The Data Hub
A Source for Real-World Hematology Data
Accelerating Genomic Therapies with Real-World Evidence
Genomic therapies have the potential to drastically improve outcomes for individuals with genetic hematologic conditions, such as sickle cell disease (SCD). As new data emerge in this area, we are getting closer to broader use of these therapies. We must be prepared to meet the need for continued monitoring when these approaches are applied in real-world treatment settings.
Reaching consensus on standards for real-world data collection and sharing will help regulators, industry, and clinicians who must track the safety and effectiveness of these therapies over time.
In 2021, the ASH Research Collaborative® partnered with the Innovative Genomics Institute, the U.S. Food and Drug Administration (FDA), and other stakeholders to take the first step toward preparing for this future. Participants in the “Accelerating Innovations for Sickle Cell Disease with Real-World Evidence” initiative recommended data to collect and methods to coordinate clinically relevant and reliable real-world data for genomic therapies for genetic blood disorders, particularly SCD.
The recommendations of this initiative are expected to be applicable to all hematologic diseases that will eventually be represented in the Data Hub.
Final Recommendations:
- The Coordinated Registry Network Work Group’s final report can be found here.
- The Genomic Therapies Work Group’s final report can be found here.
Meeting materials are posted on this page for all interested parties to review.
Roundtable Meetings & Work Groups
The first roundtable was held in March 2021 to discuss the FDA’s growing reliance on real-world evidence in regulatory decisions. The group discussed examples of how coordinated registry networks have used real-world data insights that led to improved safety, efficacy, and expanded uses. The urgency of harmonizing data collection to better understand new genomic therapies was also shared.
Second Stakeholder Roundtable Meeting
The second roundtable was held in November 2021. During the roundtable, the Coordinated Registry Network and Genomic Therapies work groups reported their findings and recommendations for next steps. Attendees provided feedback that helped shape the final reports outlining next steps.
The initiative’s executive committee launched two work groups to examine real-world evidence generation in the following settings:
Coordinated Registry Network Work Group
The Coordinated Registry Network work group explored the role of coordinated registry networks for patient-level data aggregation in hematologic conditions.
A coordinated registry network in this context links traditional clinical registries to other data sources (i.e., claims, electronic health records, and data collected via apps or remote monitoring).
Collecting data from these sources may increase utility of the data, reduce costs, and better reflect patients’ experiences compared to traditional methods.
The Coordinated Registry Network Work Group’s final report can be found here.
Genomic Therapies Work Group
The Genomic Therapies work group identified and sought consensus on data points that should be collected and procedures and assays to be used to generate actionable, regulatory-grade real-world evidence for genomic therapies.
The Genomic Therapies Work Group’s final report can be found here.
Additional Information
Committees
Executive Committee Members
The purpose of the Executive Committee is to provide strategic direction for the two expert stakeholder work groups. The Executive Committee advised on the selection of work group membership and was responsible for reviewing, commenting, and approving the work group reports.
| Name | Organization |
| Co-Chair: Bill Wood, MD, MPH | UNC-Chapel Hill, ASH RC |
| Co-Chair: Fyodor Urnov, PhD | IGI, UC Berkeley |
| Alexis Thompson, MD, MPH | Lurie Children’s Hospital, ASH RC |
| Ann Farrell, MD | FDA (CDER) |
| Carlton Haywood Jr., PhD, MA | Johns Hopkins Berman Institute of Bioethics |
| Danica Marinac-Dabic, MD, PhD, MMSc, FISPE | FDA (CDER) |
| Donna Neuberg, ScD | Dana-Farber Cancer Institute, ASH RC |
| Gregory Pappas, MD, PhD | FDA (CBER) |
| Jane Hankins, MD, MS | St. Jude Children’s Research Hospital, ASH RC |
| Jennelle Stephenson | Self |
| Kathleen Hewitt, DNP, RN | ASH RC |
| Ken Taymor, JD | IGI, UC Berkeley |
| Michelle McMurry-Heath, MD, PhD | BIO |
| Peter Marks, MD, PhD | FDA (CBER) |
| Vence L. Bonham Jr, JD | National Human Genome Research Institute |
Coordinated Registry Network (CRN) Work Group Members
The CRN Work Group developed a report with recommendations on the creation of a SCD CRN useful to the ASH RC and Executive Committee.
