Real-World Evidence for Pharma

Subtitle: A Practical Guide to Study Design, Analysis, and Execution.

Real-world evidence (RWE) has moved from the margins of pharmaceutical research to its centre. Regulators, payers, and clinicians increasingly expect observational studies to complement randomized clinical trials throughout the lifecycle of a medicine — from development and market access to post-marketing surveillance.

Real-World Evidence for Pharma is a practical guide to the design, analysis, and execution of RWE studies, written primarily for professionals working in the pharmaceutical industry and contract research organizations.

Drawing on decades of hands-on experience in drug development, registry-based research, pharmacoepidemiology, statistical analysis, and market access, the book addresses the practical challenges researchers face when working with real-world data.

Topics include:

• Healthcare data sources, including national registries, claims databases, electronic health records, and disease-specific registries
• Data quality, linkage, and the challenges of working with real healthcare data
• Statistical methods including propensity scores, inverse probability weighting, time-dependent covariates, causal inference, and power considerations
• Ethics, governance, GDPR, and HIPAA
• Post-authorization safety studies, comparative effectiveness studies, and external control arms
• Publication strategy and communicating results for regulatory and clinical impact
• Common pitfalls in the design, analysis, and reporting of RWE studies
• A comprehensive quality-control checklist for RWE projects

The book includes practical case studies from respiratory medicine, market access, pharmacoepidemiology, vaccine safety, and multinational PASS research, including the PATHOS study and a multinational PASS study using Nordic laboratory data.

The book is intended for statisticians, epidemiologists, medical affairs professionals, health economists, regulatory scientists, and market access specialists. No prior specialist knowledge of observational research is assumed. Statistical concepts are explained from first principles, making the book accessible to non-statisticians while remaining technically rigorous for experienced researchers.

Rather than presenting RWE as a perfect science, the book recognizes that data are incomplete, confounding is unavoidable, and statistical significance does not always imply clinical importance. Its aim is to provide a disciplined and practical approach to producing evidence that regulators, payers, clinicians, and pharmaceutical decision-makers can trust.