In 2018, the ASH Research Collaborative (ASH RC) was established as a non-profit supporting organization for ASH to improve the lives of people affected by blood diseases. The foundation of the ASH RC is its Data Hub — a technology platform that facilitates the exchange of information by aggregating individual-level, research-grade data on hematologic diseases. The initial two programs of the ASH RC Data Hub were established in sickle cell disease (SCD) and multiple myeloma.
The ASH RC Data Hub launched at an important time in the evolution of real-world data (RWD) as an accepted source for evidence generation in medicine and health care. The 21st Century Cures Act ushered in new technological improvements that offered the opportunity to improve the time and quality of aggregating RWD through electronic health records (EHRs) and other data sources.
As the ASH RC Data Hub has developed its two initial programs, the world has changed around it, and the potential importance of the Data Hub to the field of hematology has increased. The COVID-19 pandemic illustrated the opportunity for observational RWD to answer key questions in a timely way that might have been challenging or infeasible with traditional randomized controlled trials. The health care workforce experienced substantial attrition amidst a wave of burnout and overall workforce realignment. We have now entered an era in which RWD and programs built on RWD are urgently needed to generate evidence, improve outcomes for patients and families, and foster efficiency and meaning in clinical care delivery and research in ways that can help sustain the field of hematology.
There is no better time for ASH members to become familiar with the ASH RC Data Hub. In this article, I will describe the foundational principles of this work and how the program has been built over the past few years. We hope that you’ll come visit the ASH RC kiosk in ASH Central (Hall F) at the ASH annual meeting and join us in our mission.
RWD GENERATOR
RWD are data relating to patient health status or the delivery of health care routinely collected from a variety of sources, whether or not those sources were originally built for the purpose of evidence generation. One commonly used RWD data source is the EHR, though there are others, such as claims data, product and disease registries, and patient-generated data from paper, computers, mobile devices, and sensors.
Patient-level data derived from EHRs hold significant promise to understand hematologic conditions and treatment, though there are notable limitations. The ASH RC Data Hub has addressed these challenges, in part through leveraging common data standards including the Observational Medical Outcomes Partnership Common Data Model and the Fast Healthcare Interoperability Resource (FHIR) transmission pathways, including EHR Application Programming Interfaces (APIs) recently mandated by federal legislation.
Beyond data models and transmission standards, we also recognize that structured EHR data used primarily for billing may be insufficient to represent complicated health concepts relevant to hematology. We have developed a Data Quality Program that includes the use of electronic phenotypes and pre-populated electronic case report forms to allow for data entry, curation, and verification at the site level, by those who know their patients best. We feel this strategy will generate data of sufficient quality to be usable for research and clinical practice improvement.
We have also built a streamlined, electronic, centralized informed consent tool (eConsent) for patients to participate in the Data Hub. We can aggregate deidentified data from patients who are not consented, but the provision of consent also gives us the opportunity to take in protected health information-containing patient-level data, including unstructured text in notes and reports that can later be queried using natural language processing.
RWD LEADS TO REAL-WORLD EVIDENCE GENERATION
By providing access to RWD, the Data Hub can improve our understanding of patient characteristics, clinical practice patterns, and short- and long-term patient outcomes relevant to hematology. Under Dr. Rob Califf’s leadership, the U.S. Food and Drug Administration (FDA) is keenly focused on the potential for RWD to generate real-world evidence that is fit for purpose for regulatory research (www.fda.gov/science- research/science-and-research-special- topics/real-world-evidence). Examples cited in recent FDA guidance include the development of external control arms, observational efficacy studies, and postmarket requirements. The ASH RC Data Hub aims to meet these needs through discussions with sponsors and the FDA in the areas of SCD and multiple myeloma, including novel therapies.
In our recent real-world evidence initiative that the Data Hub co-sponsored with the Innovative Genomics Institute, with FDA collaboration, we explored how the Data Hub can serve as a coordinated registry network to provide real-world data from a variety of sources for regulatory purposes. When aggregated using this framework, multisource data have the potential to increase utility, reduce costs, and better reflect patients’ experiences compared with traditional methods.
The Data Hub is also positioned to generate evidence of direct benefit to the hematology research community, and to clinicians, patients, and families. One example of this is a nested prospective study that the Data Hub is currently launching from our multiple myeloma program.
