Update on the ASH Research Collaborative Data Hub

On behalf of the ASH Research Collaborative (ASH RC) and the Data Hub, I am pleased to provide the hematology community with an update on our progress to date and plans for 2020. The ASH RC is a non-profit organization established by ASH in 2018 to foster collaborative partnerships that accelerate progress in hematology, with the goal of improving the lives of people affected by blood diseases. As a major initiative within ASH RC, the Data Hub aims to create the largest shared information resource within the global hematology community. ASH RC also contains a Sickle Cell Disease Clinical Trials Network (SCD CTN), an ambitious project designed to accelerate the development and evaluation of therapies in a large proportion of the United States population affected by SCD. Through the Data Hub, SCD CTN, and projects still to come, the ASH RC will transform research and practice in malignant and nonmalignant hematologic diseases throughout the world, for the benefit of patients and the hematology community.

Growing the Data Hub

The Data Hub is open for data contributions toward two initial diseases, multiple myeloma (MM) and SCD. We have already integrated a critical mass of data in these diseases that will allow the research community to answer questions of scientific interest. To date, the Data Hub has integrated data from six academic centers throughout the United States and Europe, representing thousands of patients with MM and SCD. We anticipate that this data repository will grow exponentially in 2020. The Data Hub also recently announced the contribution of the ASPIRE and ENDEAVOR data sets from Amgen, representing more than 1,300 patients with MM on therapeutic clinical trials, and the FISCO registry data set from Novartis, representing observational data from 500 patients with SCD.

In 2020, as the SCD CTN is launched, the plan is for data from patients with SCD at all participating CTN sites to be incorporated into the Data Hub. These data will represent consecutive patients with SCD who are seen at these sites — not limited to those on clinical trials — and will include a significant proportion of the U.S. population with this disease. Though MM does not have a CTN within the ASH RC, we plan for a major expansion of U.S. sites contributing prospective MM data to the Data Hub in 2020.

Refining Our Data Models

For data within the Data Hub to inform research and practice, data must be high quality and fit for purpose. We ensure this through the development of disease-specific data models for SCD, MM, and others yet to come. Once initial disease-specific models are developed and published, we will iteratively revise, refine, and update new releases of these models over time.

As we use the term, a “data model” refers to a collection of attributes that explain how we identify, categorize, and make data available for use. Data models contain high-priority data elements for each disease through a process that recognizes that some elements are critical for research and practice, while not all elements can be collected for each patient every time. Data elements have specific definitions, structure, sourcing, frequency, and context of collection. We house our data within a standardized architecture but can acquire and integrate data through a variety of channels. Our data models represent the “targets” to which sites aim their data contributions, while we work with sites to make available numerous accepted data contribution methods. We also ensure that previously collected data are transformed to meet new or updated data model specifications. We develop our data models through a rigorous process that involves many stakeholder perspectives. As these harmonized data models are developed, we anticipate publication and dissemination efforts to promote standard settings for data collection in these disease communities.

Real-World Data to Generate Real-World Evidence

The Data Hub represents an ideal vehicle to meet objectives outlined in the 21st Century Cures Act and elsewhere — to generate real world evidence that informs and accelerates development and evaluation of medical therapies that make a difference in the lives of patients. With the help of a well-functioning Data Hub, we are able to inform each step of the product lifecycle with observational data generated at participating sites. This includes potential premarket applications, as in the development of synthetic external control datasets that represent current standards of care, to post-approval surveillance. Potential contexts include long term follow-up of potentially curative therapies, as well as evidence generation to better understand the benefits and risks of non-curative pharmaceuticals in the real-world setting.

For real-world evidence to be “fit for purpose” for regulatory standards, data models must be developed with careful intention, with input from the U.S. Food and Drug Administration and others. In 2020, the Data Hub will continue a series of productive conversations with the FDA that have already begun. We anticipate that the FDA will provide continued input into our data models, and there might also be opportunities to host multi-stakeholder workshops to better understand evidence generation in the disease-specific contexts within which the ASH RC Data Hub operates. We will continue to explore potential opportunities like this in the year ahead.

Listening to the Patient Voice

A primary goal of ASH RC is to serve the needs of patients with hematologic diseases by catalyzing changes to research and practice that bring about better quality of life, improved clinical outcomes, and ultimately cures. Incorporating the patient voice is critical to achieving this mission.

As the SCD CTN has been developed, a series of community engagement meetings have allowed patients, caregivers, and others to come together to share their perspectives on clinical research in SCD. These types of platforms are important ways to understand priorities, incentives, and barriers to participation in research for patients. We envision continued engagement throughout future CTN and Data Hub activities. We are also developing plans for a patient-friendly electronic informed consent process for ASH RC activities that require consent; a web-based patient portal for longitudinal engagement; community newsletters; and opportunities for participants to engage in other projects or advisory opportunities as they arise. We are also developing ways to collect data that directly reflect the patient voice. Future versions of the portal will include the ability to capture patient-reported outcomes (PROs) in areas such as symptoms and quality of life, disease-related symptoms, symptomatic toxicities, and physical function.

Engaging the Research Community

The Data Hub potentially represents a valuable resource to help the research community, and we are continuing to develop the Data Hub with the needs of the research community in mind. We are actively refining policies for analyses and publications and will make these available to the hematology community soon. We are also working with experts in MM and SCD to identify potential early analyses as “test cases” to evaluate the fitness of our current procedures. We plan to use these early analyses to iteratively inform our data models and data capture strategy to facilitate subsequent projects and widespread use of the Data Hub within the hematology research community.

Engaging the Practice Community

We also envision that the Data Hub will serve as a catalyst for practice transformation, to improve outcomes in real time for patients affected by hematologic diseases. In 2020, we are working to develop reports for sites contributing data to the Data Hub and will collaborate with sites to ensure that these reports are constructed in a way that best meets their needs. These reports will include data on consecutive patients from each site and will support quality improvement and other operational needs. We are also developing basic analytic data cuts for sites and other stakeholders so that specific subpopulations of interest can be identified and tracked. In the future, we envision alignment of ASH RC with other ASH activities designed to support and improve practice, such as development and integration of metrics associated with ASH guidelines in SCD and other conditions.

Expanding the Scope

The long-term goal of the ASH RC is to address the entire spectrum of malignant and nonmalignant hematologic diseases. This means we will need to expand our scope beyond our current focus on MM and SCD once these initial efforts have reached maturity. We anticipate a general call to the hematology community for submission of potential disease projects.

Conclusion

The ASH Research Collaborative is an ambitious and visionary project that aims to improve the lives of people affected by blood diseases. Through our initial work with the SCD CTN and the Data Hub, we have begun to lay the foundation for the ASH Research Collaborative for years to come. We are excited to build this resource for the hematology community and we welcome interest from anyone who would like to join us in our mission.