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Monday, June 16
 

11:00am EDT

Moving Toward Pragmatism in Clinical Trials
Monday June 16, 2025 11:00am - 12:00pm EDT
TBD
Component Type: Session
Level: Advanced

Pragmatism in clinical trials advances modernization of clinical research. Key stakeholders including experts from the FDA will review advantages and challenges of pragmatism in clinical trials including trials in support of new labeling claims.

Learning Objectives

State the strengths and limitations of pragmatic trials and the reasons for uneven adoption among industry and regulators; Discuss the regulatory framework and FDA initiatives supporting the use of pragmatic elements in clinical trials; Identify how to determine the appropriate context-of-use for successfully deploying pragmatic trials in clinical research.

Chair

Zhanna Jumadilova, MD, MBA

Speaker

Moving Toward Pragmatism in Clinical Trials
Adrian Hernandez, MD, MHS

Moving Toward Pragmatism in Clinical Trials
Jyothis George

Moving Toward Pragmatism in Clinical Trials
Thomas Bognanno


Speakers
TB

Thomas Bognanno

Patient Representative, Alliance Practice, United States
Founder and Partner of Alliance Practice LLC, a firm dedicated to supporting select organizations in the trauma-informed care sector. Before retirement in 2023, Bognanno served as the Chief Executive Officer of Creating Healthier Communities for 17 years. Before that, he served as... Read More →
JG

Jyothis George

Global Medical Vice President, Obesity and Related Conditions, Amgen, United Kingdom
avatar for Adrian Hernandez

Adrian Hernandez

Professor of Medicine; Executive Director, Duke Clinical Research Institute , United States
Adrian Hernandez, MD, MHS, is Executive Director, Duke Clinical Research Institute, and Vice Dean of the Duke University School of Medicine. Dr. Hernandez has research interests in improving cardiovascular health and accelerating clinical evidence through outcomes research, clinical... Read More →
ZJ

Zhanna Jumadilova

Clinical Lead, Pfizer Inc, United States
Dr. Jumadilova is a physician scientist, pharmaceutical executive with extensive industry experience in Clinical Development, Medical Affairs, and HEOR. In her roles as a senior leader, she has led numerous development programs for small molecules and biologics. Her diverse experience... Read More →
Monday June 16, 2025 11:00am - 12:00pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  09: Regulatory, Session

2:15pm EDT

Expanding Innovative Master Protocol Trial Designs to Cell and Gene Therapies for Rare Diseases
Monday June 16, 2025 2:15pm - 3:15pm EDT
TBD
Component Type: Forum
Level: Intermediate

Master Protocols, including basket trials and umbrella trials, enable efficiencies in investigating multiple conditions or multiple treatments, respectively, under a single trial design. Applying Master Protocol trial designs to Cell & Gene therapies (CGTs) can streamline clinical and regulatory processes to speed the delivery of treatments to patients with rare diseases.

Learning Objectives

Identify barriers to and solutions for leveraging Master Protocol clinical trial designs to study Cell & Gene Therapies for rare diseases.

Chair

Nancy Myers, JD

Speaker

Academic Perspective
Kiran Musunuru, MD, PhD, MPH

Researcher Perspective
Jeff Allen, PhD


Speakers
avatar for Jeff Allen

Jeff Allen

President and Chief Executive Officer, Friends of Cancer Research, United States
Jeff Allen, Ph.D. serves as the President and CEO of Friends of Cancer Research (Friends). During the past 25 years, Friends has been instrumental in the creation and implementation of policies ensuring patients receive the best treatments in the fastest and safest way possible. As... Read More →
KM

Kiran Musunuru

Professor of Medicine, University of Pennsylvania, United States
An actively practicing cardiologist and committed teacher, Kiran Musunuru, MD, PhD, MPH, ML, MRA, is Professor of Cardiovascular Medicine, Genetics, and Pediatrics in the Perelman School of Medicine at the University of Pennsylvania. His research focuses on the development of novel... Read More →
avatar for Nancy Myers

Nancy Myers

CEO and Founder, Catalyst Healthcare Consulting, United States
Nancy Bradish Myers, JD, CEO & Founder, Catalyst Healthcare Consulting, is a sought-after leader with deep expertise in health law, regulation and policy. She has led a successful strategic regulatory policy consultancy for 19 years and has been active in the DC policy arena for three... Read More →
Monday June 16, 2025 2:15pm - 3:15pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  02: ClinTrials-Ops, Forum

3:45pm EDT

Integrating Clinical Trials and Clinical Care: The Who, What, When, Where, Why and How
Monday June 16, 2025 3:45pm - 4:45pm EDT
TBD
Component Type: Session
Level: Intermediate

This session will cover the different ways clinical trials can be operationalized in clinical care, the benefits and challenges of the various approaches, and the applicability of good clinical practice requirements to them.

