Accelerating Novel Therapies for Sickle Cell Disease with RWE: ASH RC Data Hub as a Strategically Coordinated Registry Network Open Access

• ASH RC Data Hub advances real-world evidence in hematology, currently for sickle cell disease and multiple myeloma, fostering innovation.

• This paper presents ASH RC Data Hub site distributions, enrollment metrics, curation processes, and quality control initiatives.

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 ASH Research Collaborative® (ASH RC) Data Hub. With the aim of bolstering research, enhancing clinical care, and expediting evidence generation using RWE, ASH RC, and the Innovative Genomics Institute (IGI) 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: 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 approximately 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 approximately 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.