Every Patient Matters: Introduction to Multi-Dimensional Thresholding in Health Preference Research

PURPOSE: Patient preferences are routinely used to inform decisions throughout medical product development, such as regulatory approval and reimbursement. While discrete choice experiments have become the gold standard for preference elicitation, they suffer from low precisions in small samples and may fail at the individual level. This limits their application in rare diseases and in measuring preference heterogeneity in smaller subgroups. To overcome these limitations, multi-dimensional thresholding was developed as part of the European Medicines Agency (EMA) study on treatment preferences of patients with multiple myeloma. This workshop introduces the audience to the use of multi-dimensional thresholding in health preference research using the EMA patient preference study and an interactive exercise.

DESCRIPTION: Dr Ho will open the workshop by briefly discussing how patient preference information is used throughout medical product development. Dr Postmus will introduce the EMA multiple myeloma study and discuss example uses of thresholding technique for preference elicitation at the EMA and the US Food and Drug Administration (FDA). Dr Heidenreich will present on how to design multi-dimensional thresholding instruments and demonstrate their robustness with results from computational experiments. Dr Tervonen will discuss data analysis and conduct a live preference elicitation with workshop participants using their mobile devices. The results will be presented during the session and compared with results of N=560 multiple myeloma patients from the EMA study. HEOR and regulatory affairs scientists benefit from attending this introductory workshop.