Project Summary

Background: Sickle cell disease (SCD) is a genetic blood disorder that leads to clinical complications that may impact physical and social well-being. Health scientists have had significant success with clinical and community interventions to treat SCD. However, incongruencies exist between outcomes important to patients and data collected in research studies. There is a critical need to identify SCD patient-centered priorities by identifying impacts on patients and caregivers and developing a core set of priorities to disseminate to clinicians, researchers and stakeholders who implement patient-centered outcomes research and comparative clinical effectiveness research (PCOR/CER) and programs aimed at improving health outcomes for individuals with SCD. 

Proposed Solution to the Problem: The project team will use the National Health Council’s Patient-Centered Core Impact Sets (PC-CIS) Blueprint to guide the prioritization of a list of short- and long-term impacts from patient and caregiver perspectives to inform future PCOR/CER. The PC-CIS Blueprint is a six-step process: 1) planning, 2) gathering impact data from patients, 3) prioritization by patients, 4) gathering impact data from stakeholder groups, 5) prioritization by patients/others and 6) assembling the PC-CIS. 


  1. Develop a comprehensive list of existing patient-centered  outcomes (PCOs) used in healthcare services and PCOR/CER for patients with sickle cell disease and caregivers
  2. Identify healthcare services outcomes preferred by patients with SCD and caregivers as well as the underlying values that are the foundation of these preferences overall and by domains
  3. Create a comprehensive summary of the PCOs valued by patients and caregivers, comparing patient-preferred PCOs with provider PCOs; distribute the summary through the project team’s dissemination networks to inform future PCOR/CER


  • Preplanning: A working group of researchers, a patient investigator and stakeholders will plan the convening sessions to review and document existing provider PCOs used in healthcare services and CER for patients with SCD and caregivers. 
  • Convening 1 (Virtual): Present the comprehensive summary of PCOs and elicit input to generate alignment with PCOs valued by patients with a lived SCD experience and caregivers. Convening 1 will be two half-day virtual forums consisting of a patient panel, small group discussions and PCOR/CER outcomes prioritization.
  • Post Convening 1: Provide convening summary available for public comment. The public comment period will remain open for 30 days. The team will invite SCD community-based organizations to review the document and offer contributions. 
  • Convening 2 (Virtual): Present the vetted comprehensive list of patient- and caregiver-preferred PCOs, underlying values and alignment with provider PCOs to inform future PCOR/CER. 

Projected Outcomes and Outputs: 

Short-term outcomes during the project period include:

  1. Successful implementation of a convening to identify SCD healthcare services outcomes preferred by patient and caregivers, as well as the underlying values that are the foundation of these preferences overall and by domains to inform future PCOR/CER
  2. Development of a comprehensive summary of the PCOs valued by patients and caregivers, comparing patient-preferred PCOs with provider PCOs

Outputs are meeting materials, a roadmap of clinician- and patient-aligned PCOR/CER priorities, a summary report, and dissemination of priorities.

Medium-term outcomes (0-2 years post project period) include:

  1. Development of a roadmap of clinician- and patient-aligned PCOR/CER priorities

Long-term outcomes (3+ years post project period) include:

  1. Dissemination of the roadmap of clinician- and patient-aligned PCOR/CER priorities
  2. Integration of patient-aligned PCOR/CER priorities into CER

Patient and Stakeholder Engagement Plan: To engage patient stakeholders, the project team will use the Framework for Authentic Engagement in Multi-Stakeholder Initiatives: 1) defining engagement, 2) developing an engagement purpose statement, 3) moving up the community engagement continuum, 4) amplifying the community voice, 5) addressing health equity, 6) aligning metrics with community values, 7) assessing when engagement harms communities, and 8) reflection and assessment as a way forward with engagement.

Project Collaborators: The leadership team will consist of two project leads and one patient investigator. The project team will partner with the Sickle Cell Consortium as the lead community partner. The team will then establish a workgroup of community stakeholders and patients with lived SCD experience. This working group will collaborate as equal partners in the development, implementation and evaluation of the convening and dissemination.

Project Information

Tilicia Mayo-Gamble, MA, MPH, PhD
Raymona H. Lawrence, DrPH, MPH
Georgia Southern University Research and Service Foundation, Inc.

Key Dates

June 2024


Project Status
State State The state where the project originates, or where the primary institution or organization is located. View Glossary
Last updated: April 3, 2024