Learning Objectives

Describe key elements of the design and conduct of trials integrated into clinical care; Outline the various operational approaches for such trials, including the benefits and challenges; Discuss the applicability of good clinical practice requirements to various operational approaches.

Speaker

Industry Perspective
Robert DiCicco, PharmD

Site Perspective
Ryan Ferguson

MHRA Perspective
Jason Wakelin-Smith


Speakers
avatar for Robert DiCicco

Robert DiCicco

Vice President, Portfolio Management, TransCelerate Biopharma Inc., United States
Rob DiCicco is the Vice President, Portfolio Management at TransCelerate Biopharma, Inc. where for the last 3 years he has headed up a suite of programs that include Pragmatic Trials along with several digital and data driven transformational projects. He was the Chief Health Officer... Read More →
RF

Ryan Ferguson

Acting Director, Cooperative Studies Program, VA Boston Healthcare System, United States
avatar for Jason Wakelin-Smith

Jason Wakelin-Smith

Expert GCP Inspector and Head of Compliance Expert Circle, Medicines and Healthcare Products Regulatory Agency (MHRA), United Kingdom
Jason Wakelin-Smith joined the MHRA in November 2006 as a GCP Inspector becoming a Senior GCP & GLP Inspector in 2015, a Lead Senior GCP & GLP Inspector in 2017 and the Expert Inspector for GCP in April 2022. Jason has a broad range of experience within the MHRA compliance teams having... Read More →
Monday June 16, 2025 3:45pm - 4:45pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  08: RD-Quality-Compliance, Session |   02: ClinTrials-Ops, Session
 
Tuesday, June 17
 

10:30am EDT

FDA, MHRA, Health Canada Views on Data Flow: The Backbone of Effective Risk Assessments in Innovative Clinical Trial Design and Conduct
Tuesday June 17, 2025 10:30am - 11:30am EDT
TBD
Component Type: Session
Level: Intermediate

Effective data management and risk mitigation are crucial for ensuring data quality, safety, and reliability. This session explores strategies to uphold these standards in cutting-edge trials.

Learning Objectives

Outline regulatory requirements for data quality, traceability, and reliability in trials with decentralized elements, adaptive designs, and RWD; Discuss case studies with complex data flows to highlight to he importance of understanding data flow, conducting risk conducting risk assessments, and applying effective risk mitigation strategies.

Chair

Cheryl Grandinetti, PharmD

Speaker

Industry Perspectives
Michael Torok, PhD

MHRA Perspective
Jason Wakelin-Smith


Speakers
DF

Debbi Fox

Compliance and Enforcement Specialist, Health Canada, Canada
avatar for Cheryl Grandinetti

Cheryl Grandinetti

Associate Director for Clinical Policy, CDER/OC/OSI/DCCE, FDA, United States
Dr. Grandinetti is the Associate Director for Clinical Policy within the Office of Scientific Investigations’ Division of Clinical Compliance Evaluation and provides leadership and subject matter expertise on policy issues related to GCP, human subject protection, and clinical trial... Read More →
avatar for Michael Torok

Michael Torok

Vice President, Global Head of Quality Assurance Programs, Genentech, A Member of the Roche Group, United States
Michael Torok, Ph.D. is the Global Head of Quality Assurance Programs at Genentech/Roche. He is energized and driven by the mission of positioning Quality to be an accelerant in developing new therapeutics, rather than a guardrail or "policemen". To enable this shift, cross-functional... Read More →
avatar for Jason Wakelin-Smith

Jason Wakelin-Smith

Expert GCP Inspector and Head of Compliance Expert Circle, Medicines and Healthcare Products Regulatory Agency (MHRA), United Kingdom
Jason Wakelin-Smith joined the MHRA in November 2006 as a GCP Inspector becoming a Senior GCP & GLP Inspector in 2015, a Lead Senior GCP & GLP Inspector in 2017 and the Expert Inspector for GCP in April 2022. Jason has a broad range of experience within the MHRA compliance teams having... Read More →
Tuesday June 17, 2025 10:30am - 11:30am EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  08: RD-Quality-Compliance, Session

1:45pm EDT

Has Decentralized Trials Crashed, or Are We Just Getting Started? An Inflection Point for Clinical Operations in Oncology
Tuesday June 17, 2025 1:45pm - 2:45pm EDT
TBD
Component Type: Forum
Level: Intermediate

Our industry is still mired in failing to adopt proven technology solutions until it becomes a reg necessity or mainstay of core operations. Industry change agents will present evidence that demonstrates we typically operate with lagging efforts.

