DIA 2025 Global Annual Meeting

Component Type: Forum
Level: Intermediate

Opportunities and challenges for using RWE from the perspective of drug development, regulatory considerations, health technology assessments, clinician and patients will be discussed, including new collaborations between regulators and HTA.

Learning Objectives

Describe how RWE is being used through the medicinal product lifecycle by showing relevant examples; Evaluate the utility of RWE for benchmarking disease progress and/or to use as an external comparator; Discuss how HTA evaluate RWE and current collaborations with regulators.

Chair

Nancy A Dreyer, PhD, MPH, FISPE

Speaker

RWE for Clinical Decision Support
Richard Gliklich, MD

Opportunities and Challenges in Using RWE from the perspective of Drug Development
Brian Bradbury, DrSc, MA

Component Type: Session
Level: Intermediate

This session will discuss regulatory guidance and frameworks on data quality and feasibility assessments of RWD sources to support RWE study proposals for health authority and HTA engagements. It will include information about how to leverage diverse sources of targeted RWD to support and improve the evolution of new therapies and indications for product safety, outcomes, reduction in label use, and help HC professionals and industry to create a knowledge base for use of RWD.

Learning Objectives

Examine the challenges and value of real-world data (RWD) in regulatory decision-making; Discuss the guidance on data quality and feasibility of RWD for real-world evidence (RWE) studies in health authority interactions; Recognize strategies to integrate RWD into dossier data for regulatory alignment; identify challenges in standardizing data collection and sharing across industry, regulators, and academia for global acceptance.

Chair

Mehmet Burcu, PhD, MS, FISPE

Speaker

Building Transparency and Reproducibility into the Practice of Real World Evidence Research
Shirley Wang, PhD, MSc, FISPE

Facilitating robust evidence: integrating digital health data into multi-modal datasets
Laura Lasiter, 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

The FDA Accelerated Approval Program has been criticized for employing lower regulatory standards than traditional approval, undue delays in withdrawing therapies approved under Accelerated Approval for which studies have not confirmed clinical benefit, and confirmatory trials not being pursued with due diligence . Of specific focus for critics has been withdrawn treatments, which are being used as examples of the program’s ineffectiveness and basis for proposed changes. However, the purpose of the program is to allow acceptable risk in order to address patients with a large unmet need. The small percentage of drugs whose clinical benefit is ultimately not confirmed should not be viewed as a failure of the accelerated approval program. Rather, they represent an expected trade-off in expediting drug development that benefits patients with serious or life-threatening diseases. Withdrawals make up a small percentage of the total number of therapies approved under Accelerated Approval and do not reflect overall management of the program over its 30-year history, particularly improvements seen in the last decade in the time from accelerated approval to conversion. In addition, the reasons for the withdrawals are complex and need to be understood within the context of the overall impact on patient outcomes due to early access enabled by the program.

Learning Objectives

Examine the reasons for product withdrawals after Accelerated Approval and identify the different situations where a withdrawal may occur; Recognize clinical impact including life years gained and uptake after Accelerated Approval of oncology products that were later withdrawn; Describe the benefits and challenges encountered by regulators, industry, and patient organizations when it comes to the use of AAP and withdrawals.

Chair

Bridget Doherty, MPH, MS

Speaker

Industry Perspective
Kevin Bugin, PhD, MS, RAC

Non-Profit Perspective
Jeff Allen, PhD

Component Type: Session
Level: Intermediate

This session will present on recent work to harmonize RWE guidelines globally. Ongoing efforts in international collaboration on ICH M14 and the ICH reflection paper will be highlighted.

Learning Objectives

Explain recent developments in international guidelines on RWE; Discuss challenges to harmonization efforts in RWE; Recognize how to evaluate gaps in RWE harmonization needs.

Chair

Stephanie Choi, PhD

Speaker

Barriers and enablers in international RWE harmonization
Nicole Mahoney, PhD

Comparison of RWE guidelines across regions
Rachele Hendricks-Sturrup, DrSc, MA, MSc

ICH Efforts in International RWE Harmonization
David Moeny, MPH, RPh

Component Type: Forum
Level: Intermediate

This session will engage an expert panel in a discussion on traceability of RWD. Panelists will describe challenges and discuss potential solutions to maintain and document traceability of RWD used in regulatory submissions.

Learning Objectives

Describe the inherent challenges in maintaining and documenting the traceability of Real-World Data (RWD) for regulatory purposes; Discuss potential solutions, including new and existing technologies, to enhance RWD traceability; Evaluate the strengths and weaknesses of various approaches to RWD traceability in a regulatory context.

Chair

James Browning, MPH

Speaker

Healthcare Organization Research Technology Perspective
Christopher Herrick, MBA

Traceability - Tools & Implementation Perspective
Anand Shroff

Epidemiologist/Observational Researcher Perspective
Nancy A Dreyer, PhD, MPH, FISPE

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

Component Type: Session
Level: Advanced

This panel will explore available regulatory guidance for Real-World Data (RWD) quality assessments and how available implementing tools and frameworks may be applied for different types of RWD and use cases to help drug developers meet regulator expectations.

Learning Objectives

Recognize expectations for real-world data (RWD) quality assessments in studies intended for regulatory submissions; Identify available tools and frameworks for implementing RWD quality assessments; Explain ways to incorporate RWD quality assessments into internal workflows using practical approaches.

Chair

Eleanor Caplan, PharmD, PhD

Speaker

Balancing Patient Centricity with Data Quality Requirements and Standards
Jennifer Farmer, MS

Learnings about meeting RWD fitness standards from FDA use cases
Ulka B Campbell, PhD

Can we separate relevance from reliability in data quality assessments? Quality-by-design in secondary use of health data
Pamela Dobay, PhD, MSc

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: Session
Level: Intermediate

As Asia’s post-marketing surveillance evolves with the use of RWD, aligning these changes with US and European frameworks is essential. This session will cover Asia’s regulatory shifts and provide insights for global harmonization efforts.

Learning Objectives

Discuss how the integration of RWD into post-marketing surveillance has improved safety monitoring and formulate strategies for applying these practices within your own organization's regulatory and safety frameworks; Analyze case studies on PMS in Asia to propose actionable approaches for implementing database-driven RMP systems in your organization, enhancing selective safety measures.

Chair

Judy Shin, PhD

Speaker

An Overview of RWD Utilization for Drug Safety Assessment in PMDA
Shinya Watanabe

Case study using Real-World Data on Post-Marketing Safety in Korea
Bonggi Kim, PhD

Global Perspectives on Post-Marketing Safety: Leveraging RWD for Regulatory Alignment and Enhanced Safety Surveilance
Jeff Lange, PhD

Evolving Post-Marketing Safety Management in Korea: Opportunities and Challenges in Integrating RMP
Min-Jung Lim, MPharm, RPh

Component Type: Session
Level: Intermediate

Data privacy has been a long-standing challenge for RWD sharing and evidence generation across geography. This session will discuss privacy-preserving approaches that enable data access, sharing and collaboration in industry, academia and regulatory

Learning Objectives

Identify privacy-preserving approaches for accessing and sharing real-world data (RWD); Discuss use cases leveraging various applications (e.g., federated learning networks, synthetic data) in industry, academia and regulatory for RWE generation; Examine regulatory perspectives, methodological challenges and identify future development needs.

Chair

Echo Wang, DrPH, MPH

Speaker

Real-world applications of distributed data analytic solutions in real-world data networks
Darren Toh, DrSc, FISPE

Privacy-Preserving Solutions for RWD Access and Collaboration: A Life Science Industry Perspective on Challenges & Opportunities
Mehmet Burcu, PhD, MS, FISPE