Regulatory Science Symposium: Patient-Centered Drug Development and Real World Evidence/Data Session 5: Real World Evidence and Real World Data (2019)

Device Development
Drug Development
Regulatory Science
Clinical Trials
Klaus Cepeda, MD, MS, FCP

Director, Clinical Pharmacology and Quantitative Medicine at Critical Path Institute; Adjunct Professor of Clinical Pharmacy, USC School of Pharmacy


  1. Part I: Definitions
    1. Real World Data (RWD)
    2. Real World Evidence (RWE)
    3. Applications of RWE based on RWD
  2. Part II: Case Studies (drug development tools)
    1. Why do Drug Development Programs Fail?
    2. Clinical Trial Simulations
    3. Clinical Trial Simulator for Alzheimer’s disease
    4. Comprehensive Data Integration
    5. Model Endpoints/Covariates
    6. Implications for Application
    7. Extend Validation
    8. A Successful Pathway for Regulatory Endorsement of Quantitative Drug Development Tools
    9. Case Study 1
    10. Case Study 2
  3. Part III: Case Studies (medical devices)
    1. Medical Devices
    2. Aortic Valve Replacement
    3. What RWD/RWE was used?
  4. Part IV: Case Studies (drugs)
    1. Physiologically Based Pharmacokinetic/dynamic Modelling (PBPK)
    2. Conclusions


Accompanying text created by Khyati Ashtekar | Graduate Student, Regulatory Science, USC School of Pharmacy |  

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