Learning Objectives

Identify emergent solutions and innovative business practices that can enhance clinical trial operations; Evaluate the impact of adopting decentralized trials in oncology using the Beat AML Master Trial as a case study; Discuss how risk-taking and cross-sector practices can accelerate clinical trial success and improve patient outcomes.

Chair

Len Rosenberg, PhD, RPh

Speaker

Panelist
Joseph Dustin


Speakers
avatar for Joseph Dustin

Joseph Dustin

Founder and Managing Partner, eClinical Consulting, United States
For almost 20 years, Joe has been a vocal and visible force in the Life Sciences industry specifically in the realm of eClinical Technology starting in the eCOA space, and most recently finishing up 12 year run with Medidata. He has worked with top Pharmaceutical, Biotech and CRO... Read More →
avatar for Joseph Dustin

Joseph Dustin

Founder and Principal, Dauntless Eclinical Strategies, United States
Joe Dustin is a recognized leader in the clinical trials industry with over 20 years of experience driving digital innovation in Life Sciences. Throughout his career, Joe has spearheaded transformative initiatives across pharma and technology companies, notably at, Medidata Solutions... Read More →
avatar for Len Rosenberg

Len Rosenberg

Head of Clinical Operations, Beat AML, a division of The Leukemia and Lymphoma Society, United States
Len Rosenberg is an executive with proven experience in driving operational excellence at all levels within the pharmaceutical, biotechnology, CRO and eClinical sectors - diverse background includes executive management, clinical and regulatory operations, business development and... Read More →
Tuesday June 17, 2025 1:45pm - 2:45pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  02: ClinTrials-Ops, Forum

1:45pm EDT

Statistical Challenges and Innovations in Small-Sized Clinical Trials Using Composite Endpoint
Tuesday June 17, 2025 1:45pm - 2:45pm EDT
TBD
Component Type: Session
Level: Intermediate

In this session, speakers will share their insight with case studies to demonstrate how the challenges have been addressed and introduce new methods for designing appropriate wining criteria for composite endpoint in small-sized clinical trials.

Learning Objectives

Recognize the pros/cons and challenges in designing clinical trials with composite endpoints; Discuss advanced methods and strategies for analyzing composite endpoints, including the impact of winning criteria and analysis methods via win ratio, win proportion and win scores; Apply different win ratio methods by scenarios and interpret results from real case examples of small-size.

Speaker

Utilizing Win Ratio Approaches and Two-Stage Enrichment Designs for Small-Sized Clinical Trials
Jialu Wang, PhD

Application of win statistics in non-malignant hematology
Wenquan Wang, PhD

Weighted Win Score Ratio – A Generalization of Win-Ratio Approach for Composite Endpoints with Continuous Components
Qian Tang, PhD


Speakers
QT

Qian Tang

University of Iowa, United States
Qian Tang is a Ph.D. candidate in Statistics at the University of Iowa, specializing in machine learning, optimization, and statistical computation. Her research focuses on developing efficient algorithms for analyzing high-dimensional data, with applications in statistical learning... Read More →
JW

Jialu Wang

Statistician, Vertex Pharmaceuticals, United States
Jialu is a senior biostatistician at Vertex Pharmaceuticals. She closely works with health economic and research and market access teams for reimbursements and HTA submissions of multiple therapies. She also works on late Phase 3b/4 studies. Her research interests include adaptive... Read More →
avatar for Wenquan Wang

Wenquan Wang

Senior Director, Pfizer, United States
Wenquan got his PhD degree in Biostatistics from the University of Iowa. He was an assistant professor at University of Alabama at Birmingham before joining the pharmaceutical industry. He has over 20 years of experience in applying statistics in translational research and phase I-IV... Read More →
Tuesday June 17, 2025 1:45pm - 2:45pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  11: Statistics-Data Science, Session |   02: ClinTrials-Ops, Session

4:00pm EDT

Patient Engagement in Trial Design and the Evolving Landscape of FDA, EMA and Country Authority Guidance
Tuesday June 17, 2025 4:00pm - 5:00pm EDT
TBD
Component Type: Session
Level: Intermediate

With several regulatory and ethics authority initiatives to encourage patient input to trial design, how can authorities provide aligned review, guidance and expectations towards these activities and how should sponsors communicate patient engagement?