| Name | Organization |
| Co-Chair: Gregory Pappas, MD, PhD | FDA (CBER) |
| Co-Chair: Jane Hankins, MD, MS | St. Jude Children’s Research Hospital, ASH RC |
| Ajai Chari, MD | Mount Sinai |
| Alexis Thompson, MD, MPH | Lurie Children’s Hospital, ASH RC |
| Allison King, MD, MPH, PhD | Washington University, ASH RC |
| Andreas Schick, PhD | FDA (CDER) |
| Angelo DeClaro, MD (reviewer) | FDA (CDER) |
| Ann Farrell, MD (reviewer) | FDA (CDER) |
| Art Sedrakyan, MD, PhD | Cornell University |
| Barbara Moehring, PharmD, MBA | Global Blood Therapeutics |
| Bill Wood, MD, MPH | UNC-Chapel Hill, ASH RC |
| Bindu George, MD (reviewer) | FDA, MM |
| Charles Quinn, MD, MS | Cincinnati Children’s Hospital |
| Danica Dabic-Marinac, MD, PhD, MMSc, FISPE | FDA (CDER) |
| David Wormser, PhD, MSc, MPhil | Novartis |
| Emily Tucker, MS | ASH RC |
| Fyodor Urnov, PhD | IGI, UC Berkeley |
| Ken Anderson, MD | Dana-Farber Cancer Institute, ASH RC |
| Louis Jacques, MD | ADVI |
| Marcus Droege, PhD, MBA | bluebird bio |
| Nicole Gormley, MD (reviewer) | FDA (CDER) |
| Payal Desai, MD | Ohio State University |
| Saad Usmani, MD | Memorial Sloan Kettering |
| Yelena Yesha, PhD | University of Miami |
Genomic Therapies Work Group Members
The Genomic Therapies Work Group prepared a white paper for the genomic therapies field, as well as recommendations to the ASH RC and FDA for long-term follow-up and RWE guidance.
| Co-Chair: Fyodor Urnov, PhD | IGI, UC Berkeley |
| Co-Chair: Donna Neuberg, ScD | Dana-Farber Cancer Institute, ASH RC |
| Bill Wood, MD, MPH | UNC-Chapel Hill, ASH RC |
| Chrystal Louis, MD, MPH | CRISPR Therapeutics |
| Cindy Dunbar, MD | NHLBI, ASH |
| Daniel Bauer, MD, PhD | Boston Children’s Hospital |
| David Wormser, PhD | Novartis |
| Eric Smith, MD, PhD | Dana-Farber Cancer Institute |
| Erica Esrick, MD | Boston Children’s Hospital / Dana-Farber Cancer Institute |
| Hans-Peter Kiem, MD, PhD | ASGCT VP |
| Jason Fontenot, PhD | Sangamo Therapeutics |
| John Tisdale, MD | NHLBI |
| Kathleen Hewitt, DNP | ASH RC |
| Ken Taymor, JD | IGI, UC Berkeley |
| Larissa Lapteva, MD | FDA (OTAT, CBER) |
| Lea Witkowsky, PhD | IGI, UC Berkeley |
| Lisa Michaels, MD | Editas Medicine |
| Mark Walters, MD | UCSF Benioff Children’s Hospital, Oakland |
| Matthew Porteus, MD, PhD | Stanford School of Medicine |
| Mitchell Weiss, MD, PhD | St. Jude Children’s Research Hospital |
| Myriam Armant, PhD | Boston Children’s Hospital |
| Petros Giannikopoulos, MD | IGI, UC Berkeley |
| Philip Gregory, PhD | bluebird bio |
| Punam Malik, MD | Cincinnati Children’s Hospital |
| Samantha Maragh, PhD | NIST |
| Sean Burns, MD | Intellia Therapeutics |
| Shengdar Tsai, PhD | St. Jude Children’s Research Hospital |
Meeting Materials
Useful Readings
ASH RESEARCH COLLABORATIVE
The ASH Research Collaborative (ASH RC) is a non-profit organization established by the American Society of Hematology (ASH) in 2017 to improve the lives of people affected by blood diseases by fostering collaborative partnerships to accelerate progress in hematology. Learn more about ASH RC