OBJECTIVES: Specific clinical outcome indicators (COIs) able to evaluate the whole cycle of care are needed to assist both clinicians and administrators in improving quality and value of care. The Value-based Medicine in Hepatology study objective was to develop and test a set of COIs for major liver conditions, including chronic HCV and its progression to cirrhosis and liver cancer.

METHODS: In 2010, using a modified Delphi method and a RAND 9-point appropriateness scale, a panel of experts identified a set of COIs able to synthesize HCV outcomes in early and advance stage. The identified COIs were tested in an observational study involving the liver units of three major health care centers located in Lombardy.

RESULTS: The expert panel identified 10 COIs (e.g. Sustained virological response (SVR), decompensation rate, mortality or liver transplantation rate), and 5 COIs were added after the introduction of Directly Acting Antivirals (DAAs) treatment. In 2011-2012, 1533 HCV patients were enrolled and followed-up for a median time of 3.74 years. The COIs proved to be applicable in clinical practice and their values compared well with the available natural history studies. They also reported a good performance in assessing the impact of a treatment revolution as DAAs in HCV. For example, after DAAs introduction more than 35.0% of all HCV patients reported a 2 years-SVR as compared to 3.8% before. Among cirrhotic patients, this difference was even higher with almost 45.0% of 2 years-SVR after DAAs as compared to almost 1.0% before.

CONCLUSIONS: A comprehensive set of COIs was designed to sequentially capture clinical outcome at different stages of the natural history of HCV. These indicators represent a critical tool to implement a value-based approach in hepatology and to compare referral centers, to perform health technology assessment and to guide decision-making process for health authorities.