PO369: Introduction to the UK PIVOT-vWD Study: A direct to community, cross-sectional study to capture and quantify the impact, voice and outcomes of VWD

Abstract

Objectives There is a paucity of new evidence/literature on the clinical outcomes, healthcare resource use (HCRU), cost and the quality-of-life (QoL) impact on those with von Willebrand Disease (VWD). The PIVOT-VWD study aims to collect patient generated health data to quantify the impact, voice and outcomes of VWD in the UK. Specifically, in relation to the management of the condition including treatment preferences, symptomology and the impact upon work and daily activities, as well as QoL outcomes. Methods The study design is a bottom-up, cross-sectional methodology. Two survey questionnaires have been developed, one for adult (18+) respondents who will be self-reporting, and a proxy survey for those parents who answer on behalf of their child (under 18) or as a caregiver. Lived experience experts have provided review and input into the study design and survey questionnaire content. The cross-sectional survey includes questions about the individual with VWD focusing on patient characteristics, VWD background, symptomology, management, work & daily activities, quality of life, and perspectives on novel treatments. In addition, if the survey is completed by proxy, specific variables will be collected about the parent/caregiver. Any HCRU questions are captured retrospectively over the last 12 months to be able to estimate the direct healthcare and societal burden of disease. Results Data collection is currently on-going. Recruitment is supported by The Haemophilia Society who have utilised their network, social media accounts and mailing list to distribute invitations to members of the VWD community. The aim of recruitment will be to gather a target sample of 250 respondents based in the UK. The project may be expanded to other countries in the future. Conclusions Capturing the clinical & quality of life impact upon individuals with VWD is critical to supporting new and emerging therapies, which could provide direct benefits to those affected by the condition. In addition, preference research in health technology assessment (HTA) is becoming more relevant as HTA bodies widen their decision-making perspectives. Gathering the voice of the community leads to greater understanding of the priorities of the community, leading to more informed decision making and relevant treatments