In a significant advancement for von Willebrand Disease (VWD) research and treatment, an international team of experts, including patients, clinicians, and researchers, has developed the first-ever core outcome set (COS) for VWD clinical trials. The initiative, known as coreVWD, establishes standardized outcomes that should be measured in all clinical trials for both prophylaxis and perioperative VWD treatments. The project, which ran from May to December 2023, brought together 39 participants, including 14 patients, 11 clinicians, and representatives from pharmaceutical companies and healthcare organizations. Read the Full Article Here
"This is a pivotal moment for the VWD community," explains Dawn Rotellini, Chief Operating Officer of the National Bleeding Disorders Foundation (NBDF), who participated in the project. "As we enter an era of expanding treatment options, having standardized outcomes will ensure that clinical trials generate the data and ultimately the treatment that matters most to patients and healthcare providers."
The core outcome set includes 18 outcomes for prophylaxis treatment and 11 outcomes for perioperative treatment, with six outcomes overlapping between both types. Notably, the initiative placed special emphasis on women's health outcomes, recognizing the unique challenges faced by women with VWD. Seven specific outcomes related to women's health were included in the prophylaxis treatment set, addressing issues such as menstrual bleeding and pregnancy-related complications.
"VWD affects women and men differently, and historically, women's experiences have sometimes been overlooked," notes Maria E. Santaella, Vice President of Research of NBDF. "This core outcome set ensures that women's health concerns are properly represented in future clinical research."
The development of this COS comes at a crucial time, as new VWD treatments are being developed and existing treatments are expanding into new indications. The standardized outcomes will help researchers, regulators, and healthcare providers better evaluate and compare different treatment options. The next step will be to establish best practices for measuring these outcomes in clinical trials. The coreVWD initiative represents a collaborative effort to improve clinical research and ultimately enhance care for people living with VWD.
For more information about the coreVWD initiative or VWD resources, contact NBDF’s Research Department at research@bleeding.org.
Written by: Kyla Clark, Director of PR, Marketing and Communications
