OBJECTIVES:

This study aims to establish the value evaluation framework based on multi-criteria decision analysis for orphan drugs in China from the perspectives of stakeholders and the public, so as to provide evidence for the decision-making of medical insurance access.

METHODS:

Firstly，developed MCDA framework draft of orphan drugs based on literature research and EVIDEM framework. Secondly, we invited representatives from different domains to form a stakeholder group and collected expert opinions about the framework criteria and the scoring scale through the brain-storming and Delphi method. Meanwhile，an questionnaire survey of 70 sample people was conducted to determine the framework criteria and the scoring scale from the view of public. The five-point weighting method and two-step percentile distribution method were employed to weight the quantitative and qualitative criteria. Finally, based on the synthetization and comparison of all evidence, we established a MCDA framework with weight and score scale of each criterion, and send it to experts for verifying.

RESULTS:

From the perspective of stakeholders, We formed 11 quantitative criterion and 8 qualitative criterion. Through the two-step percentile distribution method, we got the weight of each criterion. The Kendall coordination coefficient of stakeholders on the weight allocation of each dimension is 0.37(p≤0.05), greater than 0.2, with statistical consistency of opinions. Compared with two-step percentile distribution method, the weight of each criterion through five-point weighting method was relatively similar and not practical. From the perspective of the public, the coefficient of variation for scoring importance was 0.3-0.6, indicating a consistent importance preference among respondents.

CONCLUSIONS:

Based on EVIDEM framework of MCDA, the study constructed the orphan drug value evaluation framework in China from two perspectives. But more factors should be taken into consideration for how to use it as a supplementary tool for decision-making of medical insurance access.