Rare Daily Staff
Global Blood Therapeutics will provide a grant of $400,000 to the nonprofit ASH Research Collaborative in support of its Data Hub Sickle Cell Disease program, which seeks to advance research and outcomes for people living with sickle cell disease by expanding its current data collection of real-world, research-grade data to include more than half of people living with SCD in the United States.
Sickle cell disease (SCD) is a lifelong inherited rare blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body. Due to a genetic mutation, individuals with SCD form abnormal hemoglobin known as sickle hemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickled – deoxygenated, crescent-shaped and rigid. The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.
Sickle cell disease affects more than 100,000 people in the United States, an estimated 52,000 people in Europe, and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa. It also affects people of Hispanic, South Asian, Southern European, and Middle Eastern ancestry.
“We have a shared mission with GBT to help transform the treatment of sickle cell disease,” said Martin Tallman, of Memorial Sloan Kettering Cancer Center, who serves as president of the ASH Research Collaborative and the American Society of Hematology. “Working closely with the SCD community, the ASH RC is committed to efficiently capturing longitudinal real-world data to better understand the natural history of sickle cell disease and to create a national repository of high-quality data to accelerate research and enhance clinical care. We aim to give the SCD community a meaningful voice in the creation of one of the world’s largest repositories of real-world, research-grade data in SCD. We appreciate GBT’s support to help us work toward improving care and outcomes for our patients with this rare disease.”
The ASH RC fosters collaborative partnerships to advance progress in hematology and improve the lives of people affected by blood diseases. The foundation of the ASH RC is its Data Hub, a multi-stakeholder, real-world data program that facilitates the exchange of information on hematologic conditions to support scientific inquiry, discovery, and quality improvement. Nearly 30 U.S. clinical sites providing SCD care are currently participating in the Data Hub and are submitting HIPAA-compliant data representing 6,000 individuals living with SCD. The Data Hub is actively enrolling up to an additional 110 clinical sites, and the ASH RC believes it is well-positioned to capture data on more than 50,000 people in the U.S. living with SCD.
GBT is the first biopharmaceutical company to provide the ASH RC with financial support through a grant that will help onboard clinical sites and integrate their data to the Data Hub. These sites are organized across 19 consortia also enrolled with the ASH RC SCD Clinical Trials Network. The purpose of the SCD Clinical Trials Network is to accelerate research that advances treatment and care of those affected by SCD.
The Data Hub can collect a wide variety of data, including electronic medical record data, clinical and laboratory data, genomic or molecular correlates, patient-reported outcomes, and aggregated population data. These data are obtained from inpatient and outpatient clinical sites, industry or government datasets, registries, other U.S. or international sources, and directly from patients. By using state-of-the-art technology to automate data clinical capture and linkages to other data sources, the Data Hub is focused on minimizing data capture burden. Patient information collected in the Data Hub complies with national and local privacy laws and regulations to protect patient privacy.
“Comprehensive real-world data can empower people with sickle cell disease and their healthcare providers to make decisions regarding their care, while also guiding the development of new treatments and guidelines that we hope will transform SCD into a well-managed disease,” said Kim Smith-Whitley, executive vice president and head of research and development at GBT. “There has never been a more important time to advance research initiatives with the potential to improve care and address long-standing gaps in health equity for the SCD community.”
Photo: Kim Smith-Whitley, executive vice president and head of research and development at GBT