VALUE AND UTILITIES AMONG EUROPEAN PATIENTS: A DECISION-ANALYTIC TOOL FOR MEASURING PATIENT PREFERENCES

Purpose:

There is a growing recognition by healthcare authorities and the pharmaceutical industry of the need to include preferences of patients within the development and drug approval process. Our study aims to test a decision tool that could be used among a representative sample of patients and eliminate the risk of relying on the non-representative voice of a single patient. 

Method:

A web-based field experiment was conducted among 62 multiple sclerosis (MS) patients to test the use of the Measuring Attractiveness by a Categorical Based Evaluation Technique (MACBETH) approach to collecting preferences. Preferences were elicited for outcomes that are possible with MS treatments, measured on three efficacy outcomes (number of relapses, time to disease progression, disease progression) and three safety outcomes (risk for PML, for liver failure, and for leukemia). Value functions were derived and logistic regression models were used to evaluate the relationship between value function shapes and several disease and demographic characteristics. 

Result:

Patients found the application easy to use and were able to provide consistent judgments indicating their most valued treatment outcome and how their values change. Fifty-eight percent of respondents indicated disease progression to be the most important treatment outcome. The majority of patient preferences for all the treatment outcomes were non- linear, meaning that they changed their values depending on the increases or decreases in the treatment outcomes. The linear or non-linear shape of patients’ value functions, indicating the preference for the treatment outcomes, were significantly predicted by a measure of disease severity.

Conclusion:

The decision analytic technique MACBETH utilizes a method that is theoretically sound and grounded in the principles of decision theory. This tool is flexible and can be easily integrated into a clinical trial or post-authorization setting and the results fed into the regulatory approval process. Regulatory authorities and the industry should consider the application of this decision analytic tool to increase their understanding of the preferences of patients and for inclusion of such data in their decision making processes.