ISPOR Europe 2024: Navigating the conference program – focus on real-world data and real-world evidence

With the European conference for ISPOR—The Professional Society for Health Economics and Outcomes Research, ISPOR Europe 2024, just around the corner, we highlight some of the key sessions of covering real-world data (RWD) and real-world evidence (RWE).

Selected sessions

The increasing interest in RWE for informing regulatory, reimbursement, and clinical decisions has made it ISPOR's top health economics and outcomes research (HEOR) trend for 2024. Throughout ISPOR Europe 2024, RWE and RWD will be central themes, featured prominently in numerous sessions over the event's three days.

Harnessing the Power of AI in RWD: Enhanced Insights Through Machine Learning and Causal Inference Advances (Monday November 18, 11:45am – 12:45pm)

The multistakeholder panel, moderated by Jackie Vanderpuye-Orgle (Parexel International, USA) and including Patrice Verpillat (European Medicines Agency [EMA], The Netherlands) and Ipek Özer Stillman (Takeda, USA) will provide diverse insights into the use of RWD and causal machine learning to improve treatment decisions. Topics include regulatory perspectives on AI, target trial emulation, machine learning methods for addressing confounding, and manufacturer strategies for evidence generation and early-phase decision-making.

Advancing Real-World Evidence and Health Technology Assessment for Medical Devices and Diagnostics (Monday November 18, 11:45am – 12:45pm)

This issue forum, moderated by Arturo Cabra (GE Healthcare, USA), will explore improving health technology assessment (HTA) frameworks for medical devices and diagnostics, highlighting the role of RWE. Lavinia Ferrante di Ruffano (University of York, York Health Economics Consortium, UK) will discuss evaluation methods for diagnostics, while David Tamblyn (The University of Adelaide, Australia) will address current HTA challenges. Chiara Capobianco (Boston Scientific, Italy) and Colleen Farrell Longacre (Medtronic, USA) will share industry perspectives on using RWE to demonstrate value.

Fit-for-Purpose Real-World Data: Principles and Developments (Monday November 18, 1:45pm – 2:45pm)

This Spotlight Session, featuring Jeremy Rassen (Aetion, Inc., USA), Daniel Prieto-Alhambra (Oxford University, UK) and Sebastian Schneeweiss (Brigham and Women's Hospital, USA) will explore principles and developments for ensuring fit-for-purpose RWD in HEOR. Topics include minimizing information bias, evaluating measurement characteristics, and using validation methods. The speakers will connect these discussions to recent FDA/EMA guidance, highlighting implications for federated data analyses and best practices for RWD producers.

Data Analysis and Real-World Interrogation Network – European Union (DARWIN EU®) – Where Are We Now and What Is in Store for the Future? (Monday November 18, 3:15pm – 4:15pm)

The session, moderated by Phani Veeranki, will provide an update on DARWIN EU's progress and future plans. Patrice Verpillat, ISPOR Europe 2024 Program Committee Co-Chair, will discuss the regulatory perspective, Shuvayu Sen (Merck & Co., Inc., USA) will provide an industry viewpoint, and Daniel Prieto-Alhambra will offer insights on maintenance. Topics include DARWIN EU's pilot projects, including the EHDS HealthData@EU Pilot, and the implications for medicine development, lifecycle management, and access across the European Union.

Innovation or Stagnation? Unpacking Issues and Solutions in the Uptake of Advanced Methods for Real World Evidence (Monday November 18, 5:00pm – 6:00pm)

The issue panel will explore challenges and opportunities in implementing advanced RWE methods, with viewpoints from HTA/regulatory bodies, industry, and academia. Stephen Duffield (National Institute for Health and Care Excellence [NICE], UK) will address NICE's perspective and the challenges the agency faces with submissions, Matthew Dixon (Bristol Myers Squibb, USA) will provide the industry perspective, Barbara Dickerman (Harvard TH Chan School of Public Health, USA) will discuss executing advanced methods (such as target trial emulation) and Grace Hsu (Cytel, USA) will share insights from case studies.

Comparing and Contrasting RWE Guidance: What Researchers Need to Know Considering the Global Picture (Tuesday November 19, 10:15am – 11:15am)

Moderator Ulka Campbell (Aetion, Inc., USA) will lead the discussing explore recent RWE guidance from agencies like the FDA, EMA, and NICE, focusing on data quality and other topics. Panelists, including Solange Corriol-Rohou (AstraZeneca, France), Páll Jónsson (NICE, UK) and Patrice Verpillat, will discuss differences and similarities in the guidance and propose areas for harmonization. The session aims to help researchers effectively design real-world studies amidst varied regulatory and HTA expectations.

ISPOR Real-World Evidence (RWE) Special Interest Group (Tuesday November 19, 11:45am – 12:45pm)

The ISPOR RWE Special Interest Group (SIG), chaired by Sandipan Bhattacharjee (Bayer US LLC, USA) and Leanne Larson (ZS Associates, USA), will discuss the expanding role of RWE in healthcare and pharmaceutical decision-making, followed by breakout sessions on data analytics, AI, regulatory aspects, and case studies in RWE.

From Data to Decisions: The Potential of RWE Transportability in Healthcare Decision Making (Tuesday November 19, 1:45pm – 2:45pm)

Blythe Adamson (Flatiron Health, USA) will lead this workshop on the use of transportability analysis to enhance the application of RWD in HTA. Alexandra Starry (Cytel, Germany) and Marian Eberl (Daiichi Sankyo Europe, Germany) will join Adamson and Stephen Duffield to explore frameworks, industry challenges, and decision-maker perspectives on using international data, aiming to inform attendees on designing effective transportability studies to improve evidence applicability across different settings.

