PURPOSE:

Real-world cost-effectiveness analyses (CEAs) that provide good estimates of health costs and outcomes associated with pharmaceuticals among patients in actual practice are particularly valuable for re-assessing values of pharmaceuticals from the post-marketing perspective.

This workshop will focus on building a real-world CEA for pharmaceuticals using glucose-lowering agents in diabetes as an example.

Participants will learn how to conduct real-world data (RWD)-based CEAs through applying science-based health economic theories, adopting advanced methodologies, and utilizing large-scale, multidimensional, individual-level, and longitudinal RWD, which all together ensure the validity and applicability of findings to local settings.

DESCRIPTION:

Attendees will obtain a working knowledge of RWD-based CEAs for pharmaceuticals.

The workshop will present a series of studies as a research framework to build RWD-based CEAs, including how to 1) identify a study cohort of comparable treatment groups for generating reliable real-world evidence on comparative treatment effectiveness/safety, 2) estimate country/setting-specific health cost and utility parameters for better reflecting patient characteristics in target clinical settings, and 3) conduct cohort study-based and model-based simulation studies for analyzing the long-term health and economic outcomes.

Dr. Ou will chair the session, and present the challenges for conducting RWD-based CEAs and introduce a framework for RWD-based CEAs (5 min.), Ms. Yang will show how to estimate treatment effectiveness/safety with rigorous methods and real-world population data (10 min.), Dr. Kuo will present how to assess country-specific health cost and utility estimates using RWD with adjustment for potential confounders (15 min.), and Mr. Peng will demonstrate how these real-world parameters can be input into study-based or risk equation-based modeling CEAs, and how these results can be provided for clinical and policy implications (15 min.). Audience participation will include a hypothetical case study for RWD-based CEAs (15 min.). This workshop will be valuable to researchers, clinicians, and industry analysts who are interested in RWD-based CEAs.