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

Area of Science:

• Real-world evidence (RWE) generation and application in hematology.
• Data science and informatics for healthcare research.
• Sickle Cell Disease (SCD) research and clinical management.

Background:

• Real-world evidence (RWE) is crucial for improving care, fostering innovation, and evaluating therapies.
• The Coordinated Registry Network (CRN) model, aggregating curated patient data, is a key RWE generation strategy.
• The ASH Research Collaborative® (ASH RC) Data Hub utilizes the CRN model to advance RWE generation.

Purpose of the Study:

• To evaluate and provide recommendations for the ASH Research Collaborative® (ASH RC) Data Hub for Sickle Cell Disease (SCD).
• To assess the Data Hub's standardization of RWE guidelines for monitoring new therapies.
• To evaluate patient engagement and collaboration among stakeholders, including regulators, industry, and clinicians.

Main Methods:

• A joint initiative by ASH RC and the Innovative Genomics Institute (IGI) to bolster SCD research using RWE.
• An expert panel employed a "maturity model" to evaluate Data Hub standardization and engagement.
• Data collection from electronic health records, with future incorporation of gene therapy data.

Main Results:

• The SCD Data Hub is currently aggregating data on approximately 27,000 individuals with SCD across 22 US hospitals.
• The program involves 56 centers, with plans to expand the cohort to approximately 50,000 patients.
• The CRN model's structure facilitates innovation through multi-site collaboration and diverse evidentiary needs.

Conclusions:

• The ASH RC Data Hub, utilizing the CRN model, is a significant step in advancing RWE generation for SCD.
• The initiative aims to enhance clinical care, expedite evidence generation, and accelerate therapeutic innovation.
• Collaborative relationships and robust data standardization are key to successful RWE generation for novel therapies.