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Patient Reported Outcomes and Its Place in NASH Research and Drug Development

Patient Reported Outcomes and Its Place in NASH Research and Drug Development

22 Aug 2021 12:24 12:36
(12 mins)
Zobair Younossi Speaker
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NAFLD is a common cause of chronic liver disease (CLD) associated with significant clinical and economic burden. NAFLD patients experience significant impairment of their health-related quality of life (HRQL) and other patient-reported outcomes (PROs). PRO is defined as “a measurement based on a report that comes directly from the patient about the status of a patient’s health without amendment or interpretation of the patient’s response by a clinician or anyone else”. PRO assessment provides perspective that is important for patients. In NASH, PROs capture comprehensive burden of NASH and impact of NASH treatment on patients’ experience. Finally, PROs are important from health policy perspective. PROs cannot be measured directly, they are assessed using validated PRO instruments such as CLD Questionnaire and its HCV, NASH and HBV versions (CLDQ, CLDQ-HCV, CLDQ-NASH and CLDQ-HBV). Other PRO tools used in liver disease include generic Short Form 36, health utilities and work productivity measures. Clinically, PROs can be surrogates of patients’ experience with NASH. In the clinical trial of NASH, inclusion of PROs as endpoints provides a better assessment of both the efficacy and the side effect profile of drug regimens. A number of PRO studies have indicated patients with NASH have significant impairment of HRQL which worsen with more advanced hepatic fibrosis and worsening severity of liver disease. In fact, NAFLD is not an asymptomatic disease with majority of patients reporting fatigue and other PRO impairment. There is some evidence that improvement of hepatic fibrosis and potentially other disease biomarkers in clinical trials of NASH can lead to improvement of PRO scores. Inclusion of a validated PRO instrument (CLDQ-NASH) in clinical trials of NASH will be important. Health utilities are important to adjust clinical outcomes (life years gained) with quality of life (QALYs) in cost-effectiveness analysis of different drugs and interventions for NASH.

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