PCORI Engagement Award: Defining Value and Supporting Equity in Sickle Cell Disease
As more treatments for SCD become available, the need to measure the true value of treatments to patients becomes more important. Currently, there is insufficient data concerning what constitutes value to SCD patients and how to measure value in decision making.
Sick Cells worked in collaboration with the University of Southern California Hematology Utilization Group Studies (USC HUGS) and the Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute at University of Washington School of Pharmacy to identify and assess core elements of value to be incorporated into SCD research measures.
The purpose of this project was to establish action items for research and value assessments to capture cost and additional burdens that matter to patients living with sickle cell disease (SCD) and caregivers. Study objectives included:
- Recruiting diverse inputs from patient populations that are representative of the community
- Prioritizing impacts and outcomes to measure value for SCD across stakeholder groups
- Evaluating current measures, methods, and data sources that address patient-important elements of value for SCD
- Establishing guidance for research and value assessments to prioritize health equity for SCD
June 1, 2022 to May 31, 2023
Scope & Methods
This collaborative project was led by a steering committee and a community advisory board. Both bodies provide relevant expertise and insights into the project design and management.
- The steering committee was composed of diverse stakeholder representatives that acted as project advisors and played an active role in creating agenda items, and planning discussions. The steering committee met monthly.
- The community advisory board (CAB) was composed of non-profit leaders within the SCD community who served as community experts. The CAB met five times during the duration of the project.
This project consisted of two roundtables involving various stakeholders and experts in SCD.
- On November 16, 2022, Roundtable One was assembled to facilitate a dialogue of important impacts and outcomes according to patients and stakeholders.
- The primary goal was to learn what data is available and what work has been done.
- There were a total of 50 participants, including 4 SCD advocates, 7 caregivers, 17 individuals from a government agency/industry, 9 researchers, and 13 SCD warriors.
- On March 30, 2023, Roundtable Two focused on Prioritizing Outcomes and Measures for Sickle Cell Disease.
- Individuals were asked to rank disease impacts to determine their importance across the SCD community.
- More than 40 participants made up of patients, caregivers, and stakeholders participated in the second roundtable.
Short-term outcomes will be published in June 2023 and will include a project summary and key principles for (1) improving the measurement of patient perspectives and (2) supporting equity in SCD research and decision-making.
This convening is funded through a Patient-Centered Outcomes Research Institute® (PCORI®) Eugene Washington PCORI® Engagement Award (#EASCS-24293). Click the button below to view more information on the Sick Cells PCORI Engagement Award