Day One

TUESDAY, SEPTEMBER 29

8:00 am Coffee & Registration

8:50 am Chair’s Opening Remarks

Pre-Clinical Value Generation & Research

9:00 am Panel: Determining Where Value Lies in RWE

Synopsis

  • A thought experiment to determine where value is found in RWE from functional perspectives

9:45 am Strategies for Leveraging Real World Data to Support Early Development and Value Proposition for Successful Product Launch

  • Tehseen Salimi Vice President of Medical Affairs and Real World Evidence, G1 Therapeutics

Synopsis

  • Evaluating the role of insights, gaps and unmet needs assessments
  • Integrating RWE practice early into the drug development continuum
  • Assessing how to meet the needs for RWE for payers, regulators, providers and patients

10:15 am Morning Break and Networking

11:15 am Opportunities for Patient Perspective Research Supporting Drug Development Lifecycle

Synopsis

  • Examining opportunities for patient perspective research throughout the product lifecycle
  • Reviewing study types to collect patient perspective data
  • Choosing appropriate instruments with study objectives in mind
  • Exploring examples of patient perspective research to highlight disease burden & unmet need

Strategy & Implementation

11:45 am How to Use RWE to Better Define Trial Protocol: Syneos’s Case Studies

Synopsis

  • Walking you through the methods to increase evidence from a wealth of experience
  • Outlining how RWE can accelerate the time to market
  • Reflecting on the benefits of synthetic and external control arms in the seeking of conditional approval

12:15 pm RWE and Patient Advocacy – Patient Centricity

Synopsis

  • Development of innovative medicines where there is no current standard of care poses unique challenges
  • Outline considerations in fully understanding patient burden and translating value into patient-relevant outcomes
  • Share experiences of how patient advocacy partnerships and RWE can support access strategie

12:45 pm Lunch & Networking

1:45 pm Interweaving RWE into the Strategic Fiber of Therapeutic Areas

Synopsis

  • Sharing previous experience in the unique randomized trial of Merkel Cell Carcinoma using an EMR data set
  • Dissecting methodologies and mistakes when integrating RWE into a trial
  • Highlighting the importance that each department has towards the shared RWE trial objective

2:15 pm Identifying Gaps in Current Patient Care Partnering to Accelerate Patient-Centric Real-World Evidence

  • Angela Dobes Vice President, IBD Plexus, Crohn’s & Colitis Foundation

Synopsis

  • Describing IBD Plexus: the largest US collaborative real-world database and biobank in the IBD field with 8 pharma members, over 80 academic and community medical centers, and over 25,000 patients
  • Detailing the identification and co-development process for high priority real world outcomes and endpoints including rare real-world patient experience data and a new PRO endpoint
  • Improving patient access to effective treatments and diagnostics by generating real-world evidence to enhance product labels and scientific communications

2:45 pm Understanding the Global RWE Regulatory Landscape

  • David Anstatt Executive Director, Center for Observational Research & Data Sciences, Bristol-Myers Squibb

Synopsis

  • Analyzing the differences in EU/US evidence demand from health authorities
  • Considering the different regulatory propensities
  • Strategizing how to best use reasonable data for each approach

3:15 pm Afternoon Break & Networking

3:45 pm RWE Pre & Post Launch Elements

Synopsis

  • Scrutinizing how this data is being used for discussion with payers, regulators, physicians etc. pre and post launch
  • Analyzing HTA/RWE pre and post launch
  • Using RWE to guide ICER assessments
  • Fostering cross functional collaboration (internal colleagues and partners) in RWE pre and post launch

4:15 pm Case Study: RWE Global Opportunities & Emerging Markets

  • Maarten Beekman Respiratory Medical Director, International Region, AstraZeneca

Synopsis

  • Developing a global RWE framework based on evidence gaps
  • Exploring ways to create and collate data in emerging markets
  • Explaining ways to present RWE data to prove value in changing clinical practice

4:45 pm Setting Standards for RWE in Comparative Effectiveness Research: Methods, Transparency & Governance

  • Dan Ollendorf Director, Value Measurement & Global Health Initiatives, Center for the Evaluation of Value and Risk in Health (CEVR)

Synopsis

  • Articulating robust approaches to ensure that RWE data is fit for purpose and methods are applied in a clear and interoperable manner across settings
  • Providing standards for multi-stakeholder transparency regarding study intent, design, and outcome
  • Hypothesizing possible venues for a repository of RWE studies and guidance for content, governance and maintenance

5:15 pm Chair’s Closing Remarks & End of Day 1