June 9, 2023, 9am -11 am (Japan time)
Lecture: 60 minutes; Q&A: 30 minutes
JAPhMed Real World Analytics Webinar:
Fee: Free
Title: The role of statistics in HTA/RWE application

Weili He and Hongwei Wang

Medical Affairs & Health Technology Assessment Statistics, AbbVie

Abstract:

Evidence-based decision-making is essential for drug development and a continuum across all stages.
This covers the internal go/no-go decision of early pipeline, the regulatory and reimbursement decisionby health authorities, and life-cycle management for value demonstration. This robust evidencegeneration requires multidisciplinary collaboration and statistics plays a central role in fit-for-purpose
study design and analytic framework selection, assessment of robustness and results interpretation.
While randomized clinical trials remain the gold standard to establish efficacy and safety profile ofmedical products, its strict protocol specification, high cost, ethical consideration and lack of venerability
to more heterogeneous patient population in routine clinical practice calls for real-world studies. In thistalk, we will introduce the opportunities and challenges of leveraging real-world evidence (RWE) for
more effective drug development, and highlight the importance of applying statistical principals andcausal inference methodologies in such research endeavor. Specifically, in preparation for the regulatory
and reimbursement decisions, clinical trials with external control using historical data, real-world studies
quantifying natural history of disease under standard of care, network meta-analysis synthesizing direct
and indirect evidence, and populate scenarios for economic modeling are some examples. After initial
regulatory approval, real-world studies to support label expansion, fulfill regulatory commitments,generate treatment usage pattern and comparative effectiveness are important components of theholistic evidence planning. Statisticians with in-depth understanding of real-world data sources,
methodology to account for the inherent confounding and bias, advanced analytics for predictivemodeling and complex data will be your important research partners.

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[Speakers]
Weili HeDr. Weili He has over 25 years of experience working in the biopharmaceutical industry. She is currently
a Distinguished Research Fellow and head of Medical Affairs and Health Technology Assessment
statistics at AbbVie. She has a PhD in Biostatistics. Weili’s areas of expertise span across clinical trials,
real-world studies and evidence generations, statistical methodologies in clinical trials, observational
research, innovative adaptive designs, and benefit-risk assessment. She is the lead or co-author of
more than 60 peer-reviewed publications in statistics or medical journals and lead editor of two books
on adaptive design and benefit-risk assessment, respectively. She is the co-founder and co-chair of theAmerican Statistical Association (ASA) Biopharmaceutical Section (BIOP) Real-world Evidence ScientificWorking Group from 2018 to 2022. She is also the Fonder and Co-chair of a newly formed ASA BIOP HTA
Scientific Working Group. Weili is the BIOP Chair-Elect, Chair, and Past Chair from 2020-2022. Sheserves as an Associate Editor of Statistics in Biopharmaceutical Research since 2014, and is an elected
Fellow of ASA since 2018.

Hongwei Wang
Dr. Hongwei Wang is currently a Director of Medical Affairs & Health Technology Assessment Statisticsand Research Fellow at AbbVie. He has a PhD in Statistics from Rutgers University and close to 20 yearsof industry experience in evidence planning and generation. His research interests include designing andanalyzing real-world studies, network meta-analysis and advanced analytics. Before joining AbbVie inJun 2017, he worked at Merck and Sanofi.

[Chair / Discussant]Toshifumi Sugitani
Dr. Toshifumi Sugitani is currently a Director of RWE at Syneos Health Japan. He has a PhD in
Biostatistics. Toshi’s area of expertise span across statistical methodologies in confirmatory clinical trial
designs (e.g., Graphical approaches, Adaptive designs), dose-response studies (e.g, MCP-Mod),observational studies, real-world studies and evidence generation using large healthcare databases inJapan (e.g, MDV, JMDC, C-CAT), patient-preference studies, and marketing science for sales data inpharma industry in Japan (e.g., time-series analysis using state-space modelling). He is the lead or coauthor of more than 20 peer-reviewed publications in statistics or medical journals and the co-author ofa book chapter of “Multiple comparison” in 2nd edition of International Encyclopedia of Statistical
Science (in press). He received a Research Encouragement Award from the Biometric Society of Japan in2020.

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