Value evaluation by multi-criteria decision analysis for orphan medicinal product

A study published in Pharmacoeconomics and Policy provides one of the first empirical tests of a structured multi-criteria decision analysis (MCDA) framework to evaluate OMPs within the context of China's health insurance system. The key finding is that this transparent, multi-dimensional method can work in practice, offering a potential solution to a major challenge in healthcare policy. Overall, the empirical study successfully translates the MCDA framework into a practical tool, offering a more transparent and multi-faceted way to evaluate high-impact drugs for rare diseases within reimbursement systems.

Core findings and context

Applying the framework to three orphan medicinal products (OMPs), laronidase, emicizumab, and dimethyl fumarate (DMF) produced highly consistent quantitative scores between independent assessor groups. Laronidase scored the highest, followed by emicizumab, then DMF. This is significant because traditional health technology assessment (HTA) often struggles with OMPs due to high costs and limited data. As the authors note, MCDA helps shift decision-making "from 'black box operation' to 'transparent value selection’, and adds structured deliberation on crucial factors like unmet need and disease severity, which are hard to capture in standard cost-effectiveness analyses.

Notable methods and insights

A methodological strength was the use of a separate stakeholder expert group for independent scoring, validating the framework's consistency. An interesting finding was the divergence between results: while quantitative scoring was consistent, assessments on qualitative criteria (e.g., policy alignment) varied. This highlights that MCDA provides structure but still requires deliberation for value-based judgments.

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Contact the author: Ming Hu, Center for Drug Policy and Pharmacoeconomics Research, West China School of Pharmacy, Sichuan University, Chengdu, 610041, China. huming@scu.edu.cn

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