Introduction
Treatment options are expanding for von Willebrand disease (VWD). A core outcome set (COS)—a minimum set of agreed-upon outcomes to be used in every clinical trial for a given condition—provides guidance on which outcomes are most important to measure to ensure necessary data is collected for a variety of stakeholders and enable comparison across products and trials.
Aim
coreVWD aimed to develop a COS for trials for prophylaxis and perioperative treatments for VWD.
Methods
A modified Delphi consensus process was used to condense/prioritize a long list of potential outcomes. Over three Delphi rounds, a multi-stakeholder panel (patients, clinicians, pharmaceutical company representatives, HTA organizations, payer, and government organization representatives) rated each outcome from 1 (not important to include in a COS) to 9 (essential to include). Outcomes were eliminated or retained based on pre-determined criteria; a special provision to elevate patient priorities was included. An in-person consensus meeting was held after Delphi round 2.
Results
Thirty-nine panellists participated. The final COS for prophylaxis treatment included 18 outcomes, seven of which are part of a special subset selected for women, girls and people with the potential to menstruate. There were 11 outcomes in the final perioperative branch COS. Six outcomes overlapped both COS.
Conclusions
The coreVWD COS represents a consensus list of outcomes for clinical trials for both factor and non-factor VWD therapies. These outcomes will be useful across the lifecycle of a product, from clinical development through regulatory and market access phases and into patient-provider decision-making.
Elizabeth Clearfield, Benjamin Kim, Sarah Ford, Nathan T. Connell, Maria E. Santaella, Michelle Lavin, Manon E. L. Degenaar-Dujardin, Emily Ayoub, Veronica H. Flood, Dawn Rotellini, Mark W. Skinner, Paula James, the coreVWD Panel
Disease Category: Blood disorders
Disease Name: congenital bleeding disorders, Haemophilia/hemophilia and other bleeding disorders
Age Range: 18 - 120
Sex:
Nature of Intervention: Any
- Clinical experts
- Patient/ support group representatives
- Pharmaceutical industry representatives
- Regulatory agency representatives
- COS for clinical trials or clinical research
- Consensus meeting
- Delphi process
- Literature review
- Survey