DIA 2025 Global Annual Meeting

Component Type: Tutorial
CE: ACPE 2.75 Application UAN: 0286-0000-25-502-L04-P; CME 2.75; IACET 2.75; RN 2.75

Pre-registration required and is an additional fee.

Already registered? Log in to DIA's My Account My Events.

This Short Course will be offered virtually – Join from anywhere!In this short course, participants will gain hands-on experience in applying Risk-Based Quality Management (RBQM) principles, grounded in the Quality by Design (QbD) framework, to real-world clinical trial scenarios. Working in small groups, participants will analyze a three-page real-life study synopsis, using guidance from ICH E6(R2/R3), to identify critical data, critical processes, and potential risks. Following an instructor-led introduction to key concepts like risk scoring and mitigation strategies, participants will identify and prioritize risks, create actionable risk statements, develop mitigation plans, define Key Risk Indicators (KRIs), and establish Quality Tolerance Limits (QTLs).With feedback from expert instructors, participants will refine their critical thinking and learn how to distinguish key data and processes in clinical trials. This approach emphasizes practical relevance, equipping attendees with tools to apply RBQM and QbD principles to their own protocols immediately. Through collaborative group work and actionable insights, attendees will leave with a clear understanding of how to embed ICH E6(R3)-aligned QbD strategies into their risk management practices.

Receive $50 off your Global Annual Meeting total purchase by registering for two or more Short Courses. Purchases must be made at the same time to receive the discount. Discounts will be reflected on the last page of the cart.
Receive $50 off your Global Annual Meeting total purchase by registering for two or more Short Courses. Purchases must be made at the same time to receive the discount. Discounts will be reflected on the last page of the cart.

Who should attend?

Professionals involved in or interested in clinical research, particularly risk managers, project managers, study managers, clinical data managers, statisticians, lead monitors, auditors, and those involved in risk-based quality management and risk-based monitoring activities.

Learning Objectives

• Identify critical data and critical processes, determine the risks and define a risk statement
• Evaluate the risks regarding likelihood, impact and detectability, and define suitable mitigation actions, KRIs and QTLs
• Evaluate compliance with ICH E6 R2/R3 and increase the potential for study success

Component Type: Tutorial
CE: ACPE 2.75 Application UAN: 0286-0000-25-503-L04-P; CME 2.75; IACET 2.75; PDU 2.75 PMI 2166LKA26P; RN 2.75

Pre-registration required and is an additional fee. *Please note: Short Courses are stand-alone events. Registration for the main conference, forum, etc. are not mandatory. Already registered? Log in to DIA's My Account My Events. This Short Course will be offered virtually – Join from anywhere!This short course provides a focused, in-depth exploration of how established project management frameworks, tools, and best practices can be applied specifically to the drug development process. Participants will begin by examining a range of methodologies—such as Agile, Waterfall, and hybrid approaches—to understand which strategies work best for managing the multifaceted challenges of clinical trials. Through guided instruction, attendees will learn how to adapt these methodologies to meet stringent regulatory requirements, handle complex demands, and maintain stringent quality standards. In addition to foundational methodologies, this course delves into practical tools and templates that streamline project planning, risk management, and communication. These tools help teams navigate the intricate timelines associated with bringing a drug to market, enabling participants to anticipate and mitigate risks before they become obstacles. By learning to identify common bottlenecks and implement effective contingency plans, attendees can keep projects on schedule and budget, ensuring that new therapies reach patients faster. A central component of the course focuses on enhancing cross-functional collaboration. Participants will learn strategies to improve communication among scientific, clinical, regulatory, and operational teams, ensuring that everyone shares a common understanding of project goals and responsibilities. This improved alignment not only accelerates decision-making but also fosters a more cohesive and productive working environment. To reinforce these concepts, the course provides real-world examples and case studies to give participants hands-on experience in applying project management principles, evaluating risk, adjusting timelines, and coordinating team efforts in real-world scenarios. In addition, advanced tools and techniques are introduced, equipping learners with cutting-edge resources that support informed decision-making and efficiency throughout the drug development lifecycle. By the end of this program, participants will have gained a practical, customizable toolkit they can immediately apply in their daily work. Participants will emerge with greater confidence in managing complex drug development and clinical research projects, ultimately improving trial outcomes and accelerating the delivery of innovative therapies to the patients who need them. Receive $50 off your Global Annual Meeting total purchase by registering for two or more Short Courses. Purchases must be made at the same time to receive the discount. Discounts will be reflected on the last page of the cart. Registered attendees for this virtual Short Course will receive access to the course recording for 2 full months post-course! This allows you to remain flexible with your schedule and not worry if you need to step out momentarily. Have a conflict with the dates of the course, but are interested in the content? Register anyway and you will receive access to the recording!

