Data to be drawn from a descriptive, retrospective, cross sectional burden of illness study design developed by HCD Economics and University of Chester A peer-review manuscript published on 7th January 2021, in the journal ‘Expert Review of Pharmacoeconomics and Outcomes Research’ (impact factor 2.032), reports on the study rationale and protocol of the PICTURE study. PFIC Burden of Illness: Quantifying the global socio-economic burden The PICTURE study will employ a study design developed by HCD Economics and University of Chester in 2014. Founded on the principle of Public and Patient Involvement and Engagement (PPIE), the methodology has generated real-world evidence that can inform and support advocacy for clinical implementation of new therapies including rare diseases.

PICTURE recruits physicians, caregivers and, via caregivers, paediatric patients diagnosed with progressive familial intrahepatic cholestasis (PFIC). PFIC is a rare liver disorder that develops as a result of a recessive genetic defect in bile secretion. Cholestasis manifests as variable jaundice and extensive pruritus during infancy or early childhood. PFIC can lead to the development of significant liver fibrosis and end-stage liver disease (ESLD). Death or liver transplantation may occur from infancy to adolescence. The prevalence of PFIC is estimated at between 1 per 50,000 to 1 per 100,000 births.

The overall aim of the PICTURE study is to quantify the clinical, economic, and humanistic burden on PFIC patients, on their caregivers, and ultimately on society across the United States of America, France, Germany, and the United Kingdom. An additional goal of this study is to support the clinical implementation of new therapies emerging in the PFIC treatment landscape.

As with previous HCD Economics studies employing this methodology, the PICTURE study involves the establishment of an ad-hoc steering committee called the Expert Review Group (ERG), consisting of specialized clinicians and allied health professionals, academics, and patient advocacy representatives (Children’s Liver Disease Foundation (CLDF) and PFIC Advocacy and Resource Network). The role of the ERG in this study is to ensure quality standards are maintained and to provide overall study oversight. The ERG provides feedback at different stages of the project, including recommendations on the study design, approval of the protocol and all study materials and publication and communication of findings.

PICTURE will be the first and only international multicentre study to assess the burden of the disease in a cohort of PFIC patients from a societal perspective. Due to differences in the health system organization and financing results reported will include country-specific analysis that can be used to inform national health policies.

The full manuscript is available now Full article: Burden of illness of progressive familial intrahepatic cholestasis in the US, UK, France, and Germany: study rationale and protocol of the PICTURE study (

For more information on the PICTURE study please contact Sonia O’Hara

For more information on earlier and future studies that will employ the real-world evidence methodology employed for PICTURE please contact Alison Rose