Sickle Cell Disease Research Network
Facilitating Sickle Cell Disease Research to Improve Clinical Outcomes
Real-World Evidence Consortium for Sickle Cell Disease (SCD)
Solving Real-World Data Challenges Through a Multistakeholder Approach
The use of real-world data (RWD) to generate real world evidence (RWE) in both the pre- and post-approval settings for regulatory review is accelerating considering evolving guidance from the Food and Drug Administration (FDA) and European Medicines Agency (EMA). Though relevance and reliability have been established as standards to determine whether RWD are fit-for-purpose, the translation of these criteria to ways in which RWD is produced and submitted for review remains a challenge.
To address this gap, the ASH Research Collaborative (ASH RC) and the Reagan-Udall Foundation for the Food and Drug Administration (FDA Foundation) launched the Real-World Evidence Consortium (RWE-C) for Sickle Cell Disease (SCD).
The Consortium brings together multidisciplinary experts to develop consensus-based recommendations on clinical outcomes important to treating people with SCD and apply those standards to real-world data sets. The overarching goal is to improve the lives of individuals living with SCD through real-world evidence generation and cutting-edge research.
The Consortium consists of membership from SCD experts from the ASH RC SCD Research Network, real world data and informatics experts, epidemiologists and researchers, individuals living with SCD, and industry.
Each Consortium project produces:
- Consensus recommendations defining optimal RWD representation of key clinical concepts of interest (e.g., vaso-occlusive events)
- Analytic validations through testing and evaluations of data standards in the ASH RC Data Hub and Network
- Publications of the standards, methodologies, and findings
- Workshops with the FDA to discuss and refine the standards and to review the performance of the standards using the ASH RC Data Hub and Network
Check back to see updates and publications produced by the Consortium.
Governance
Steering Committee
Provides scientific leadership and strategic direction for Consortium initiatives
- Johan Cherian, MD – NovoNordisk
- Lewis Hsu, MD – University of Illinois Chicago
- Tilicia Mayo-Gamble, PhD – Georgia Southern University
- Donna Neuberg, ScD – Harvard School of Public Health
- Pablo Rendo, MD – Sanofi
- Andres Rondon, PhD – Sanofi
- Ashima Singh, PhD – Medical College of Wisconsin
- John J. Strouse, MD, PhD – Duke University Medical Center
- William Wood, MD, MPH – University of North Caroline School of Medicine
- Ted Wun, MD, FACP – UC Davis Clinical and Translational Science Center
Innovation Committee
Provides multidisciplinary expertise to inform data standards, analytic approaches, and implementation within real-world data environments
- Chris Barnowski, Sr. Medical Director – Novo Nordisk
- Amanda Borens, Founder – Tesselate Data Consulting
- Amanda Brandow, Physician – Medical College of Wisconsin
- David C. Brousseau, Pediatrician-in-Chief – Nemours Children’s Health
- Peter Camacho, Manager, Data Management & Reporting – Children’s Hospital of Philadelphia
- Elizabeth Ford, Patient Experience – University of Illinois Chicago
- Johan Cherian, Global Medical Director – Novo Nordisk
- Mariame Kayle, Associate Professor – Duke University Medical Center
- Adrien Ramanana Rahary, External Consultant in Real World Data and Evidence – Sanofi
- Andres Rondon, RWE Clinical Development Head – Sanofi
- Alexis Thompson, Chief, Division of Hematology – Children’s Hospital of Philadelphia
- Lori Vick, Assistant Professor – University of South Carolina
- Neil Zakai, Professor, Hematology – University of Vermont Medical Center
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