Who should attend?

Professionals working in or interested in drug development who seek to improve their project management knowledge and skillset. Roles that would benefit from this course include project managers, clinical research associates, data managers, regulatory specialists, and clinical operations staff, amongst others, who aim to streamline processes and deliver projects on time and on budget to accelerate the development of new therapies.

Learning Objectives

• Identify key project management methodologies and tools to improve efficiency in drug development
• Discuss drug development timelines by learning strategies to manage risks, streamline timelines, and overcome bottlenecks
• Apply Cross-Functional Collaboration to ensure smoother project execution and faster delivery of therapies

Component Type: Tutorial
CE: ACPE 2.75 Application UAN: 0286-0000-25-504-L04-P; CME 2.75; IACET 2.75; RN 2.75

Pre-registration required and is an additional fee. *Please note: Short Courses are stand-alone events. Registration for the main conference, forum, etc. are not mandatory. Already registered? Log in to DIA's My Account My Events. This Short Course will be offered virtually – Join from anywhere! Cell and Gene Therapies (CGTs) are a rapidly expanding area in the pharmaceutical sector, offering the potential to address serious unmet medical needs in many indications. Yet some of the conceptual frameworks in this field differ dramatically from those of traditional pharmaceutical medicines, specifically with regard to CMC, Non-clinical and Clinical requirements and Health Authority expectations. This short course will help those who have pharmaceutical sector experience but are newcomers to the CGT field ‘get into the right frame of mind’ to think about CGT development. Participants will be introduced to requirements for each of the major disciplines for regulatory submissions: CMC, Non-clinical and Clinical. The course will also explain the rationale behind the CMC approach to CGT manufacture and basic CMC regulatory expectations for CGT products, including process and product characterization, comparability and process validation. Biological mechanisms for CGTs will also be discussed, with a focus on how mechanistic understanding can inform the design of in vitro and animal studies to provide non-clinical evidence of safety and efficacy. Key principles for clinical development of CGTs will be presented, with an emphasis on clinical study design elements that are critical for Health Authority evaluation of benefit-risk. Case studies of recent FDA approvals will be showcased to highlight key clinical considerations and changes in the regulatory environment for CGTs. The course features ex-Health Authority Regulators presenters who have a combined 20 years of experience at the FDA and EMA. Receive $50 off your Global Annual Meeting total purchase by registering for two or more Short Courses. Purchases must be made at the same time to receive the discount. Discounts will be reflected on the last page of the cart. Registered attendees for this virtual Short Course will receive access to the course recording for 2 full months post-course! This allows you to remain flexible with your schedule and not worry if you need to step out momentarily. Have a conflict with the dates of the course, but are interested in the content? Register anyway and you will receive access to the recording!

Who should attend?

Professionals involved in or interested in gaining an understanding of the basics of cell and gene therapy development, including regulatory personnel, clinical operations personnel, researchers and investigators, medical writers, and commercial specialists.

Learning Objectives

• Demonstrate why cell and gene therapies (CGT) require a different and very specialized regulatory approach compared to small molecules and biologics
• Develop knowledge of CGT-specific considerations and requirements for CMC, Non-Clinical, and Clinical development
• Analyze important regulatory considerations for CGT development programs

Component Type: Tutorial
CE: ACPE 6.50 Application UAN: 0286-0000-25-509-L04-P; CME 6.50; RN 6.50

Pre-registration required and is an additional fee. *Please note: Short Courses are stand-alone events. Registration for the main conference, forum, etc. are not mandatory. Already registered? Log in to DIA's My Account My Events. Patient experience data, through patient-reported outcomes (PROs) and other clinical outcome assessments (COAs) are an increasingly important component of regulatory drug submissions and health technology assessments (HTAs). Instructors will take participants on an in-depth evaluation of the key topics important in successful ePRO implementation, including:

• Selecting, adapting and developing patient-reported outcome measures to meet protocol objectives
• The case for ePRO – when to select an electronic approach, and when paper might suffice
• ePRO implementation best practices to ensure good measurement science
• Migration validity considerations and evidentiary requirements
• Language, translation and licensing considerations
• Implementation steps including vendor selection and user acceptance testing (UAT) best practices
• Modality choices – web vs app, and bring-your-own-device (BYOD) considerations
• Hot topics such as data change processes, mixing modes, special populations, etc.
• Q&A – discussion of participants' own hot topics.

