Sickle cell disease (SCD) refers to a group of inherited red blood cell disorders, affecting approximately 90,000-100,000 Americans. The defect causes the oxygen-carrying red blood cells to be crescent shaped, like a sickle. SCD is a multisystem disease, associated with episodes of acute illness and progressive organ damage, and it is one of the most common severe monogenic disorders worldwide. SCD is often characterized by acute and chronic complications that progress with increasing age, and it negatively impacts the patient’s quality of life, leading to chronic morbidity resulting in high utilization of healthcare resources and reduced survival.
Improved diagnostics and therapeutic approaches have greatly increased life expectancy of individuals with SCD in the past 40 years, extending from 14 years to 40-60 years. While studies suggest that limited present therapies are effective in ameliorating some of the disease effects, there are barriers to their utilization, as well as adherence issues, which remain to be addressed.
Patient-centered outcomes research and comparative effectiveness research (PCOR/CER) are needed on multiple fronts for SCD. These would include understanding the barriers to treatment utilization and how they may be overcome; refinement of current therapies; exploration of new therapeutic approaches; patient outcomes measures for SCD; and new patient-centered partnerships with clinicians, academic institutions, industry, and foundations.
The Sickle Cell Disease Association of America (SCDAA) proposes to develop the National Sickle Cell Advocate Network (NSCAN), a national advocate network of SCD patients, families, caregivers, and other stakeholder experts that can partner with clinicians and researchers to design, implement, and eventually disseminate PCOR/CER projects that improve outcomes for patients with SCD. In addition, the founding members of the NSCAN will serve as mentors/peer facilitators to further expand the network and seek to fully participate in national and local committees at the National Institutes of Health (NIH), the Agency for Healthcare Research and Quality (AHRQ), the Centers for Disease Control and Prevention (CDC), the Health Resources and Services Administration (HRSA), the Food and Drug Administration (FDA), the United States Agency for International Development (USAID), the Patient-Centered Outcomes Research Institute (PCORI), local and state department of health agencies, and other organizations to share their experience with SCD and represent the patient for better treatment and further research.
NSCAN, with the assistance of an Expert Advisory Committee, will develop and implement a patient-centered training curriculum and mentor program in an effort to:
- Receive updates from clinicians and patients regarding therapeutic options for SCD
- Determine/develop relevant patient outcome measures for SCD
- Create partnerships with academic institutions, industry, and foundations to advance PCOR/CER projects
- Increase SCD patient partnering with clinicians on PCOR/CER projects. Upon completion of a developed curriculum and implemented program, 25 trained patient/parent facilitators will expand the network in their respective areas/states to 50 additional network participants/facilitators, an effort to educate over 625 advocates.
Project Collaborators: SCDAA has enlisted several collaborators to ensure the success of this project. Such collaborators include: The SCDAA Medical Advisory Council, 42 community-based member organizations, The New Jersey Sickle Cell Association, Inc., and Leidos, Inc.
Project Resource: Advisory Committee Call Notes November 2016