Multi-Criteria Decision Analysis for Benefit-Risk Analysis and Pharmaceutical Pricing

• Activity Number: 501
• Type: Contributed
• Date/Time: Wednesday, August 6, 2014 : 10:30 AM to 12:20 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Kao-Tai Tsai*+ and Bruce Dornseif
• Companies: and Celgene Corporation
• Keywords: Benefit Risk Analysis ; MCDA ; QALY ; Clinical Trials

Abstract

As a conventional practice, the National Institute for Health and Care Excellence (NICE) of the United Kingdom utilizes quality adjusted life years (QALY) for pharmaceutical pricing. Given the multivariate nature of benefit-risk of a drug, QALY is an effort to summarize the overall efficacy and safety effects using the estimated quality of life with somewhat subjective utility weighting. With the inherent weakness of quality of life data and subjectivity of the utility weighting, benefit and risk measure using QALY can be improved. Multi-Criteria Decision Analysis (MCDA) is a multivariate approach for decision analysis which can be implemented for benefit-risk analysis purposes. Even though MCDA takes into consideration selected efficacy and safety endpoints of interest, it primarily uses the presence or absence of the endpoint events and summarizes those for the study. Since the duration of an event has larger impact to a patient's well-being and societal cost, with respect to benefit-risk than just the status of presence, we propose an approach considering the duration, instead of only the status of events. For the endpoints in the MCDA, we estimate the mean durations and the covariance matrix for all endpoints so that statistical inference can be performed.