Learning Objectives

Discuss US (FDA) and EU (EUCTR and country ethics committees) initiatives for systematic conduct and documentation of patient input to trial design; Explain learnings from sponsor survey on sponsor submission/advice experience and guidance needs; Identify gaps and opportunities for authority (regulatory and ethics committee) harmonization in expectations and guidance; Describe emerging markets.

Chair

Melissa Pauline Herman, MSc

Speakers
MP

Melissa Pauline Herman

Senior Specialist, Patient Insights, Lundbeck A/S, Denmark
Melissa Herman is a Senior Specialist in the H. Lundbeck A/S Global Patient Insights team, working to systematically integrate patient experience data and engagement across neurology and neuro-rare development programs.
Tuesday June 17, 2025 4:00pm - 5:00pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  09: Regulatory, Session |   02: ClinTrials-Ops, Session

4:00pm EDT

Externally Controlled Trials in Clinical Development: Challenges and Mitigation Strategies
Tuesday June 17, 2025 4:00pm - 5:00pm EDT
TBD
Component Type: Session
Level: Intermediate

Externally controlled clinical trials emerges as a viable alternative to traditional RCTs but come with risks such as confounding and bias. This session will focus on discussing these challenges and how to mitigate them via design and analysis.

Learning Objectives

Identify and describe various sources of bias in externally controlled clinical trials, and illustrate their impact on causal effect estimation; Explain statistical methods developed to estimate causal treatment effect from externally controlled trials; Discuss regulatory perspectives on the use of external controls for drug development and registration.

Chair

Xiang Zhang, PhD

Speaker

Industry Perspective
Ran Duan, PhD

Industry Perspective
Jingyu (Julia) Luan, PhD


Speakers
RD

Ran Duan

Director Biometrics, Vertex Pharmaceuticals, United States
avatar for Jingyu (Julia) Luan

Jingyu (Julia) Luan

Executive Regulatory Science Director, BioPharmaceuticals R&D, AstraZeneca, United States
Dr. Jingyu (Julia) Luan is an Executive Regulatory Science Director in AstraZeneca, overseeing the global regulatory strategy and supporting the research, development and commercialization of CVRM products. She is a core member of CVRM Regulatory Leadership Team. Prior to AZ, she... Read More →
XZ

Xiang Zhang

Head of Medical Affairs and HTA Statistics/Co-lead FORExcellence, CSL Behring, United States
Xiang Zhang is the Head of Medical Affairs and HTA Statistics and a co-lead of the Forum for Observational Research Excellence (FORExcellence) at CSL. He leads a team of statisticians, epidemiologists, and RWE scientists that promotes best practices in RWE generation at CSL. His team... Read More →
Tuesday June 17, 2025 4:00pm - 5:00pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  11: Statistics-Data Science, Session |   02: ClinTrials-Ops, Session
 
Wednesday, June 18
 

10:30am EDT

Regulatory and HTA Considerations When Using External Evidence to Support Single Arm Trials During Global Drug Development
Wednesday June 18, 2025 10:30am - 11:30am EDT
TBD
Component Type: Session
Level: Basic

Single Arm Trials (SATs) are an important tool for drug development. The session while focusing on EMA and FDA guidance documents on SATs, will discuss how external information can support the needs and expectations of regulators and HTA globally.

Learning Objectives

Identify the appropriate settings and considerations for using external evidence in designing single-arm trials; Discuss strategies for advancing global drug development with single-arm trials, addressing differing perspectives among regulators and between regulators and HTA representatives.

