Effectiveness Research in the Evolving HCV Landscape

Excerpt

As antiviral therapy for chronic hepatitis C virus (HCV) infection continues to evolve, understanding the effectiveness of antiviral regimens is critical for making informed treatment decisions for HCV management. While the terms “efficacy” and “effectiveness” are often used interchangeably, they hold different meanings: Efficacy is a measure of the capacity of a treatment to produce the desired effect in a controlled environment, such as in a randomized controlled trial whereas effectiveness describes the actual effect of the treatment in practice [1]. In HCV antiviral clinical trials, like other clinical trials, patients are screened using stringent predetermined criteria, closely monitored, and any treatment deviations must follow detailed protocols. As such, information provided in traditional randomized clinical trials is often limited when applied to clinical practice where variation of patient characteristics, setting, care coordination, and management cannot be controlled. These elements are likely to influence the magnitude of the effect in practice depending on who is treated, how they are treated, which conditions are being treated and other nuances. Effectiveness studies, which examine effects (outcomes) in actual practice, provide practical information to address the “where,” “how,” and “for whom” a treatment might be recommended depending on the questions addressed, patient population, groups compared, setting, and measured outcomes. Though the randomized controlled trial is often held up as the “gold standard” for medical decision making, truly informed decision making requires information obtained from clinical practice. …