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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.

Ruiz-Casas, Leonardo
; 
O'Hara, Sonia
; 
Mighiu, Claudia
; 
Finnegan, Alan
; 
Taylor, Alison
; 
Ventura, Emily
; 
Dhawan, Anil
; 
Murray, Karen F.
; 
Schattenberg, Jörn M.
; 
Willemse, Jose
... show 2 more
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Affiliation
HCD Economics, Madrid; HCD Economics, Daresbury; University of Chester; Children's Liver Disease Foundation; PFIC Advocacy and Resource Network; King's College Hospital; Cleveland Clinic Children's Hospital; University Medical Center of the Johannes Gutenberg-University; Dutch Liver Patient Association
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2021-01-07
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Health and Social Care
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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
Ruiz-Casas, L., O'Hara, S., Mighiu, C., Finnegan, A., Taylor, A., Ventura, E., Dhawan, A., Murray, K. F., Schattenberg, J. M., Willemse, J., Karakaidos, M., & Brrang, H. (2021). Burden of illness of progressive familial intrahepatic cholestasis in the US, UK, France, and Germany: Study rationale and protocol of the PICTURE study. Expert Review of Pharmacoeconomics & Outcomes Research, 21(2), 247-253. https://doi.org/10.1080/14737167.2021.1859371
Publisher
Taylor & Francis
Journal
Expert Review of Pharmacoeconomics & Outcomes Research
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URI
http://hdl.handle.net/10034/624191
DOI
10.1080/14737167.2021.1859371
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Article
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ISSN
1473-7167
EISSN
1744-8379
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https://www.tandfonline.com/doi/full/10.1080/14737167.2021.1859371