Abstract

In recent years multi-criteria decision analysis (MCDA) has gained interest in healthcare, particularly as a potential methodology for assessing the value of orphan drugs. Several frameworks and methodologies have been proposed for developing a MCDA tool. However, there is no consensus on the weighting that each criterion should be allocated. This primary data collection study sought to obtain feedback on the weighting of criteria from clinicians, health economists, insurers, payers, patients and the public. A literature review was conducted to identify the criteria that are considered important in assessing the value of orphan drugs. The key criteria were used to develop an interactive, online tool with options to weight each criterion, with a total maximum of 100% for all weightings. Descriptions and definitions of the model and criteria were included. Once each respondent completed their options, the tool allowed respondents to compare their options with the pooled averages of prior respondents. This tool was shared electronically with a wide range of people involved in healthcare. Various analyses were performed. The 13 most commonly cited criteria in the literature review were included. Overall, results indicate that the 65 respondents considered that “Disease Severity” should be weighted highest of all criteria, with “Dynamic Efficiency” being considered the least important. By contrast, subset analysis suggests that government/health technology assessment agency staff considered that “Value for Money” should be weighted far more than any other criterion. Respondents from the pharmaceutical industry gave the most weight to “Disease Severity”. Clinical staff weighted “Disease Severity” and “Value for Money” almost the same, at 16% and 14.3%, respectively. Respondents from the USA and the UK weighted “Treatment Efficacy” at 21.6% and 10%, respectively. The large variation in the subset analyses demonstrate the need for reaching a consensus on the weighting of criteria in MCDA frameworks for orphan drugs.

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