Receive $50 off your Global Annual Meeting total purchase by registering for two or more Short Courses. Purchases must be made at the same time to receive the discount. Discounts will be reflected on the last page of the cart.

Who should attend?

Professionals who have experience in clinical trials and patient engagement and wish to develop their skills on how PRO measurement is best implemented in clinical drug development programs.

Learning Objectives

• Demonstrate when to select electronic capture, when to use web vs app, and when to enable patients to use their own devices (BYOD)
• Apply industry best practices to ePRO implementation, translations and other processes such as PRO data changes
• Describe the implementation steps, possible bottlenecks and mitigations, and user acceptance testing best practices

Component Type: Session
Level: Basic

Digital health technologies are advancing drug development, aligning with FDA's PDUFA VII commitments. This session explores their impact, featuring perspectives from academia, industry, and FDA.

Learning Objectives

Describe the evolving application and impact of digital health technologies in clinical research; Identify best practices for integrating digital health technologies with traditional clinical outcome assessments; Discuss the challenges and regulatory considerations associated with the use of digital health technologies.

Chair

Tae Hyun Jung, PhD

Speaker

Academic Perspective
Vadim Zipunnikov, PhD

Wearable Based Digital Measures for Tracking Early-Stage Parkinson’s Disease Progression Show Potential for use as Digital Endpoints in PD Trials
Sooyoon Shin, PhD

Advancing Clinical Development: The Role of Digital Technology Across the Lifecycle
Tarik Yardibi, PhD

The Moment of Change
Tae Hyun Jung, PhD

Component Type: Forum
Level: Intermediate

This forum will discuss Good Clinical Practice (GCP) inspection methods, collaboration and training among regulators and their inspectors as they respond to the evolving clinical trial regulatory landscape such as the implementation of ICH E6 R3 and novel trial methodologies

Learning Objectives

Describe the reasons why regulators may inspect clinical trials in different ways; Compare the various inspection approaches used by different regulators; Explain how regulators are harmonizing inspection approaches and collaborating to share information, resources, and training; Analyze how regulators are preparing to implement ICH E6 R3 and its implications for inspections and inspector training.

Chair

Mandy Kaur Budwal-Jagait, MSc

Speaker

Why do GCP Inspectors Do Things Differently and How can Approaches be Both Harmonized and Optimized
Kavita C. Dada, PharmD, RAC

Why do GCP Inspectors Do Things Differently and How can Approaches be Both Harmonized and Optimized
Reza Salehzadeh-Asl

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

Component Type: Session
Level: Intermediate

Go beyond platitudes and qualitative advice on planning, deploying, and measuring the success of your recruitment campaign. Enjoy this Master Class in Recruitment Campaign Management from some of the industry's best minds

Learning Objectives

Discuss how to build an evidence based forecast for centralized patient recruitment campaigns; Describe ways to optimize channel mixes, vendor management, performance models, technology selection; Identify methods to measure and optimize campaign performance and ROI.

Chair

Chris Venezia

Speaker

Case Study Presentation
Tom Julian

Case Study Presentation
Kelly Johnston McKee, MS

Component Type: Forum
Level: Advanced

This session will delve into the historical and ethical context for distinguishing between clinical care and research and how the blurring poses a risk for engagement with patients. Experts in ethics and health literacy will discuss potential pitfall

Learning Objectives

Recognize the ethical and historical context for distinguishing between clinical care and research; Describe the regulatory and ethical challenges that are exposed when sponsors and investigators push patients to clinical research; Discuss how language and health literacy have the potential to impact an individual’s decision to participate in research.

Chair

Karla Childers, MS

Speaker

Bioethics perspective
Lindsay McNair, MD, MPH, MS

Patient and health literacy perspective
Catina O'Leary, PhD

IRB perspective
David Borasky, MPH

Component Type: Session
Level: Intermediate

This forum will present: Health Authority (HA) expectations related to QbD, RBQM, and issue management. Specific examples of how study teams use technology to overcome challenges in achieving QbD and effective RBQM and issue management practices.

