International Experiences and Implications of the Value Assessment Case Study Based on the Medical Insurance Accessment of Ophthalmic Gene Therapy Products
- VernacularTitle:基于眼科基因治疗产品医保准入价值评估案例研究的国际经验与启示
- Author:
Dandan AI
1
;
Cheng'axin DUAN
;
Mo YU
Author Information
1. 北京市卫生经济学会 北京 100053
- Publication Type:Journal Article
- Keywords:
rare disease;
cell and gene therapy;
value evaluation;
ophthalmology
- From:
Chinese Health Economics
2025;44(10):105-108
- CountryChina
- Language:Chinese
-
Abstract:
Objective:By reviewing the experiences and challenges of assessing the value of ophthalmic gene therapy,it aimed to provide references for optimizing the reimbursement of this kind of drug.Methods:Based on case analysis,it summarized the key considerations and challenges faced by Health Technology Assessment(HTA)agencies conducting technical reviews of gene therapy for ophthalmic conditions in typical countries,including the US.,Canada,the UK.,France,and the Netherlands.Results:Disease severity and medical demand,drug safety,efficacy,cost-effectiveness,budget impact,and social value are the factors for HTA institutions to evaluate the value of ophthalmic gene therapy,patient participation and ethical fairness were taken into account by some institutions.HTA agencies generally put forward the following problems:large uncertainty of the long-term benefits,the difficulty of evaluating the value for caregivers,and the uncertainty of the discount rate.Providing methodological guidance for extrapolating clinical benefits of gene therapy,incorporating a wide range of value elements such as disease severity,and introducing innovative payment agreements are common responses.Conclusion:It is proposed to adjust the threshold for willingness to pay,consider the diversified value domains,gradually introducing finance-based or performance-based innovative payment models,and optimize value assessment and medical insurance access pathways.
