While there are currently only two U.S. Food and Drug Administration (FDA)-approved drugs to treat the disease, there is a robust sickle cell disease (SCD) drug development pipeline poised to drive demand for SCD clinical trials to a new level, providing a prime opportunity to advance treatment and care of those affected by SCD.
At the same time, there are many challenges in conducting clinical trials for SCD, including a shortage of primary investigators, clinical trial sites, and enrolled patients; lack of access to a centralized data repository; poor coordination among sites; and flawed study designs. In addition, the voice of the patient has been largely absent in decisions about clinical trials in SCD, which has led to failures in patient enrollment and support.
The ASH Research Collaborative (ASH RC) SCD Clinical Trials Network will be launched with a mission of improving outcomes for individuals with SCD by expediting SCD therapy development and facilitating innovation in clinical trial research. It will provide the infrastructure for identifying patient cohorts for trials, matching trial sponsors with sites, facilitating recruitment of eligible patients, and ensuring optimally designed trials and an efficient, coordinated approach. Through patient engagement and optimized clinical trial execution, the Network will help bring new and more effective therapies to individuals with SCD.
We are seeking clinical research sites at institutions interested in joining this network of stakeholders with a shared commitment to:
- Forging new relationships with patients to increase their understanding of clinical trials and trust in SCD researchers
- Eliminating inefficiencies through the use of a centralized data repository (the ASH RC Data Hub) and institutional review board
- Focusing on the research opportunities that hold the most promise for patients.
The Network will promote quality, patient safety, and efficiency in SCD clinical trials by centralizing functions and sharing best practices. While traditional responsibilities for running a clinical trial will remain with the participating sites and industry, the Network’s activities will address many of the common barriers faced in clinical trials research:
- Patient engagement, education, and recruitment
- Matching sponsors with trial sites
- Ensuring an efficient coordinated approach to clinical trials research
The ASH RC Clinical Trials Network LOI submission deadline closed on January 31, 2019. Applications are being reviewed for their merit. Applicants can expect to receive a response in the Spring of 2019.
Former ASH President Alexis Thompson, MD, MPH, hosted a Q&A session on the SCD CTN during the 60th ASH Annual Meeting in San Diego. View the video below to learn more.