Learning Objectives

Discuss Health Authority (HA) expectations when designing and executing clinical studies, specifically for using risk proportionate approaches in Quality by Design (QbD), Risk Based Quality Management (RBQM) and issue management practices; Describe real life examples of technologies that facilitate the achievement of HA expectations for QbD, RBQM, and issue management.

Chair

Michael Torok, PhD

Speaker

Industry Perspective
Michael Torok, PhD

Industry Perspective
Kevin Richards

Component Type: Session
Level: Intermediate

In this session, leaders from Health Canada will provide an update on regulatory priorities for Canada. The audience will be invited to submit questions of general interest.

Learning Objectives

Describe Health Canada’s regulatory initiatives and strategic priorities; Identify opportunities for engagement with the Canadian regulator.

Chair

Alysha Croker, PhD

Component Type: Session
Level: Intermediate

In this session, speakers from industry, academia, and the US Food and Drug Administration (FDA) will discuss innovative use cases, experiences, and challenges of implementing linkage of traditional clinical trial data to RWD.

Learning Objectives

Discuss use cases of trial linkage to RWD and the benefits for patients, decision makers and sponsors; Explain requirements and best practices in implementing trial linkage to RWD; Describe current scientific and operational challenges and strategies to overcome them; Identify areas in need of further development to support the design and execution of global hybrid pragmatic studies.

Chair

Mehdi Najafzadeh, PhD

Speaker

Challenges and lessons in Linkage of RWD to Trials: An Academic Perspective
Elisabetta Patorno, DrPH, MD

Pragmatic approaches in hybrid designs: Industry perspective
Mehmet Burcu, PhD, MS, FISPE

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

Component Type: Forum
Level: Advanced

This forum will provide information regarding new requirements for Diversity Action Plans. A panel of experts will describe details of these requirements, implementation challenges for rare genetic diseases, and strategies to address these challenges.

Learning Objectives

Describe how regulatory requirements for Diversity Action Plans might apply to orphan drug development; Plan regulatory strategy for inclusive and representative rare disease drug development; Identify challenges regarding compliance to Diversity Action Plan requirements in rare disease settings.

Chair

Mark Tobolowsky, JD

Component Type: Workshop
Level: Intermediate

Imagine a future where AI measures patient burden from provided inputs. In this interactive workshop, participants will design input logic for AI using mock data and protocols, then present their rationale to foster collaborative learning.

Learning Objectives

Define and categorize key factors affecting patient burden in clinical trials; Analyze patient-related data to design effective AI inputs; Evaluate and justify the role of human insight in enhancing AI-driven patient burden assessments for patient-centric trial outcomes.

Chair

Maya Zlatanova

Component Type: Session
Level: Intermediate

This session will present multiple case studies illustrating the successful implementation of AI-driven RBQM solutions in real-world clinical trials. Attendees will gain insight into the practical applications of AI technologies.

Learning Objectives

Recognize the key benefits and challenges of applying AI technologies in RBQM; Identify insights into real-world examples of AI's impact on clinical trial risk management; Discuss strategies for integrating AI into existing RBQM frameworks to improve decision-making and compliance; Describe how AI enhances collaboration across clinical trial stakeholders and supports more proactive risk-based strategies.

Chair

Artem Andrianov, PhD, MBA

Component Type: Forum
Level: Intermediate

In a rapidly evolving clinical trial landscape, breaking down financial barriers is essential to foster participant enrollment, improve retention, and ensure the success of trials.

Learning Objectives

Identify key findings from DIA’s Participant Compensation Landscape Analysis and discuss their implications to promote diversity and support clinical trial outcomes; Recognize participant-centric strategies to address financial barriers in clinical research, enhancing diversity in trial participation; Discuss collaborative approaches to further understand participant compensation practices.

Chair

Carie Pierce, MS

Speaker

Moderator
Donna Libretti Cooke, JD

Component Type: Session
Level: Advanced

This session will explore how Intelligent Automation (IA) and AI are transforming R&D and Regulatory Affairs by automating document-heavy processes and driving data-driven decisions. Through real-world use cases presented by industry sponsors, attendees will learn how IA and AI are being applied to improve efficiencies, reduce timelines, and optimize regulatory outcomes.

