66th ASH Annual Meeting: ASH Research Collaborative Data Hub Sickle Cell Disease Network: Accelerating Research and Collaborative Clinical Care through Real-World Evidence Generation

Special-Interest Sessions

Research, Sickle Cell Disease, Clinical Practice (Health Services and Quality), Clinical Research, Education, Hemoglobinopathies, Diseases, Real-world evidence, Treatment Considerations, Biological therapies, Registries

Room 11 (San Diego Convention Center)

Co-chairs:

Alexis Thompson, MD, MPH, Children's Hospital of Philadelphia and Perelman School of Medicine, University of Pennsylvania and Ted Wun, MD, Division of Hematology and Oncology, UC Davis Comprehensive Cancer Center

Disclosures:

Thompson: Beam Therapeutics: Consultancy, Research Funding; Editas: Consultancy, Research Funding; Novartis: Research Funding; CRISPR/Vertex: Consultancy, Research Funding; Global Blood Therapeutics: Divested equity in a private or publicly-traded company in the past 24 months; bluebird bio: Consultancy, Research Funding. Wun: Pfizer, Inc: Membership on an entity's Board of Directors or advisory committees.

During this 90-minute special-interest session, sickle cell disease (SCD) registry and real-world evidence (RWE) leaders will discuss progress across numerous SCD data initiatives, including the ASH Research Collaborative's SCD Data Hub and other federal and non-federal programs. Specific topics include examples of using real-world data (RWD) to generate RWE, how RWD can be used to monitor clinical practice and outcomes for patients who receive cell and gene therapies, various approaches to ensuring data quality when using electronic health record data (EHR), federal priorities related to RWE generation that supports regulated research, and a new project to increase SCD-specific data standards with EHRs.

Representing Sickle Cell Disease Health Concepts in Real World Evidence Generation: Examples from Real World Data (RWD) Initiatives and the Importance to the Sickle Cell Disease Community

Alexis Thompson, MD, MPH

Children's Hospital of Philadelphia, Philadelphia, PA

The Role of RWD in Long-Term Follow-up of Cell and Gene Therapies in Sickle Cell Disease: The CIBMTR experience

Patty Steinert, PhD, MBA

Medical College of Wisconsin, Milwaukee, WI

Challenges in Data Quality: Where EHR Data Acquisition Approaches and Highly-Curated Datasets Intersect (1)

Sophie Lanzkron, MD

Thomas Jefferson University, Baltimore, MD; Thomas Jefferson University, Philadelphia, PA

Challenges in Data Quality: Where EHR Data Acquisition Approaches and Highly-Curated Datasets Intersect (2)

Ashima Singh, PhD, MS

Department of Pediatrics, Section of Hematology/Oncology/Bone Marrow Transplantation, Medical College of Wisconsin, Pewaukee, WI