With funding support from an industry sponsor, we have developed a decentralized study to look at real-world effectiveness of the bivalent COVID-19 booster vaccine in individuals with myeloma. The project will feature electronic consenting for patients, a portal for provision of patient-reported outcomes, and the ability for participants to obtain vaccines and antibody testing locally for convenience. Participants grant permission to share their antibody testing results in addition to their complete EHR, which will flow directly to the Data Hub.
This decentralized platform could serve as a template for a national RWD pandemic preparedness network and could also support non-COVID-19- related rapid RWD studies in hematology.
POWERING COLLABORATIVE PRACTICE IMPROVEMENT: THE ASH RC SCD LEARNING COMMUNITY
The primary purposes of the Data Hub are to accelerate research and improve clinical practice in order to achieve better outcomes for people living with hematologic conditions worldwide. Our efforts to promote practice change occur through the provision of near-real-time dashboards to sites and external stakeholders, and also through dedicated efforts built upon established quality improvement methodology. An important recent example of this is the recently launched Sickle Cell Disease Learning Community.
Inspired by the National Academies of Sciences, Engineering, and Medicine Blueprint Report, in addition to longstanding information illustrating the evidence- to-practice gap in SCD, the ASH RC sought and obtained support from the U.S. Department of Health and Human Services’ Office of Minority Health to establish a nationwide learning community in sickle cell disease. The global aim of the Learning Community is to profoundly improve the life course and quality of life of individuals living with SCD.
The Data Hub partnered with the Anderson Center at Cincinnati Children’s Hospital Medical Center — world leaders in learning community development in a variety of clinical practice areas — and included input from patients, families, clinicians, researchers, industry, government representatives, and many others in building the Learning Community. With the assistance of a multistakeholder design group and technical advisory group, the Learning Community launched its activities with a group of pediatric and adult pilot sites and focused its efforts on two main priorities: improving the reliable use of disease-modifying therapies and improving the reliable use of co-developed pain management plans.
In late September of 2022, the ASH RC hosted an inaugural in-person meeting of Learning Community participant sites, with activities organized under the mantra, “all teach, all learn.” Participants left the meeting energized and committed to addressing common challenges together, with the knowledge that they can accelerate the pace of change and dramatically expand their impact when all improve. Now that the pilot has launched successfully, the Learning Community will become available to all sites for participation in 2023, with ongoing operational support from the ASH RC and the ASH Committee on Quality.
WHAT’S TO COME
Despite this promising start, we believe that the best is yet to come for the ASH RC Data Hub, and the coming year is poised to become one of transformative growth. In addition to launching our decentralized study in multiple myeloma, we will be expanding our myeloma site participation from five to 15 and soon will make participating site data available through dashboards.
Our Sickle Cell Disease Learning Community will expand nationwide. Though we already have more than 8,000 individuals living with SCD represented in the Data Hub, accelerating the number of sites submitting production- level data using EHR FHIR APIs will grow the population represented in the Data Hub exponentially.
Activation of our eConsent portal in both programs will allow for invested participation by our patients, families, and communities. Soon, with implementation of our data quality program, our dataset will become available for research proposal applications from the hematology community.
Perhaps most importantly for ASH members, we plan to accelerate the evolution of the Data Hub to a program in which clinicians and researchers are deeply invested. Though technology can make our lives in the clinic more complicated at times, when used well it offers the potential to bring efficiency and value.
The ASH RC Data Hub is not an EHR vendor and is not a for-profit company. It exists in part to serve and represent the needs of all of us in the field of hematology who are committed to better outcomes for our patients. With tools to inform our research and care delivery and a platform that fosters and enables our calling as hematology professionals, the Data Hub has the potential to point to a better future for us and for our patients.
Again, we hope that you’ll come by to see us at the annual meeting to learn more about what we do and to see how you can become involved.
ASH RESEARCH COLLABORATIVE KIOSK
ASH Central, Hall F
MONDAY, DECEMBER 12
8:00 a.m. – 5:00 p.m.
MONDAY, DECEMBER 13
8:00 a.m. – 2:45 p.m.
William Wood, MD, is Professor of Medicine, Division of Hematology, University of North Carolina at Chapel Hill, Chapel Hill, NC.