9:00 am Check-In & Coffee
9:25 am Chair’s Opening Remarks
Integrating Real-World Evidence Across the Product Lifecycle to Drive Actionable, Cross- Functional Impact
9:30 am The Role of RWE in Integrated Evidence Plans
Synopsis
- Integration of Real-World Evidence (RWE) into iEP
- The role and purpose of RWE in iEP
- Dissemination of case study findings
10:00 am Enhancing Trial Relevance with Big Data & Pragmatic Design: Rethinking Inclusion & Exclusion Criteria
Synopsis
- Leverage real-world datasets and big data analytics to assess how traditional eligibility criteria may limit trial generalizability and impact patient access
- Explore the use of pragmatic trial design to better reflect real-world populations, treatment pathways, and comorbidities
- Share case studies and methodologies for redefining inclusion and exclusion criteria to optimize patient recruitment, retention, and evidence applicability
10:30 am Supporting Data-Driven Decisions with Real-World Evidence for Ultra- Rare Pediatric Neurological Diseases
Synopsis
- Explore innovative approaches to generating real-world evidence in ultra-rare pediatric neurological conditions, where traditional data is limited
- Address challenges in evidence generation, including data availability, small sample sizes, data heterogeneity, and follow-up
- Drive regulatory, clinical, and access decision makers through cross-functional collaborations and data sources tailored to rare pediatric populations
11:00 am Morning Break & Networking
Implementing Patient Voice to Enhance RWE Reliability & Accuracy
11:30 am Capturing the Lived Experience by Integrating Patient-Reported Outcomes into Real-World Data Streams
Synopsis
- Demonstrating how structured PRO collection enhances clinical relevance and granularity of RWE studies
- Discussing methodologies for embedding patient-reported data into electronic health records, registries, and digital health platforms
- Exploring the impact of PROs on treatment effectiveness evaluation, particularly in chronic and rare disease contexts
12:00 pm Roundtable: Embedding the Patient Voice in RWE From Insight to Impact
Synopsis
- Integrate patient-reported outcomes, preferences, and experiences to enrich RWE datasets and align evidence with real-world clinical value
- Explore frameworks and tools for capturing structured and unstructured patient voice data across diverse populations and settings
- Assess the impact of patient-centric RWE on regulatory submissions, payer decisions, and lifecycle evidence generation strategies
12:30 pm Lunch & Networking
Harnessing Innovative Real-World Insights
1:30 pm Leveraging Retrospective Observational Studies for Rapid, Scalable Evidence Generation
Synopsis
- Outlining best practices for designing retrospective cohort and case-control studies using EHRs and claims data
- Addressing common biases and confounding factors with methodological safeguards such as propensity scoring and sensitivity analysis
- Highlighting successful use cases in post-market safety surveillance and comparative effectiveness.
2:00 pm Roundtable: Driving Accountability & Innovation by Clarifying Ownership of Data Quality in a Decentralized RWE Landscape
Synopsis
- Assessing how the rise of internal RWE hubs, external providers and tech entrants reshapes responsibility for data quality
- Debating who should own and maintain quality standards pharma, providers, tech platforms, or new RWE custodians
- Proposing governance frameworks that align data stewardship with end-user needs and regulatory standards