Project Summary

Background: Hemophilia has a number of treatment options, and there is varied ability to compare outcomes across all of them. The inclusion of patients and community members as partners in developing patient-centered outcomes in hemophilia has been limited and may not represent patients’ priorities.

Proposed Solution to the Problem: In this project, patients, caregivers of patients, patient advocates, payers, pharmaceutical representatives and community members will be invited to be part of a Stakeholder Advisory Committee (SAC). This project will take place at the Hemophilia Treatment Center in Cleveland, Ohio.

Objectives: The aim of the SAC will be to identify patient-centered outcomes in hemophilia that can be used for patient-centered outcomes research/comparative clinical effectiveness research (PCOR/CER). The focus will be on patient outcomes of highest priority to the Committee.

Activities: After the SAC members have agreed to participate, there will be an orientation to current outcomes measured in research and CER in hemophilia. Over the course of three meetings and through Delphi-like surveys completed between meetings, the SAC will develop a list of patient-centered outcome priorities in hemophilia research. Additionally, committee members as well as clinician stakeholders who assist with facilitating the committee meetings will provide feedback between meetings. This feedback will be used by the project team to improve subsequent meetings.

Projected Outcomes and Outputs:

Short-term outcomes during the project period:

  1. Summary of a literature review on key outcomes used in treatments of hemophilia, challenges and opportunities. 

  2. Strategy for how to engage a SAC on a broad area of hemophilia treatment outcomes.; 

  3. List of patient-created outcome priorities that will be used to inform future PCOR/CER projects.

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

The team will develop consensus on patient-centered outcome  priorities for research investment in PCOR/CER in hemophilia and buy-in across stakeholders for approaches to research design.

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

In partnership with the SAC, the team will develop a framework for at least one research project addressing the priorities from this project. The team will also continue to work with the SAC while conducting that research project.

Patient and Stakeholder Engagement Plan: The clinical team members will reach out to their patients and caregivers of patients as well as ask the community advocacy organization to reach out similarly to invite patients to participate in the SAC. The team will also reach out to community and national advocacy organizations to participate in the SAC. The SAC will meet three times and complete surveys in between meetings. Throughout the project, SAC members and clinical stakeholders will give feedback to inform and improve the project and process.

Project Collaborators: University Hospitals Cleveland Medical Center and Case Western Reserve University are collaborating to implement this project.

Project Information

Sanjay Ahuja, MBA, M.D.
University Hospitals Cleveland Medical Center

Key Dates

November 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