ArticleOrphanet journal of rare diseases2024
Establishment of a value assessment framework for orphan medicinal products in China.
Article in Orphanet journal of rare diseases, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
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Who cites it
3 citing papers in PubMed.
- The impact of China's drug regulatory reform on access to orphan drugs: a cross-sectional study of diseases listed in the rare disease catalog.Frontiers in pharmacology · 2026Article
- Reforming China's Rare Disease Security System: Risk Management Perspectives and a Dedicated Insurance Innovation.Healthcare (Basel, Switzerland) · 2025Article
- Establishment of a value assessment framework for orphan drugs in China: an application of the discrete choice experiment in multicriteria decision analysis.Frontiers in pharmacology · 2025Article
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Authors and funding
4 authors.
Funding
Abstract
backgroundThis study aimed to establish a suitable value assessment framework for orphan medicinal products in China based on the multi-criteria decision analysis (MCDA) method.
methodsFirst, a draft framework of the MCDA criteria was built based on a systematic literature evaluation and the EVIDEM framework tools. Second, stakeholder groups were formed and expert opinions were collected through the brainstorming and expert consultation methods. Third, from the perspective of stakeholders, the five-point weighting method and a two-step percentile distribution method were employed to weigh the quantitative criteria in the framework for orphan drug value evaluation. Meanwhile, from the public perspective, a survey was conducted on a sample of 71 people to obtain the scoring scale of the framework for orphan drugs through a two-step percentile distribution method. Finally, based on the synthetization and comparison of all evidence and methods, we developed the framework criteria and scoring scale for the orphan medicinal products.
resultsCombined with the stakeholder selection and suggestions in the stakeholder workshop, the framework criteria for the evaluation were constructed based on China's national conditions, which included 11 quantitative and 8 qualitative criteria. The two-step percentile distribution method was selected as the weighting method.
conclusionsMCDA is feasible for the value assessment of orphan drugs in China and can be used as a supplementary tool for drug access decisions in medical insurance. It is suggested to further improve the value assessment framework of orphan medicinal products, scientifically evaluate the MCDA framework weighting method, explore a framework system suitable for China's national conditions.
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