Learning Objectives

Discuss AI applications in drug discovery, clinical trials, regulatory, manufacturing, and pharmacovigilance; Explain practical use cases of AI in Regulatory Operations; Discuss advancing fit-for-purpose, risk-based frameworks for AI and global regulatory alignment.

Chair

Venkatraman Balasubramanian, PhD, MBA

Speaker

AI: Accelerating Clinical Trials Through Smarter Site Selection
Kevin Bugin, PhD, MS, RAC

Harnessing AI and Automation in Regulatory:  Insights from the Recent DIA RIM Intelligent Automation Survey
Cary Smithson, MBA

Industry Perspective
Cedric Berger, PhD, MBA

AI-Driven Drug Development: Transforming Life Sciences with Data and Innovation
Sridevi Nagarajan, PhD, MS, MSc

Component Type: Workshop
Level: Intermediate

Digitized protocols can support more efficient workflows and help clinical operations teams accelerate trials. This interactive session will discuss the benefits of protocol digitization along with strategies and opportunities for adoption.

Learning Objectives

Learn how digital protocols can reduce the time and effort needed to manage trials from end-to-end, ultimately accelerating trials and bringing drugs to market faster. Discover how digital protocols are already driving connections and efficiencies at research sites. Understand how industry peers are addressing protocol digitization, including challenges, opportunities, and learnings.

Chair

Denise Meade, MBA

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

Component Type: Session
Level: Intermediate

This session will review the 2024 revision of the Declaration of Helsinki, one of the foundational codes of clinical research ethics, and the rationale and practical implications of the changes made in the current version.

Learning Objectives

Discuss the history of the Declaration of Helsinki and its relevance and importance for the healthcare industry; Recognize the changes made in the 2024 version and how they will impact the conduct of biopharma clinical trials; Discuss multi-stakeholder efforts that are working towards operationalizing ethical principles in the DOH and where more collaboration and alignment is needed.

Chair

Melissa Heidelberg, MS

Speaker

Speaker
Karla Childers, MS

Speaker
Ann Meeker-O'Connell, MS

Component Type: Forum
Level: Intermediate

This session examines the relationship between protocol complexity, participant burden, and enrollment challenges, alongside the development and impact of participant feedback questionnaires. Patient advisors and attendees will interpret results and discuss improvements in trial performance.

Learning Objectives

Discuss patient participation burden based on protocol design characteristics and requirements; Identify design elements driving elevated levels of burden and impacting clinical trial performance; Recognize how to manipulate results into practical improvements for clinical research projects; State the value of participant feedback and compose rationale for inclusion of feedback in future studies; Describe strategies to reduce and address clinical trial and portfolio-wide participation burden.

Chair

Lani Hashimoto

Speaker

Addressing Participation Burden in Clinical Trials: Primary Causes, Outcomes and Mitigation Strategies and Practices
Kenneth Getz, MBA

Participant Feedback Forum: Active Interpretation of Signals by Patient Advisors and Attendees
Lani Hashimoto

Component Type: Session
Level: Intermediate

Through use cases and maturity models, this session will focus on strategies and barriers to integrating RBQM in clinical trials.

Learning Objectives

Interpret key changes in ICH E6(R3) regarding RBQM and data governance; Implement key principles, components, and change management strategies for RBQM; Assess and benchmark organizational adoption of RBQM components.

Chair

Madeleine Whitehead

Speaker

Navigating the Future of Clinical Trials: Embracing ICH E6(R3) with Risk-Based Quality Management
Nicole Stansbury

New and Expanded Assessment of Risk-Based Quality Manangemnt Adoption: Assessing Implementation Progress and Impactased
Abigail Dirks, MS

Component Type: Session
Level: Intermediate

In this forum, speakers from the National Medical Products Administration (NMPA) and the Center for Drug Evaluation (CDE) will present and discuss the latest information on regulation, regulatory science, and drug review and approval. Speakers will also discuss recent development in clinical research in China. Attendees will have an opportunity to discuss the issues related to drug regulation with NMPA officials.

Learning Objectives

Describe the latest information on the National Medical Products Administration’s activities in drug regulation; Describe the recent update on the progress in drug review, approval and clinical research in China; Identify challenges in drug development and regulation in China.

