Accelerating novel therapies for sickle cell disease with RWE: ASH RC Data Hub as a strategically coordinated registry network

Real-world evidence (RWE) has been used to improve quality of care, accelerate innovation, and evaluate emerging therapies for drugs, devices, and biologics. The Coordinated Registry Network (CRN), aggregating and linking highly curated patient data, has emerged as a model for RWE generation that has guided the development of the American Society of Hematology Research Collaborative (ASH RC) Data Hub. With the aim of bolstering research, enhancing clinical care, and expediting evidence generation using RWE, the ASH RC and the Innovative Genomics Institute launched a joint initiative, "Accelerating Innovations for Sickle Cell Disease (SCD) with Real-World Evidence," to evaluate and make recommendations to the Data Hub. An expert panel used a "maturity model" to evaluate the following: (1) Data Hub standardization of RWE guidelines to ensure successful monitoring of new therapies' safety and effectiveness; and (2) the Data Hub's engagement with patients (including collection of data on patient experience) and collaboration among partners, including regulators, industry, and clinicians. The Data Hub gathers patient-level data from electronic health records, with plans to incorporate data from individuals receiving gene therapy. The program is currently curating and aggregating data on ∼27 000 individuals with SCD cared for across 22 hospitals in the United States. The SCD Data Hub program includes a total of 56 centers, some of which are not yet contributing data, which will increase the total cohort to ∼50 000 patients. The unique organizational structure of the CRN model facilitates acceleration of innovation through collaborative relationships between multiple clinical sites and partners with various evidentiary needs.