Core Outcome Measures in Effectiveness Trials

coreSCD: Core Outcomes in Sickle Cell Disease

General Information

The goal of this project is to develop multi-stakeholder consensus on a core set of outcomes to be used in pivotal and post-approval clinical trials of drug therapies for Sickle Cell Disease (SCD). Consensus on a core set will be developed through an iterative survey and engagement process called a modified Delphi survey. Through this process, stakeholders gain mutual understanding and develop a shared sense of the most important outcomes to include in all studies, especially those outcomes reflecting the greatest concerns of patients. The Delphi will be conducted using methods and practices based on the COMET Initiative approach but adapted for robust multi-stakeholder participation and timelier project completion. While the current project scope is limited to developing consensus on what outcomes should be measured, a second phase of the project may be added to reach consensus on how the agreed-upon core outcomes should be measured.

This project will be conducted through the Center for Medical Technology Policy's Green Park Collaborative in partnership with the Sickle Cell Disease Association of America.

Further Study Information

Current Stage:
March 2019 - February 2020
Funding source(s):
The project will be funded through a pre-competitive collaborative model, with sponsorship fees provided by participating life sciences companies. Fundraising is currently underway.

Health Area

Disease Category
Blood disorders

Disease Name
Sickle Cell Disease

Target Population

Age Range


Nature / type of Intervention


Consensus conference
Consensus meeting
Delphi process
Literature review

The consensus process will begin with a targeted literature review and key informant interviews to determine the scope of the core outcomes set and develop a list of candidate outcomes. Following orientation, stakeholders will participate in 2-3 delphi survey rounds to reduce the candidate outcomes list. The reduced list will be discussed at an in-person consensus meeting followed by a final round of online voting.

Stakeholders Involved

Clinical experts
Consumers (caregivers)
Consumers (patients)
Governmental agencies
Patient/ support group representatives
Pharmaceutical industry representatives
Policy makers
Regulatory agency representatives

Study Type

COS for clinical trials or clinical research

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