Chair

Ling Su, PhD

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

Component Type: Forum
Level: Intermediate

This panel will discuss the importance of end-to-end support systems that connect trial management, data collection, and patient engagement. This panel will discuss the importance of end-to-end support systems that connect trial management, data collection, and patient engagement. The session will address the challenges sites face in adopting new technologies, from budget constraints to staff resistance, and the challenge of integrating automation into existing workflows.

Learning Objectives

Explain how automation and integration reduce site burden and improve trial efficiency; Identify compliance challenges in implementing automated systems; Discuss best practices for overcoming barriers to technology adoption.

Chair

Denise N Bronner, PhD

Speaker

Strategic Implementation of Automation
Amber Hill

Overcoming Resistance & Driving Adoption
Kimberly Tableman, MSc

Seamless Data Integration & Compliance
Cal Collins

Component Type: Forum
Level: Advanced

We present trends and insights from an analysis of participant payment language of more than 7,500 consent forms reviewed by a large, independent IRB and discuss the ethical implications of insufficient compensation for trial participants.

Learning Objectives

Explain trends and insights in clinical trial participant payments based on 7500 clinical trial ICFs collected and analyzed between 2019 and 2024; Examine the language used to describe payments and how that impacts participants; Discuss ethical issues and assumptions about IRB approval of participant compensation for research.

Chair

Kelly Fitzgerald, PhD

Speaker

Panelist
Kelly Fitzgerald, PhD

Panelist
Donna Libretti Cooke, JD

Component Type: Forum
Level: Intermediate

This session will focus on evaluating key global regulatory challenges in combined clinical trials and harmonization of clinical practices to support medical research innovation.

Learning Objectives

Evaluate the challenges of conducting clinical studies in the US and Europe within a fragmented regulatory landscape; Describe how to analyze specific clinical trial challenges at the intersection of IVDR and CTR; Explain key solutions proposed by COMBINE project recommendations and outlining forward-looking strategies for successful implementation.

Chair

Adriana Racolta, PhD, RAC

Speaker

Pharma Industry Approaches to Harmonize Conduct of Trials Under Fragmented Global Regulatory Frameworks
Lauren Tobe, JD

Diagnostic Industry Perspective and Experience: Innovative Regulatory Approaches for Implementing Diagnostic Testing in Clinical Development of Medicines
Jennifer Tucker, MA

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

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

Component Type: Forum
Level: Intermediate

This session will share healthcare professionals' (HCP) perceptions about participating in clinical research as non-investigators. We will address the challenges and opportunities for HCP participation, aligned to the FDA guidance on DCTs as well as Point of Care Trials.

Learning Objectives

Analyze healthcare professionals' (HCPs) perceptions, both positive and negative, about participating in clinical research; Evaluate which routine care trial assessments HCPs might conduct as part of trials and assess study fit; Identify key strategies to engage HCPs in trial participation without requiring them to become investigators; Discuss resources to assist teams in navigating regulatory documentation.

Chair

Jane Myles, MSc

Speaker

Regulator Perspective
Timil Patel, MD

Industry Perspective
Shelly Barnes

Component Type: Session
Level: Intermediate

Poor study management, misconduct, and data falsification threaten regulatory decisions on product safety and efficacy. This session will cover regulatory guidelines and case studies of clinical trial electronic systems, risk assessment strategies.

Learning Objectives

Describe regulatory expectations for electronic systems in clinical trials, including electronic health records (EHRs); Examine criteria for evaluating whether these systems are suitable for trials. Discuss methods for identifying and mitigating fraud and misconduct risks in these systems; State case studies of fraud and mismanagement to emphasize the need for thorough risk assessments.

Chair

Junko Sato, PhD

Speaker

How to Combat Fraud?
Kentaro Ichinose

MHRA Perspective
Jason Wakelin-Smith

Component Type: Session
Level: Intermediate

Partnerships are crucial to overcoming the challenges clinical trials face in Africa. We will present the current landscape, provide key pointers on navigating it, and share case studies demonstrating the key importance of involving Africa in trials.

Learning Objectives

Recognize the importance of including Africa in clinical trials; Identify challenges of conducting clinical trials in Africa; Apply knowledge to successfully conduct global clinical trials in Africa.

Chair

Kwaku Marfo

Component Type: Forum
Level: Intermediate

This highlights the challenges of using real-world data (RWD) for patient referral in clinical trials, focusing on issues like data variability, lack of investigator training, and insufficient incentives. It proposes strategic solutions to overcome these barriers.