Real-World Evidence Policy: Is Harmonization Between Regulatory and HTA a Help or Hindrance? (Tuesday November 19, 5:00pm – 6:00pm)

This workshop, led by Rita Peeters (J&J MedTech, Belgium), will delve into the potential benefits and challenges of harmonizing RWE policies between regulatory and HTA bodies. Speakers Katharine Cresswell (NICE, UK), Patrice Verpillat, and Niklas Hedberg (Dental and Pharmaceuticals Benefits Agency [TLV], Sweden) will provide insights on policy evolution, gaps, and differing perspectives. The session will include interactive discussions and real-time polling to identify consensus on whether harmonization supports or hinders effective RWE policy development in healthcare decision-making.

Take My Word for It: Can Patient-Centric Social Listening Enrich RWE? (Wednesday November 20, 10:00am – 11:00am)

In an increasingly “connected world”, social media platforms offer a wealth of patient experiences, providing valuable insights into disease burden, quality of life, and treatment outcomes. Moderated by Siddharth Ramanan (Costello Medical, UK), this panel will delve into the potential of social media listening (SML) as a source of RWD. Jackie Cuyvers (Convosphere, UK) will discuss the use of AI in SML, Mary Jo Lamberti (Tufts University, USA) will highlight challenges and applications in clinical settings, and Jeanette Kusel (NICE, UK) will provide a decision-maker’s perspective on standards for SML in health technology assessments.

Latest research in RWD and RWE

Complementing these sessions, ISPOR Europe 2024 will feature a wide range of research posters exploring the latest advancements in RWD and RWE, covering topics such as innovative methodologies for RWD collection and analysis, applications of RWE in HTA, effectiveness and safety studies using observational data, and the integration of RWE in regulatory decision-making.

On Monday November 18 (5:00pm – 6:00pm) the Podium Session “RWE Methods and Applications" will include posters covering current considerations for using RWE in decision-making, followed by discussions on applications and methods for incorporating RWE in evidence generation, featuring:

• A Novel Framework for Quantitative Bias Analysis in the Presence of Non-Proportional Hazards Using the Accelerated Failure Time Model – Amy Macdougall (Arcturis Data, UK)
• Current and Future Use of RWE in HTA Decision-Making: Payers View Globally – Sabina Heinz (Ipsos, UK)

A second Podium Session “Real-World Analyses of Electronic Health Records” on Tuesday November 19 (5:00pm – 6:00pm) will feature research on methods to curate and analyze electronic health records, including:

• Optimizing Electronic Medical Records Data Completeness With on-Premise Artificial Intelligence: A Study on Large Language Models Enhancing Medication Documentation – Or Shaked (Briya Labs, Israel)
• Primary Care Resource Utilization and Costs of Imminent Subsequent Fractures in Postmenopausal Women: A Distributed Network Analysis Using Data Mapped to OMOP Common Data Model from Five European Countries – Gianluca Fabiano (University of Oxford, UK)

Other notable posters being presented include:

• Navigating Real-World Data Collection From Early Access Programs in the UK: An Underutilized Opportunity – Rakesh Davda (Bionical Emas Limited, UK; Monday 18, Poster Session 1)
• Development of Oncology EHR-Derived RWD in Europe and Japan – Blythe Adamson (Flatiron Health, USA; Monday 18, Poster Session 2)
• Is Individual-Patient Data Essential for Transportability, or Can Aggregated Data Suffice? – Per-Olof Thuresson (F. Hoffmann-La Roche Ltd, Switzerland; Monday 18, Poster Session 2)
• Reviewing the HTA and Regulatory Policy Landscape on Acceptability Standards for Real-World Evidence – Ravinder Claire (NICE, UK; Monday 18, Poster Session 2)
• Researcher Difficulties Using Secondary Data Sources to Generate Real-World Evidence: Results From an Online Survey – David Thompson (Rubidoux Research LLC, USA; Monday 18, Poster Session 2)
• How Does Real-World Evidence Support Regulatory and HTA Agency Assessments and Beyond? – Lucy Sam (Avalere Health, UK; Tuesday 19, Poster Session 3)
• Leveraging Real-World Evidence to Extend Drug-Drug Interaction Assessment From Drug Development to Clinical Care: A Targeted Review of the Literature – Nadia Quignot (Certara, France; Tuesday 19, Poster Session 4)
• Identifying Fit-for-Emulation Data: Adaptation of a Structured Data Feasibility Assessment Process for Real-World Oncology Trial Emulations – Adina Estrin (Aetion, USA; Tuesday 19, Poster Session 4)
• The Potential of Real-World Data (RWD) for Informing FDA Biosimilar and Interchangeable Biosimilar Regulatory Decisions: Recommendations From a Multi-Stakeholder Expert Panel – Cate Lockhart (Chief Science Officer, AMCP and Executive Director at Biologics and Biosimilars Collective Intelligence Consortium, USA; Wednesday 20, Poster Session 5)
• Optimizing RWE Design and Conduct by Leveraging Computable Operational Definitions Indication Libraries: Development, Verification, and Value – Aaron Kamauu (Navidence LLC, USA; Wednesday 20, Poster Session 5)

Stay up-to-date with The Evidence Base

The Evidence Base will be providing exclusive coverage of ISPOR Europe 2024 (November 17–20, 2024, Barcelona, Spain), providing daily rounds-ups of many of the sessions as well as deep dives into the three plenaries. Register on The Evidence Base and follow us on LinkedIn to make sure you don't miss any of our coverage and to ensure you stay up-to-date with the latest insights and developments from the conference.