Real-World Evidence: What Is It and Why Is It so Important In MS?

This program will include a discussion of data that were presented in abstract form. These data should be considered preliminary until published in a peer-reviewed journal.

Introduction

Information Gained From RCTs and Observational Data

Information Gained From RCTs and Observational Data: Limitations

Sources of RWE: Advantages and Limitations

Checklist for High Quality RWE Studies

Summary of Key Points

Sources of RWE: Structured Databases

MSBase Registry

MSBase: Learnings About Effect of Early Treatment on Conversion to SPMS

Potential for Learning About Treatment Optimization

MS Registries and Cohorts: A Sample

Treatment-Related Data Collection in Germany: PANGAEA Observational Study

Need for Widespread Data Collection: Complementary to RCTs

Advantages and Challenges of RCTs and RWE

Propensity Score

Propensity Score Matching

Propensity Score Matching: Controlling for Variables

Propensity Score-Based Methods in Cardiology

Propensity Score-Based Method: Coffee and Mortality

Distribution of Propensity Scores Before and After Matching

MSBase Propensity-Matched Data: Escalation to Natalizumab vs Fingolimod in Active RRMS

Propensity-Matched Comparison of Sick Days: PEARL and PANGAEA Observational Studies

Bias in Observational Studies: Checklist

Smoking and MS Progression: Swedish National MS Registry

Swedish Registry Disability Outcomes: Developing an Age-Related MS Severity Score

Vast Amounts of Data Are Needed

Individualized Treatment and Big-Data RWE: A Combined Approach

Impactful RWE Requires Structured Data Collection

Concluding Remarks

Abbreviations