Learning Objectives

Identify innovative strategies for patient recruitment using RWD referrals that challenge existing practices; Propose new methods for integrating naïve sites into the clinical trial process to expand participation; Evaluate the potential impact of broader patient inclusion on clinical trial outcomes.

Chair

Sanghita Bhattacharya, PhD

Speaker

Patient Referral: Is It Hope or Hype?
Joan Chambers

Patient Referral: Is It Hope or Hype?
Barbara Isquith Arone, MS

Patient Referral: Is It Hope or Hype?
Karen Correa, PhD

Patient Referral: Is It Hope or Hype?
Irfan A. Khan, MD

Component Type: Forum
Level: Advanced

The session will describe informed consent in research participants with cognitive decline, discussing behavioral cues and consent/assent requirements for ethical practices when participants lose the ability to provide informed consent during a study

Learning Objectives

Recognize the ethical and operational challenges of engaging participants with cognitive impairment; Describe ethical options for retaining participants with cognitive impairment in clinical trials; Review best practices for informed consent/ caregiver consent/participant assent when the ability to give consent is lost during the course of a trial

Chair

Karla Childers, MS

Speaker

Sharing an IRB Perspective
Linda Reuter, MS

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.

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

Component Type: Session
Level: Intermediate

Hear directly from the regulators and industry about the challenges and opportunities in applying the ICH E17 guidelines. Some cases based on ICH E17 guidelines will be presented in the panel. Panelists will be asked to make comments for each case regarding points to consider in planning a study or evaluating results.

Learning Objectives

Explain how ICH E17 guidelines are being implemented in the conduct of multiregional clinical trials; Evaluate the limitations, gaps, and opportunities for applying the guideline globally.

Chair

Yoshiaki Uyama, PhD, MS, RPh

Speaker

Panelist
Adriane Alves de Oliveira, MSc

Panelist
Hideki Hiraoka

Panelist
William Wang, PhD

Component Type: Session
Level: Intermediate

Japan's regulatory authorities have enacted various measures to strengthen drug R&D and accelerate clinical development, particularly for regenerative medicine, pediatric, and orphan drugs, offering great prospects for biopharma/ biotech companies.

Learning Objectives

Discuss PMDA's efforts to address Japan's drug lag and promote an accelerated clinical development environment; Describe the trial landscape and analyze the challenges in developing Regenerative Medicine, Orphan, and Pediatric drugs in Japan; Develop strategies for Japan to engage in multi-regional trials, considering the unique aspects of conducting clinical studies in the country.

Chair

Edward C. Ian, MBA

Speaker

Industry Perspective
Sumati Nambiar, DrMed, MPH

Panelist
Teppei KIUCHI

Component Type: Session
Level: Intermediate

Industry leaders explore how biopharma is leveraging data foundations and technological advancements, including AI/ML, to improve access and representation in clinical trials, moving from DEI planning to tangible progress in clinical research.

Learning Objectives

Identify key areas where tech and analytics facilitate health equity advancement, focusing on achievable, tech-enabled outcomes; Illustrate concrete use cases where tech and data can support equitable clinical research, such as community outreach, recruitment, and measurement against diversity action plans; Discuss non-technical success factors needed to wholly support data-driven tech

Chair

Katrina Rice, MS

Component Type: Session
Level: Intermediate

This session will be based on a recent effort that examined lung cancer studies to determine how and whether exclusion criteria could hinder the participation of racial minority patients. The panel will feature experts from sponsors, research sites, and academia to discuss challenges and areas of opportunities for re-thinking how to make lung cancer protocols more inclusive, and will include perspectives from the clinical operations, regulatory, research sites, and academia.

Learning Objectives

Identify areas within a study protocol that can adversely exclude marginalized patient populations; Identify organizational challenges that may hinder redesign efforts, and the implications these challenges can have on the clinical operations and regulatory front; Discuss approaches, strategies, and lessons-learned to help simplify protocol design.