Chair

Solange Corriol-Rohou, DrMed, MD, PhD

Speaker

Panelist
François Houyez

Panelist
Anja Schiel, PhD


Speakers
avatar for Solange Corriol-Rohou

Solange Corriol-Rohou

Senior Director, Global Regulatory Affairs & Policy, R&D, Europe, AstraZeneca , France
Pulmonologist and immuno-allergist by training, Solange joined AstraZeneca R&D in 2004 and is currently Sr. Global Policy Director, with responsibilities in the Respiratory/Infection, Oncology and Vaccine/Immune therapies franchises. Over the past 20 years, moving from the French... Read More →
avatar for François Houyez

François Houyez

Treatment Information and Access Director, Health Policy Advisor, European Organisation for Rare Diseases (EURORDIS), France
François is a patient and works at the European Organisation for Rare Diseases as Director of Treatment Information and Access since 2003. He's been involved in EUnetHTA since the first Joint Action back in 2010, and supervises Eurordis policy and advocacy on HTA. François also... Read More →
avatar for Anja Schiel

Anja Schiel

Special Advisor, Lead Methodologist/Statistician; NoMA, Norwegian Medicines Agency (NoMA), Norway
Anja Schiel has studied Biology at the Johannes Gutenberg-University, Mainz, Germany. She received her PhD from the Free University in Amsterdam in 2006 and worked several years as Post-Doc before starting at the Norwegian Medicines Agency (NoMA) in 2012. At NoMA she is working as... Read More →
Wednesday June 18, 2025 10:30am - 11:30am EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  03: Data-Tech, Session |   01: ClinSafety-PV, Session

10:30am EDT

Do Flexible Trial Approaches Impede Data Quality? Perception vs Reality
Wednesday June 18, 2025 10:30am - 11:30am EDT
TBD
Component Type: Forum
Level: Basic

This panel will discuss whether flexible operational approaches like decentralization, embedding trials in clinical practice, and allowing setting adjustments will affect data quality. Real and perceived impediments to data quality will be explored.

Learning Objectives

Identify flexible trial approaches and their benefits and limitations; Assess when flexible trial approaches may impede data quality; Discuss solutions for maintaining data quality.

Chair

Lindsay Kehoe, MS

Speaker

Do Flexible Trial Approaches Impede Data Quality? Perception vs Reality
Catherine Gregor, MA, MBA

Do Flexible Trial Approaches Impede Data Quality? Perception vs Reality
Ken Wiley. Jr.


Speakers
avatar for Cheryl Grandinetti

Cheryl Grandinetti

Associate Director for Clinical Policy, CDER/OC/OSI/DCCE, FDA, United States
Dr. Grandinetti is the Associate Director for Clinical Policy within the Office of Scientific Investigations’ Division of Clinical Compliance Evaluation and provides leadership and subject matter expertise on policy issues related to GCP, human subject protection, and clinical trial... Read More →
avatar for Catherine Gregor

Catherine Gregor

Chief Clinical Trial Officer, Florence Healthcare, United States
Catherine Gregor is a transformational leader and business advisor with close to two decades of experience in clinical research. She currently serves as the Chief Clinical Trial Officer for Florence Healthcare, a software company focused on connecting sponsors and sites worldwide... Read More →
KW

Ken Wiley. Jr.

Section Chief, National Center For Advancing Translational Sciences, United States
avatar for Lindsay Kehoe

Lindsay Kehoe

Senior Project Manager, Lead Emerging Programs, Clinical Trials Transformation Initiative (CTTI), United States
Lindsay Kehoe, M.S., is a senior project manager and lead Emerging Programs at the Clinical Trials Transformation Initiative (CTTI), where she manages the development and implementation of CTTI projects. She has convened teams around disease progression modeling, AI, integrating trials... Read More →
Wednesday June 18, 2025 10:30am - 11:30am EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  08: RD-Quality-Compliance, Forum |   02: ClinTrials-Ops, Forum

10:30am EDT

Clinical Trial Innovation at Scale: Opportunities to Increase Adoption and Acceptance
Wednesday June 18, 2025 10:30am - 11:30am EDT
TBD
Component Type: Forum
Level: Intermediate

This forum will address the implementation of innovative clinical trial designs. Specific examples will be discussed and regulatory efforts to foster innovation will be examined to identify opportunities to increase stakeholder understanding.

Learning Objectives

Identify precedents and opportunities to adopt innovative trial designs in severe, chronic diseases; Describe and assess regulatory efforts to foster clinical trial innovation; Identify mechanisms to enhance public awareness regarding implementation of clinical trial innovation in drug development.

