1st Edition

Real-World Evidence in a Patient-Centric Digital Era

Edited By Kelly H. Zou, Lobna A. Salem, Amrit Ray Copyright 2022
    208 Pages 17 B/W Illustrations
    by Chapman & Hall

    208 Pages 17 B/W Illustrations
    by Chapman & Hall

    208 Pages 17 B/W Illustrations
    by Chapman & Hall

    Real-world evidence is defined as evidence generated from real-world data outside randomized controlled trials. As scientific discoveries and methodologies continue to advance, real-world data and their companion technologies offer powerful new tools for evidence generation. Real-World Evidence in a Patient-Centric Digital Era provides perspectives, examples, and insights on the innovative application of real-world evidence to meet patient needs and improve healthcare, with a focus on the pharmaceutical industry.

    This book presents an overview of key analytical issues and best practices. Special attention is paid to the development, methodologies, and other salient features of the statistical and data science techniques that are customarily used to generate real-world evidence. It provides a review of key topics and emerging trends in cutting-edge data science and health innovation.


    • Provides an overview of statistical and analytic methodologies in real-world evidence to generate insights on healthcare, with a special focus on the pharmaceutical industry
    • Examines timely topics of high relevance to industry such as bioethical considerations, regulatory standards, and compliance requirements
    • Highlights emerging and current trends, and provides guidelines for best practices
    • Illustrates methods through examples and use-case studies to demonstrate impact
    • Provides guidance on software choices and digital applications for successful analytics

    Real-World Evidence in a Patient-Centric Digital Era will be a vital reference for medical researchers, health technology innovators, data scientists, epidemiologists, population health analysts, health economists, outcomes researchers, policymakers, and analysts in the healthcare industry.

    Preface: Real World Evidence and Digital Innovation to Combat Noncommunicable Diseases
    Kelly H. Zou, Lobna A. Salem, Joseph P. Cook, Anurita Majumdar and Amrit Ray

    1. Real World Evidence Generation
    Joseph S. Imperato, Joseph P. Cook, Diana Morgenstern, Kim Gilchrist, Tarek A. Hassan, Jorge Saenz and Danute Ducinskiene

    2. Applications of RWE for Regulatory Uses
    Eleanor E. Panico, Corinne S. Pillai, Ewa Filipowska and Kelly H. Zou

    3. Patient Data Privacy, Protected Health Information and Ethics of Real-World Evidence
    Corinne S. Pillai, Eleanor E. Panico, Kelly H. Zou and Ewa Filipowska

    4. Real-World Data, Big Data and Artificial Intelligence: Recent Development and Emerging Trends in the European Union
    Kelly H. Zou

    5. Patient Centricity and Precision Medicine
    Diana Morgenstern, Mina B. Riad, Claudia Zavala and Amrit Ray

    6. Health Information Technology
    Joseph P. Cook, Gabriel Jipa, Claudia Zavata and Lobna A. Salem

    7. Digital Health Technologies and Innovations
    Kelly H. Zou, Mina B. Riad, Shaantanu Donde, Joan van der Horn and Tarek A. Hassan

    8. Economic Analysis and Outcome Assessment
    Jean-Pascal Roussy and Kelly H. Zou

    9. Partnerships and Collaborations
    Salman Rizvi and Urooj A. Siddiqui

    10. Global Perspective: China Big Data Collaboration to Improve Patient Care
    Zhi Xia Xie, Jim Z. Li, Yvonne Huang, Olive Jin, Wei Yu and Kelly H. Zou

    11. The Future of Patient-Centric Data-Driven Healthcare
    Kelly H. Zou, Lobna A. Salem and Amrit Ray


    Kelly H. Zou, Ph.D., PStat® is Head of Global Medical Analytics and Real-world Evidence, Viatris. She is an elected Fellow of the American Statistical Association and an Accredited Professional Statistician. Previously at Pfizer Inc, she was Vice President and Head of Medical Analytics & Insights; Senior Director of Real-world Evidence, Group Lead of Methods & Algorithms and Analytic Science Lead; Senior Director of Statistics. She was Associate Professor of Radiology at Harvard Medical School, as well as Director of Biostatistics at its affiliated teaching hospitals. She was Associate Director of Rates at Barclays Capital. Dr. Zou is the Vice Chair of the Methods and Data Council of AcademyHealth and Methodologies Working Group, Digital Medicine Society. She has published extensive on both clinical and methodological topics and over 160 peer-reviewed publications. She has served on the editorial board of "Significance," "Statistics in Medicine," "Academic Radiology" and "Radiology." She was the theme editor of "Mathematical and Statistical Methods for Diagnoses and Therapies" in "Philosophical Transactions of the Royal Society A." She has authored "Statistical Evaluation of Diagnostic Performance: Topics in ROC Analysis," "Patient-Reported Outcomes: Measurement, Implementation and Interpretation," and "Statistical Topics in Health Economics and Outcomes Research," published by Chapman and Hall/CRC, Taylor & Francis. She also authored a book chapter in "Leadership and Women in Statistics."

    Lobna Salem, MD, MBA is Head of Medical Affairs, Developed Markets, Japan, Asian and New Zealand at Viatris. Previously, she was Chief Medical Office, Developed Markets, Upjohn, a Division of Pfizer Inc. She has more than 25 years of experience in bio pharmaceutical industry working across different cultures with experience spanning global, region, and country roles. In addition, Dr. Salem has held different cross-functional roles in medical affairs, clinical development, regulatory interactions and commercial functions. She has had experience working in Asia, Emerging Markets, China, Europe and the US. Lobna has been recognized for driving innovation across Research and Development in Legacy Upjohn, a Division of Pfizer. She is a Clinical Pathologist/Hematologist by training; she received her Medical degree from Cairo University, Egypt, her MBA from Edinburgh business school UK and MSc from Hibernia College Ireland.

    Amrit Ray, MD, MBA is a globally-experienced industry leader, physician researcher, and biopharmaceuticals expert. Dr. Ray is Managing Partner at Principled Impact, LLC. His passion is advancing medical breakthroughs and championing healthcare access for patients. He has over 20 years of healthcare experience and has held three C-level roles of increasing responsibility at major companies, leading up to 3,500 professionals in 90 countries. Previously, Dr. Ray was Global President, Head of R&D and Medical, and Executive Leadership Team member at Pfizer Upjohn where he oversaw all aspects of research, Phases I-IV development, regulatory, safety, and medical affairs worldwide. He served as the company’s most senior decision-maker and spokesperson on patient matters. Before joining Pfizer, he was the global Chief Medical Officer for pharmaceuticals at Johnson & Johnson. He earned Immunology and Medical degrees from Edinburgh Medical School, and an MBA from Dartmouth College’s Tuck School of Business. Following antibody bench research at Sir Joseph Lister Labs, obesity clinical research at the Mayo Clinic, and clinical training at Edinburgh Royal Infirmary, he began his career serving as a hospital doctor delivering patient care in the UK's National Health Service. In industry, Dr. Ray has championed the development and launch of several new medicines including in Oncology, Immunology, Neuroscience, Cardio-Metabolism, and other areas. Dr. Ray has wide experience as an independent board director and private equity senior advisor. He serves as an Executive in Residence in bioethics at Columbia University, a Visiting Professor in the Faculty of Medical Sciences, Newcastle University, UK, and a board director at the EveryLife Foundation for rare diseases.