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罕见病药品预算影响分析研究进展
Development of Budget Impact Analysis of Orphan Drugs
归纳概括国内外相关文献,总结世界各国罕见病药品对药品预算影响。罕见病具有疾病种类多、单个病种发病率低、危害严重、诊断困难、有效干预和治疗药物少等特点。随着美国和欧盟等国家的立法,罕见病药品数量呈逐渐上升趋势。目前,美国已批准600余种罕见病药品,欧盟批准100余种罕见病药品。罕见病种类、接受治疗患者人数、批准上市罕见病药品数、罕见病药品治疗年费用和药品的可及性等是影响预算的主要因素。研究表明,随着罕见病药品批准数量的增加,罕见病药品占总药品费用比例呈缓慢上升趋势,同时,也存在国家和地区差异,但总体上对药品预算的影响较小且相对稳定;少数治疗肿瘤和代谢性疾病的罕见病药品对总药品费用的影响较大。
Budget impact analysis of drugs for rare diseases in different countries are reviewed. There are more than 7 000 kinds rare disease characterized by low incidence, serious influence on patients and society, difficulty in diagnosis, less effective intervention and therapeutic drugs. With the orphan drug legislation of the United States, the European Union and other countries, the number of drugs for rare diseases is increasing. At present, the United States has approved more than 600 drugs for rare diseases, and the European Union has approved more than 100 drugs for rare diseases. The types of rare diseases, the number of patients receiving treatment, the number of approved drugs for rare diseases, the annual cost of drug treatment for rare diseases and the availability of drugs are the main factors affecting the budget. The study shows that with the increase in the number of drugs approved for rare diseases, the proportion of drugs expenditure for rare diseases in the total drug expenses showed a slow rising trend, and there are national and regional differences. The overall budget impact of orphan drugs is small and fairly stable relative to total pharmaceutical expenditure. In addition, a small number of rare diseases drugs used to treat tumors and metabolic diseases have a greater impact on the total drug cost.
罕见病 / 罕见病药品 / 预算影响分析 / 价格 / 报销 {{custom_keyword}} /
rare diseases / orphan drugs / budget impact analysis / price / reimbursement {{custom_keyword}} /
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国家重点研发计划精准医学研究重点专项“罕见病临床队列研究”项目资助(2016YFC0901500);中国医学科学院医学与健康科技创新工程资助(2017-I2M-1-011)
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