Sickle Cell Disease Research Network
Facilitating Sickle Cell Disease Research to Improve Clinical Outcomes
Solving Real-World Data Challenges Through a Multistakeholder Approach
The use of real-world data (RWD) to generate real-world evidence (RWE) in both pre- and post-approval settings is gaining momentum, driven by evolving guidance from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). While relevance and reliability are established criteria for determining whether RWD is fit-for-purpose, applying these criteria consistently across data production and regulatory submissions remains challenging.
About the RWE Consortium for Sickle Cell Disease
The use of real-world data (RWD) to generate real-world evidence (RWE) in both pre- and post-approval settings is gaining momentum, driven by evolving guidance from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). While relevance and reliability are established criteria for determining whether RWD is fit-for-purpose, applying these criteria consistently across data production and regulatory submissions remains challenging.
Who’s Involved
The Consortium brings together:
- Experts from the ASH RC SCD Research Network
- Clinicians and researchers
- Industry partners
- Informatics professionals
- Individuals living with SCD
Consortium Project Deliverables
Each project led by the Consortium will produce:
- A consensus recommendations document defining optimal RWD representations of key clinical outcomes (e.g., vaso-occlusive events)
- Analytic outputs to test and validate these standards using the ASH RC Data Hub and SCD Research Network engagement
- Peer-reviewed publications outlining the methodologies and standards for using EHR data for SCD research
- Workshops with the FDA to refine the standards and evaluate their performance within the ASH RC infrastructure
ASH RESEARCH COLLABORATIVE
The ASH Research Collaborative (ASH RC) is a non-profit organization established by the American Society of Hematology (ASH) in 2017 to improve the lives of people affected by blood diseases by fostering collaborative partnerships to accelerate progress in hematology. Learn more about ASH RC