Real World Evidence in Medical Product Development 1st editon by Weili He, Yixin Fang, Hongwei Wang 3031263286 9783031263286

Part I. Real-World Data and Evidence to Accelerate Medical Product Development

The Need for Real-World Evidence in Medical Product Development and Future Directions

Overview of the Current Real-World Evidence Regulatory Landscape

Key Considerations in Forming Research Questions and Conducting Research in Real-World Setting

Part II. Fit-for-Use RWD Assessment and Data Standards

Assessment of Fit-for-Use Real-World Data Sources and Applications

Key Variables Ascertainment and Validation in RW Setting

Data Standards and Platform Interoperability

Privacy-Preserving Record Linkage for Real-World Data

Part III. Causal Inference Framework and Methodologies in RWE Research

Causal Inference with Targeted Learning for Producing and Evaluating Real-World Evidence

Estimand in Real-World Evidence Study: From Frameworks to Application

Clinical Studies Leveraging Real-World Data Using Propensity Score-based Methods

Recent Statistical Development for Comparative Effectiveness Research Beyond Propensity-Score Methods

Innovative Hybrid Designs and Analytical Approaches Leveraging Real-World Data and Clinical Trial Data

Statistical Challenges for Causal Inference Using Time-to-Event Real-World Data

Sensitivity Analyses for Unmeasured Confounding: This Is the Way

Sensitivity Analysis in the Analysis of Real-World Data

Personalized Medicine with Advanced Analytics

Use of Real-World Evidence in Health Technology Assessment Submissions

Examples of Applying Causal-Inference Roadmap to Real-World Studies

Part IV. Application and Case Studies

Applications Using Real-World Evidence to Accelerate Medical Product Development

The Use of Real-World Data to Support the Assessment of the Benefit and Risk of a Medicine to Treat Spinal Muscular Atrophy

Back Matter