Project Summary

Background: After decades of relatively little progress being made for sickle cell disease (SCD), several new therapies have been introduced in recent years. As new treatments become available, patients and stakeholders have faced new challenges measuring the "value" of treatments to patients. Currently, there is a paucity of data concerning what constitutes value to SCD patients and how to best measure and include elements of value in decision making. Current data lacks representative diversity and fails to capture real-world factors that matter to patients. Thus, there is a critical need to identify and assess core elements of value that can be integrated into methods and measures of research and value assessment to improve health equity as core component of healthcare decision making for SCD.

Proposed Solution to the Problem: Sick Cells proposes to convene a group of stakeholders to drive consensus on important impacts to measure value in SCD. Through this convening, stakeholders will foster new partnerships and facilitate discussion around development of priority elements, methods, and engagement plans for future patient-centered outcomes research (PCOR), clinical comparative effectiveness research (CER), and value assessments.

Objectives: The primary goal of this project is to develop consensus recommendations on how to define, collect, and use data that represent core elements of value for SCD. Specific project objectives include:

  • Recruit diverse inputs from representative patient populations encompassing the heterogeneity of SCD
  • Prioritize impacts and outcomes to measure value across stakeholder groups
  • Evaluate current measures, methods, and data sources that address patient-important elements of value 
  • Establish guidance for research and value assessments to prioritize health equity
  • Increase transparency in decision-making

Activities: Roundtable One will assemble stakeholders and facilitate a virtual dialogue on the full range of impacts and outcomes patients and other stakeholders report as important. Roundtable Two will assemble stakeholders and facilitate a virtual dialogue to evaluate needs and share principal ways to address key data gaps and support equity. Following the roundtables, Sick Cells, in partnership with USC HUGS and the CHOICE Institute, will establish guidance for future research and value assessments in SCD. A meeting summary and key principles document will be published and shared publicly.

Projected Outcomes and Outputs:
Short-term outcomes during the project period include to publish key principles for (1) improving measurement of patient perspectives and (2) supporting equity in SCD research and decision making.

Medium-term outcomes (0-2 years post-project period) include: to develop PCOR/CER that reflect appropriate end points and utilize patient-centric methods to support equity.

Long-term outcomes (3+ years post-project period) include: to establish health equity as a priority and foundational element of research, value assessments, and decision making in SCD.

Patient and Stakeholder Engagement Plan: Sick Cells seeks to include a broad coalition of stakeholders through two distinct groups: (1) Community Advisory Board (CAB) and (2) Steering Committee. The CAB, composed of non-profit leaders from within the SCD community, will serve as community experts for this project.  The Steering Committee, composed of diverse stakeholder representatives acting as key project advisors, will play an active role in creating agenda items, planning discussions, and facilitating outputs.

Project Collaborators: With leadership from the Sick Cells, and through collaboration with the University of Southern California (USC) Hematology Utilization Group Studies (HUGS) and The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute at University of Washington (UW) School of Pharmacy, this project will establish action items for research and value assessments to capture cost and other burdens that matter to SCD patients and caregivers. 

Project Information

Ashley Valentine, BA, MS, Mres
Maggie Jalowsky, BS
Sick Cells

Key Dates

May 2023


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