Day Two | NOW LIVE!
Wednesday 11, June 2025
08:00 am Coffee + Light Breakfast
8:20 am Chair’s Opening Remarks
Optimizing HEOR & Market Access Approaches to Maximize RWE Utility
8:30 am Understanding FMV Assessments in Global Medical Affairs for Research & HEOR
Synopsis
- Providing clarity around the purpose of FMVs in research and HEOR
- What is required in an FMV assessment for a clinical study?
- Case study-based examples of FMV application
9:00 am Introduction to MDV Database
Synopsis
- Improve healthcare using real-world data through collection, analysis, and utilization services for pharma, academia, and healthcare institutions
- Provide hospital and insurance databases with specific strengths, supporting clinical research, drug development, and healthcare policy
- Focus on data privacy, standardization, and ethical considerations to ensure secure and accurate medical data usage
9:10 am Optimizing Understanding Around How RWE Can Support Pricing & Reimbursement Strategies
Synopsis
- Explore how real-world evidence (RWE) can substantiate the clinical and economic value of therapies to strengthen pricing and reimbursement proposals
- Understand how to tailor RWE to meet the specific needs of payers, including costeffectiveness analysis and patient outcomes
- Learn how to use RWE to navigate pricing negotiations and reimbursement processes, ensuring that evidence supports broader access to treatments
9:40 am Optimizing Evidence Generation in Early Clinical Stages to Enhance the Value Proposition for Market Access & Reimbursement
Synopsis
- Discover how incorporating real-world evidence early can strengthen clinical trial design and endpoint selection
- Explore strategies to align early evidence generation with payer and regulator expectations to support market access and reimbursement discussions
- Learn best practices for leveraging early-stage data to build robust value propositions and accelerate time-to-market
10:25 am Morning Break
Optimizing Study Designs & Empowering Patients to Elevate RWE Utility
10:55 am Methodologies for Designing Robust ECAs to Enhance the Quality & Reliability of ECA Studies
Synopsis
- Explore best practices for selecting appropriate external control populations, ensuring that data sources are comparable, and the methodology is robust for reliable outcomes
- Addressing potential biases and confounding factors in ECA design through statistical techniques, matching methods, and sensitivity analyses to improve study validity
- Key strategies for ensuring the reproducibility and generalizability of ECA results, including careful consideration of data quality, external validity, and regulatory acceptance
11:25 am Maximizing Synergies Between Industry & Public Research to Elevate RWE Utility
Synopsis
- Explore strategies to strengthen collaboration between industry and public research entities to enhance the quality and scope of real-world evidence (RWE)
- Learn how to optimize data sharing, infrastructure, and expertise to maximize the utility and impact of RWE across both sectors
- Discover methods for aligning industry goals with public research priorities to ensure that RWE supports both commercial needs and societal health outcomes
12:10 pm Improving Patient Experience Data Collection in Real-World Settings to Advance the Utility of Treatments
Synopsis
- Optimizing patient-reported outcomes (PROs), surveys, and mobile health apps to gather real-time feedback on patient experiences and treatment effectiveness in realworld settings
- Strategies to improve the consistency and reliability of patient experience data through standardized questionnaires, targeted follow-ups, and integration with electronic health records (EHRs)
- Analyzing patient experience data to identify unmet needs, refine treatment protocols, and shape personalized care strategies to improve overall treatment outcomes
12:40 pm Lunch
1:40 pm AI-Powered Insights: How NICE is thinking about AI-enhanced Real-World Evidence
Synopsis
- AI is looking to affect all parts of the evidence-to-recommendation pipeline. Through its Statement of Intent for AI, NICE has signaled its intention to develop its approach to the use and appraisal of AI, and to develop guidance
- Dr. Stephen Duffield will discuss NICE’s position statement on the appropriate use of AI, as relates to real world evidence
- Case studies and pilot studies will also be described, and how NICE is learning from these to further develop it’s best practice recommendations, including NICE’s RWE framework
2:10 pm Role of Digital Twins in Clinical Development and Data Generation in Supporting IEP Generation
Synopsis
• Simulate patient outcomes to optimize trial design
• Integrate real-time data to strengthen IEP evidence
• Support regulatory and payer decisions with predictive models
2:40 pm 2:40 pm Afternoon Break & Networking
3:10 pm Real-World Evidence as an Investment: Measuring Impact
Synopsis
- Why measuring RWE impact matters short and long-term
- What can we learn from R&D return calculations
- Building RWE impact assessment best practices: suggested metrics
3:40 pm Strategies for Ensuring Data Privacy & Security While Protecting Patient Data & Enabling Valuable Research
Synopsis
- Explore techniques like data anonymization, pseudonymization, and federated learning to safeguard patient data without compromising research quality
- Understand how to align RWE studies with data protection laws such as GDPR, HIPAA, and other regional regulations
- Learn best practices for securing healthcare data, including encryption, access controls, and real-time monitoring, to foster trust and compliance