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    Burden of illness of progressive familial intrahepatic cholestasis in the US, UK, France, and Germany: study rationale and protocol of the PICTURE study.

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    Authors
    Ruiz-Casas, Leonardo
    O'Hara, Sonia; orcid: 0000-0002-9119-8336
    Mighiu, Claudia
    Finnegan, Alan
    Taylor, Alison
    Ventura, Emily
    Dhawan, Anil
    Murray, Karen F
    Schattenberg, Jorn; orcid: 0000-0002-4224-4703
    Willemse, Jose
    Karakaidos, Melanie
    Brrang, Harpreet
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    Publication Date
    2021-01-07
    
    Metadata
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    Abstract
    : Progressive familial intrahepatic cholestasis (PFIC) is an ultra-rare disease with a considerable burden on pediatric patients and their caregivers, impacting quality of life (QoL). The mortality rates highlight a significant need for efficacious treatments. Real-world data on associated costs and QoL are needed to gauge the potential impact of new pharmacological treatments. : Clinical and socio-economic burden of PFIC on patients/caregivers, health systems, and society will be assessed. Patient/caregiver- and physician-level retrospective cross-sectional data will be collected from the US, UK, France, and Germany, for PFIC types 1, 2, 3. A representative sample of physicians will provide clinical and resource utilization information using an electronic Case Report Form (eCRF). Patient/caregiver surveys will collect socio-economic and QoL data, enabling assessment of PFIC impact on QoL. Mean costs (direct medical/non-medical, indirect) will be calculated. The study materials were reviewed by medical professionals and patient representatives and received ethical approval from the University of Chester. : The study aims to reveal the unmet medical need, disease burden, resource utilization, and costs of PFIC, to raise awareness with policymakers and healthcare professionals, and provide support for the patient/caregiver community. As novel PFIC therapies recently emerged, this study will yield quantifiable data for health technology assessments.
    Citation
    Expert review of pharmacoeconomics & outcomes research, page 1-7
    URI
    http://hdl.handle.net/10034/624191
    Type
    article
    Description
    From PubMed via Jisc Publications Router
    Publication status: aheadofprint
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