PCORI convened a multi-stakeholder workshop to identify comparative clinical effectiveness research questions to fill evidence gaps and improve outcomes for individuals with sickle cell disease (SCD), with a focus on care transitions and pain management. To ensure that we focused on important evidence gaps that can influence clinical practice, we asked representatives of a wide range of stakeholder groups, including patients, caregivers, clinicians, industry, payers, and researchers to participate in this discussion.
Sickle cell disease (SCD) is a chronic genetic disorder affecting the body’s red blood cells. It is estimated that between 70,000 and 100,000 Americans, predominately African Americans, have SCD. This disorder induces a series of disease-related complications, such as acute chest syndrome, pain crises, and stroke. These patients are also prone to lack of care coordination and difficulties when transitioning from childhood to adulthood.
Currently, practices for the treatment of SCD are being used with limited evidence, leaving health care professionals and patients with little information to make informed health care decisions regarding treatment. Thus, well-designed studies are desperately needed.
In light of this, the Addressing Disparities program, in conjunction with other programs at PCORI, is working on identifying comparative effectiveness research questions to fill evidence gaps and improve outcomes for individuals with sickle cell disease, with a focus on care transitions and pain management. We are excited about the possibility of potentially focusing PCORI funding on this important topic. To ensure that we focused on important evidence gaps that can influence clinical practice, we convened a workgroup of experts, patients, and stakeholders to achieve the following:
- Obtain patient, researcher, and other stakeholder input;
- Confirm the importance and timeliness of particular research topics on SCD;
- Refine identified high-impact comparative effectiveness research questions that will result in findings that are likely to endure and that have not been or are not currently being studied;
- Understand the potential for research to lead to rapid improvement in practice, decision-making, and outcomes; and
- Seek consensus on identified research gaps and specific comparative effectiveness research questions that address those gaps.
In-person attendance was by invitation only, but the public was welcome to listen in via teleconference/webinar.
PCORI Workshops Introduction (9:00 am - 9:30 am ET)
Main Meeting Teleconference/Webinar (9:45 am - 5:00 pm ET)
- Topic Brief
- Consolidated CER Themes and Questions for Breakout Groups
- Submitted Stakeholder Questions
- Participants List
- Introduction Presentation Slides
- Main Presentation Slides
- Workshops/Breakout Groups Chair Biographies
- Meeting Summary
Teleconference Audio Recordings
Breakout One: Care Transitions
Breakout Two: Pain Management