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The ASH Research Collaborative (ASH RC) was established by ASH in 2018 with the goal of fostering collaborative partnerships to accelerate progress in hematology and improve lives of those affected by blood diseases. The ASH RC has two main components: the Data Hub and the Sickle Cell Disease (SCD) Clinical Trials Network (CTN). For an update on the Data Hub, visit page B-36 of the Saturday issue of ASH News Daily. The SCD CTN, launched this year, strives to improve outcomes for individuals with SCD by expediting SCD therapy development and facilitating innovation in clinical trials research.

ASH News Daily spoke with Chair of the SCD CTN Oversight Committee Charles S. Abrams, MD, of the University of Pennsylvania and Children’s Hospital of Philadelphia, about the significance of the clinical trials network for the SCD community and the progress of this initiative. Dr. Abrams explained that after a meeting with broad group of stakeholders, including people living with SCD, the overwhelming consensus was that a clinical trials network was needed to promote progress against this disease, and that ASH had the convening power to help foster interactions among scientists, clinicians, patients, and trial sponsors in order to make it happen.

Historically, there have been numerous barriers that have prevented successful clinical trials in SCD, including a shortage of primary investigators, poor coordination and lack of data sharing among sites, flawed study designs, and not enough enrolled patients. Dr. Abrams explained that the ASH RC Clinical Trials Network for sickle cell is a collaborative effort that addresses these barriers in a number of ways. It will match trial sponsors with sites, facilitate the recruitment of eligible patients, optimize trial design, and ensure a coordinated approach. According to Dr. Abrams, “Our long-term vision is that this is going to lead to FDA approval of drugs more rapidly, and we’re going to be able to improve the lives of patients with sickle cell.”

One of the efficiencies of the Network is that the ASH RC Data Hub will serve as the central data repository for all of its sites. Dr. Abrams noted that this will not only enable researchers to study the natural history of the disease, but “when clinical trials become available, pharmaceutical companies and scientists around the world will be able to easily find patients who fit the criteria for their trials and will lead to a more efficient and, we hope, rapid process for enrolling patients. In so many ways, the Data Hub and CTN are complementary to each other.”

When asked what makes the CTN different from other networks that came before it, Dr. Abrams cited the fact that “patient engagement is a critical component of the process to ensure that the patient perspective is considered every step of the way.” To that end, over the past several months, the ASH RC hosted SCD community engagement workshops in eight cities across the United States, involving roughly 500 members of the SCD community, to learn more about what areas of research individuals living with SCD would most like to see come to fruition. “The insights gained from these sessions has been immeasurable and will help us to prioritize the areas of research that will be most valued by the SCD community.” He noted that there are plans to publish the findings from these workshops in early 2020 to help inform future efforts.

Dr. Abrams is hopeful about this initiative as well as the growing interest in it. “When we first issued the request for proposals from potential sites, we debated what the ideal size would be to start.” However, it quickly became clear there was enthusiasm from many different stakeholders. “We’ve met with the National Institutes of Health and with the U.S. Food and Drug Administration on several occasions. We have met with patient groups, and more recently, we’ve been engaging local sites that potentially would be members of the network,” he explained. He added that there has been strong interest by biotech and pharmaceutical companies. “It’s been a joy to see that people are all stepping forward and saying, ‘We want to help with this,’” Dr. Abrams commented.

Currently, there are 26 sites that have applied to be part of the Network. Dr. Abrams explained that the ASH RC is committed to working with all 26 sites as they move through the multi-step process to acceptance, which includes a site visit and contributing data to the ASH RC Data Hub. “Ultimately we look forward to welcoming as many sites as are able to demonstrate they can meet the high standards required to be part of the Network,” he said. “We have a number of sites that are moving quickly through the process and hope to make our first announcement in early 2020.”



Learn More

To hear more details about the current state of the SCD CTN from Dr. Abrams himself, attend the ASH RC Update Session taking place Monday at 6:15 p.m. (Room W207BC, Orange County Convention Center – map it). Visit the ASH RC Booth in ASH Central for more information and resources.

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