Real-world evidence (RWE) and real-world data (RWD) are increasingly influencing regulatory affairs in the biopharmaceutical and healthcare industry. This change has been brought about due to the recognition of the value of real-world data in assessing the safety, efficacy, and effectiveness of therapies beyond traditional clinical trials.
 
[1] Increased Importance of Post-Marketing Surveillance: With RWE, regulatory agencies now have increased access to post-marketing surveillance data. This data is helping in better identification of adverse events and monitoring the safety of products in the market.
 
[2] Regulatory Decision Making: RWE is becoming increasingly significant in regulatory decision-making, including label extensions, post-approval studies requirements, and in some cases, approval decisions themselves. Regulatory bodies such as the FDA and EMA are now considering RWE as part of their evidence package, though with caution and scrutiny.
 
[3] Guidelines and Policies: Regulatory bodies are developing guidelines for the use of RWE in regulatory decision-making. The FDA, for instance, has issued guidance on the use of RWE to support approval of new indications for already approved drugs or to satisfy post-approval study requirements.
 
[4] Innovative Trial Design: The increased use of RWE is promoting the use of innovative clinical trial designs like pragmatic clinical trials (PCTs), which make use of real-world data.
 
[5] Technology and Data Privacy Considerations: With RWE coming largely from electronic health records, insurance claims data, and wearables, regulatory affairs, especially study start-up professionals, will need to grapple with data privacy and cybersecurity issues. There’s an increasing need for regulations that guide the use and protection of such data.
 
[6] International Harmonization: As RWE can be collected globally, there are efforts to harmonize the guidelines across different countries to make multinational studies feasible.
 
[7] Transparency and Trust: Ensuring the validity and reliability of RWE studies is a growing concern. This has led to an increased focus on transparency in data collection, analysis methods, and reporting, all of which regulatory affairs must oversee, especially in the context pf RWE used for regulatory submissions, such as marketing authorisations..
 
Thus, the intersection of RWE and regulatory affairs is evolving and holds potential for improving healthcare decisions and outcomes. Regulatory affairs professionals must adapt to these changes, requiring new skills and competencies in data analysis and understanding of the evolving regulatory landscape for RWE.

Real-World Evidence (RWE) can include data from sources such as electronic health records (EHRs), insurance claims and billing activities, patient registries, patient-generated data, and data gathered from other sources that reflect the patient’s health status. Regulatory authorities, such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), inspect RWE studies to ensure that they’re conducted to the highest standards of scientific integrity, validity, and reliability.
 
Here are some key aspects that are usually inspected in the context of regulatory authority inspections:
 
[1] Data Quality and Integrity: Regulatory authorities review the systems and processes in place to ensure data accuracy, completeness, and consistency. This can include reviewing data collection methods, data management systems, and any algorithms used to process or analyse the data.
 
[2] Methodology: The design and conduct of RWE studies are critically reviewed. This includes the study protocol, statistical analysis plan, and any modifications made during the study. The methodology should be clear, robust, and scientifically sound to generate reliable evidence. Where consideration of a scientific standard or best practice is recommended (e.g., ISPE GPP) and not followed, this should be justified.
 
[3] Regulatory Compliance: While RWE (observational) studies may not fall under the GCP umbrella, ethical conduct, patient privacy, and data protection are still paramount. Compliance with laws like HIPAA in the United States or GDPR in the European Union, is essential, as is compliance with the local laws that govern RWE studies.
 
To prepare for a regulatory authority inspection, consider the following steps:
 
[1] Ensure data quality: Maintain rigorous data management practices. Make sure your data is accurate, complete, and auditable.
 
[2] Document everything: Keep comprehensive records of all activities related to the study. This includes detailed protocols, data collection methods, data handling procedures, and analysis plans. Also, document any changes made during the study and the reasons for those changes.
 
[3] Protect patient privacy: Ensure that your data management systems meet the required standards for protecting patient privacy. Anonymize data whenever possible and follow all relevant data protection laws.
 
[4] Prepare a team: Have a team that’s familiar with the study and prepared to answer questions from inspectors. This team should be knowledgeable about the study design, data management, and analysis.
 
[5] Conduct internal audits: Regular internal audits can help identify potential issues before an inspection. They can also provide opportunities to improve your procedures and systems.
 
Remember, each regulatory authority may have its own specific requirements, so it’s essential to understand and comply with the regulations of the jurisdiction in which you’re operating.