Chair

Jennifer Kim, PhD

Speaker

Perspectives from the Clinical Research Sites: Challenges Enrolling Racial Minority Participants
Julieta Argüelles, DrMed

Sponsor Perspective: Overcoming Re-Design Efforts of Study Protocols
Kim Ribeiro, MS

Application of Large Language Models (LLM) to Examine Lung Cancer Protocol Exclusion Criteria
Abigail Dirks, MS

Sponsor Perspective: Implications of Protocol Complexity on Regulatory Oncology
Laura Huggins

Component Type: Forum
Level: Advanced

Join us for an engaging discussion on an ongoing DIA study on enhancing the integration of tolerability patient experience data into immune-oncology clinical trials. This session will explore the study’s efforts to address regulatory requirements for the collection of core tolerability patient experience data and the need for a comprehensive, multidimensional approach to assessing tolerability in cancer treatments. With a multistakeholder focus, the study aims to define tolerability more holistically by incorporating treatment-related factors, convenience, and patient beliefs. Learn about the study’s progress, next steps, and its potential to align with regulatory needs while addressing real-world patient concerns.

Learning Objectives

Recognize the significance of advancing patient-centric tolerability measurement in immune-oncology trials to enhance patient outcomes; List insights into the ongoing study and future steps aimed at addressing the real-world needs of patients through a multistakeholder framework.

Chair

Maria Paula Bautista Acelas, MSc

Speaker

Moderator
Rohini Sen, PhD, MS

Unlocking Insights: Progress and Key Findings from the Tolerability Study
Ryan Murphy, PhD

Panelist
Catherine Coulouvrat, DrMed

Panelist
Lia Ridout

Component Type: Forum
Level: Intermediate

This forum on obesity drug development helps the audience understand the present landscape, participate in discussions on the prevention and treatment of comorbidities, and assess various strategies in enhancing clinical trial efficiency.

Learning Objectives

Discuss the present landscape, opportunities, and challenges of obesity drug development; Identify the prevention and treatment of comorbidities associated with obesity; Assess various considerations and strategies in enhancing the efficiency of obesity drug development.

Chair

Jingyu (Julia) Luan, PhD

Speaker

Panelist
Maria Vassileva, PhD

Panelist
John Sharretts

Panelist
Brad Jordan, PhD

Component Type: Forum
Level: Intermediate

This annual town hall provides a forum for senior FDA officials to reflect on key trends and discuss opportunities in rare disease drug development and review. Officials answer moderated and audience burning questions facing the work that they do.

Learning Objectives

Describe new FDA programs and initiatives intended to help facilitate development of drugs for rare diseases; Evaluate opportunities for sponsors and patient stakeholders to engage with regulators; Identify current trends in challenges and opportunities facing orphan drug development.

Chair

James Valentine, JD, MHS

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

Component Type: Forum
Level: Intermediate

A cross-functional panel of experts will role-play a risk review meeting discussion to identify, assess, categorize, and prioritize risks of a mock protocol decentralized clinical trial (DCT) and outline potential mitigation activities.

Learning Objectives

Outline why risk identification is a fundamental consideration when planning a Decentralized/Hybrid Clinical trial; Define roles and responsibilities for key stakeholders to identify risks for Decentralized/Hybrid Clinical Trial; Identify who are the key stakeholders for a decentralized/Hybrid Clinical trial; Develop fit-for-purpose Oversight Plans.

Chair

Paula Horowitz

Speaker

Quality Risk Management for a Decentralized/Hybrid Clinical Trial: A Role Play by Representatives of Key Stakeholders
GINA Lisette Williamson, BSN, MBA

Quality Risk Management for a Decentralized/Hybrid Clinical Trial: A Role Play by Representatives of Key Stakeholders
Maryrose Petrizzo, MS

Quality Risk Management for a Decentralized/Hybrid Clinical Trial: A Role Play by Representatives of Key Stakeholders
Terry Katz, MS

Component Type: Session
Level: Intermediate

Alternative methods to a frequentist approach, such as Bayesian, may be superior in certain situations; particularly, when analyzing trial data to support approvals in small populations. Regulators are increasingly considering novel methods.

Learning Objectives

Examine global considerations for leveraging Bayesian approaches in pivotal trials to support marketing authorization; State and debate the situations and circumstances where the Bayesian approach may be the most appropriate method of analysis; Discuss health authority considerations that could facilitate the use of alternative analytical methods beyond the frequentist approach; Identify ways to bridge existing gaps in applying Bayesian methods in clinical research.

Chair

Philip Hougaard, DrSc, PhD

Component Type: Forum
Level: Intermediate

The FDA Town Hall is the forum to hear from FDA leadership about Center priorities. The audience will be invited to submit questions of general interest.

Learning Objectives

Discuss FDA Center priorities.

Chair

Amy Bertha