meets the appropriate regulatory standards and complies with the relevant requirements.
 
[2] Data Integrity and Quality: The quality of RWD can vary widely, depending on the source. If the evidence is being used to support high-stakes decisions (like regulatory approval), the data used must be of very high integrity and quality. For instance, data from electronic health records or insurance claims may be sufficient for some types of observational studies, but they might not be adequate for regulatory decisions without additional validation.
 
[3] Study Design: The intent of the study impacts the choice of study design. For instance, randomized controlled trials are considered the gold standard for demonstrating cause-effect relationships and are often required for regulatory approval. However, if the goal is to understand how a treatment works in routine clinical practice, an observational study using RWD might be more appropriate.
 
[4] Data Analysis: The intent of the RWE study also determines the methods used to analyze the data. For example, methods to control for confounding factors, which can bias the results, might be more critical in studies used for regulatory decision-making than in those used for exploratory analyses.
 
[5] Ethical Considerations: Depending on the intent, different ethical considerations might apply. For instance, if the RWE study is designed to inform clinical decision-making, it’s crucial to ensure that patient privacy is protected, informed consent is obtained (when required), and results are communicated transparently.
 
In summary, understanding the intended use of RWE is crucial in the design and conduct of an RWE study because it impacts everything from the regulatory requirements to the quality and integrity of data used, to the choice of study design and data analysis methods, to the ethical considerations. These considerations can substantially influence the validity, generalizability, and acceptability of the study’s results.