Chair

Samantha Roberts, PhD

Speaker

Industry Reflection
Rasika Kalamegham, PhD

Industry Reflection
Victoria A. Dohnal, RAC


Speakers
avatar for Victoria Dohnal

Victoria Dohnal

Director, Global Regulatory Policy and Intelligence, Johnson & Johnson, United States
Victoria Dohnal is currently Director, Regulatory Policy, North America at Johnson and Johnson (J&J) where she is responsible for driving US regulatory policy work related to rare diseases, patient focused drug development, cell and gene therapies, precision medicine, and PDUFA VII... Read More →
avatar for Rasika Kalamegham

Rasika Kalamegham

Head, US Regulatory Policy, Genentech, A Member of the Roche Group, United States
Rasika Kalamegham, Ph.D, is Head of U.S. Regulatory Policy at Genentech. She oversees all FDA related policy work related to the pipeline and serves as the principal representative to Congressional and advocacy relations for regulatory policy. She plays an active role in many trade... Read More →
avatar for Samantha Roberts

Samantha Roberts

Senior Director, Regulatory Intelligence, AstraZeneca, United States
Samantha Roberts leads Respiratory & Immunology Regulatory Intelligence and Portfolio Policy at AstraZeneca. Previously she was in Genentech’s US Regulatory Policy team where she focused on topics including patient-focused drug development, innovative trials, diversity and inclusion... Read More →
Wednesday June 18, 2025 10:30am - 11:30am EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  09: Regulatory, Forum |   02: ClinTrials-Ops, Forum

4:00pm EDT

Modernizing Evidence in Oncology: Real World Data and Artificial Intelligence in Clinical Drug Development
Wednesday June 18, 2025 4:00pm - 5:00pm EDT
TBD
Component Type: Forum
Level: Intermediate

Regulatory, industry, and academic organizations discuss modern approaches to clinical drug development by describing novel methods and use cases integrating real-world data and AI to generate real-world evidence, with focus on oncology.

Learning Objectives

Discuss global regulatory guidance and frameworks supporting innovative designs using real-world data as well as approaches to inclusion of artificial intelligence (AI); Describe innovative approaches and statistical methods for use of hybrid trial designs and examples of AI informed studies for clinical decision making; Discuss areas of current need and promote community collaboration.

Wednesday June 18, 2025 4:00pm - 5:00pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  11: Statistics-Data Science, Forum

4:00pm EDT

Considerations for Adaptive Design Using Bayesian Methods: An Update on ICH E20
Wednesday June 18, 2025 4:00pm - 5:00pm EDT
TBD
Component Type: Session
Level: Advanced

In this session, we will provide a brief overview of the evolving ICH E20 effort with the goal to provide a transparent and harmonized set of principles for the design, conduct, analysis, and interpretation of adaptive clinical trials.

Learning Objectives

Discuss considerations specific to adaptive design using Bayesian methods; Recognize opportunities and challenges of using Bayesian methods in the regulatory setting.

Chair

Amy Xia, PhD

Speaker

E20 Updates
Amy Xia, PhD

FDA Bayesian updates
Mark Rothmann, PhD

Bayesian Trial Design Case Studies
John Zhong, PhD


Speakers
avatar for Mark Rothmann

Mark Rothmann

Division Director, OTS, OB, CDER, FDA, United States
Dr. Rothmann is the Director of the Division of Biometrics II. He earned his Ph. D. in Statistics at the University of Iowa in 1990. He then spent nine years as a professor at various universities before coming to the FDA in 1999. At the FDA, he has been involved in the review on... Read More →
avatar for Amy Xia

Amy Xia

Vice President, Center for Design and Analysis, Amgen Inc., United States
Amy Xia is Vice President, Center for Design and Analysis at Amgen. Amy has worked on designing, implementing, and analyzing Phase I-IV clinical trials as well as observational studies over the past two decades. Currently, she heads up the Center for Design and Analysis organization... Read More →
avatar for John Zhong

John Zhong

Vice President, Head of Biometrics, REGENXBIO, Inc., United States
Dr. Zhong is the Vice President of Biometrics at REGENXBIO. Prior to REGENXBIO, he was a Group Head at Biogen, accountable for Innovative Analytics, Rare Disease Statistics, and others. He has 20 years of industry experience successfully bringing the needed treatments to patients... Read More →
Wednesday June 18, 2025 4:00pm - 5:00pm EDT
TBD Walter E. Washington Convention Center 801 Allen Y. Lew Place, NW Washington, DC 20001-3614 USA
  11: Statistics-Data Science, Session |   02: ClinTrials-Ops, Session |   09: Regulatory, Session
 
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