0000000000650202
AUTHOR
Leonardo Ruiz-casas
Burden of illness of progressive familial intrahepatic cholestasis in the US, UK, France, and Germany: study rationale and protocol of the PICTURE study
Background: 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. Methods: 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 physi…
The Cost of Non-Alcoholic Steatohepatitis (NASH) in Europe and USA: The GAIN Study
Background: Non-alcoholic fatty liver disease (NAFLD) is one of the most common chronic liver disorders. The progressive form of NAFLD, non-alcoholic steatohepatitis (NASH), leads to cirrhosis in a proportion of patients and so is associated with a substantial socio-economic burden which, coupled with a steadily rising prevalence, is becoming a growing public health challenge. The Global Assessment of the Impact of NASH (GAIN) study is a socioeconomic, prevalence-based burden of illness study across Europe (France, Germany, Italy, Spain and the United Kingdom) and the United States to determine the socio-economic burden of NASH in the real-world. Methods: GAIN was a retrospective, cross-sec…