Establishment of a value assessment framework for orphan medicinal products in China.

Background: This study aimed to establish a suitable value assessment framework for orphan medicinal products in China based on the multi-criteria decision analysis (MCDA) method.

Methods: First, 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.

Results: Combined 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.

Conclusions: MCDA 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.