Discrepancies between multicriteria decision analysis-based ranking and intuitive ranking for pharmaceutical benefit-risk profiles in a hypothetical setting.

WHAT IS KNOWN AND OBJECTIVE: Multicriteria decision analysis (MCDA) has been generally considered a promising decision-making methodology for the assessment of drug benefit-risk profiles. There have been many discussions in both public and private sectors on its feasibility and applicability, but it has not been employed in official decision-makings. For the purpose of examining to what extent MCDA would reflect the first-hand, intuitive preference of evaluators in practical pharmaceutical assessments, we conducted a questionnaire survey involving the participation of employees of pharmaceutical companies.

METHODS: Showing profiles of the efficacy and safety of four hypothetical drugs, each respondent was asked to rank them following the standard MCDA process and then to rank them intuitively (i.e. without applying any analytical framework).

RESULTS AND DISCUSSION: These two approaches resulted in substantially different ranking patterns from the same individuals, and the concordance rate was surprisingly low (17%). Although many respondents intuitively showed a preference for mild, balanced risk-benefit profiles over profiles with a conspicuous advantage in either risk or benefit, the ranking orders based on MCDA scores did not reflect the intuitive preference.

WHAT IS NEW AND CONCLUSION: Observed discrepancies between the rankings seemed to be primarily attributed to the structural characteristics of MCDA, which assumes that evaluation on each benefit and risk component should have monotonic impact on final scores. It would be difficult for MCDA to reflect commonly observed non-monotonic preferences for risk and benefit profiles. Possible drawbacks of MCDA should be further investigated prior to the real-world application of its benefit-risk assessment.