coreSCD: multi-stakeholder consensus on core outcomes for sickle cell disease clinical trials

Background: With the dramatic increase in the pipeline for new sickle cell disease (SCD)therapies in recent years, the time is ripe to ensure a robust body of evidence is available for decision making by regulators, payers, clinicians, and patients. Harmonization of the outcomes selected across interventional trials enables optimal post-trial appraisal and decision making through valid pooled analyses and indirect comparisons. We employed a structured, multi-stakeholder consensus process to develop core outcome sets (COS) for use in clinical trials of SCD interventions.

Methods: CoreSCD utilized a modified Delphi method adapted from the standards recommended by the Core Outcome Measures in Effectiveness Trials (COMET) Initiative. An initial list of candidate outcomes was developed through a targeted literature review and input from an 11-member advisory committee. A 44-member multi-stakeholder Delphi Panel was established and included patients and family members, advocates, clinicians, researchers, payers, health technology assessors, representatives from government agencies, and industry representatives. Patients/advocates comprised 25% of the Delphi Panel and orientation and training was provided prior to the consensus process to ensure all were prepared to participate meaningfully. Panelists completed three rounds of an online survey to rate the importance of candidate outcomes for inclusion in the COS. Summary data was provided between each voting round and an in-person consensus meeting was held between the second and third round of voting. Consensus rules were applied following each round of voting to eliminate outcomes that did not meet predetermined criteria for retention.

Results: Consensus was reached for two core outcome sets. The final COS for trials of disease-modifying therapies includes ten outcomes and the COS for trials of acute interventions includes six outcomes. Both core sets include clinical outcomes as well as outcomes related to functioning/quality of life, resource utilization, and survival/mortality.

Conclusions: Use of the COS in clinical development programs for SCD will help to ensure that relevant, consistent outcomes are available for decision making across the product lifecycle.

Contributors

Ellen Tambor , Matoya Robinson, Lewis Hsu, Hsing-Yuan Chang and Jennifer Al Naber on behalf of the coreSCD Panel

Publication

Journal: BMC Med Res Methodol
Volume: 21
Issue: 219
Pages: -
Year: 2021
DOI:

Further Study Information

Current Stage: Completed
Date: March 2019 - 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: 0 - 120

Sex: Either

Nature of Intervention: Drug

Stakeholders Involved

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

Study Type

- COS for clinical trials or clinical research

Method(s)

- Consensus conference
- Consensus meeting
- Delphi process
- Interview
- Literature review
- Survey

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.

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