罕见的疾病有各种类型,发病率低,复杂的条件,而且往往难以诊断。由于中国人口众多,有相当数量的罕见疾病患者,但是孤儿药短缺。因此,这些患者经常发现自己处于无法获得或负担不起必要药物的情况。为了解决这一迫切的临床需求,中国实施了一系列孤儿药政策,旨在提高药物的可及性和可负担性。在药物可及性方面,鼓励公司通过实施税收优惠来加快药物开发,罕见病临床研究指导,并提供6年的数据保护期,市场独占期最长为7年。此外,临床试验豁免,接受海外临床试验数据,并建立了优先考虑海外临床紧急新药的清单,以加快药品注册申请,review,检查,和审批流程。在药物负担能力方面,罕见病药物的进口增值税已下调3%,各省市建立了代表性的罕见病保护模式,其中包括专项资金,医疗援助计划,和严重疾病保险。国家医保目录进行了调整,减轻了罕见病患者的经济负担,导致截至2024年3月,目录涵盖的孤儿药数量增加到95。通过比较美国的孤儿药政策,欧洲联盟,Japan,澳大利亚,和其他国家(或地区),为进一步完善我国孤儿药政策提供相关建议,从而为罕见疾病患者带来更多的治疗选择和希望。
Rare diseases have various types, low incidence rates, complex conditions, and are often difficult to diagnose. Due to China\'s large population, there is a significant number of rare disease patients, but there is a shortage of orphan drugs. Consequently, these patients often find themselves in a situation where necessary medications are either unavailable or unaffordable. To address this urgent clinical need, China has implemented a series of orphan drug
policies aimed at improving drug accessibility and affordability. In terms of drug accessibility, companies are encouraged to expedite drug development through the implementation of tax incentives, guidance for clinical research on rare diseases, and the provision of data protection periods of 6 years, along with market exclusivity periods limited to a maximum of 7 years. Moreover, exemptions for clinical trials, acceptance of overseas clinical trial data, and the creation of a list prioritizing clinically urgent new drugs from overseas have been introduced to expedite the drug registration application, review, inspection, and approval processes. In terms of drug affordability, the import value-added tax on rare disease drugs has been reduced by 3%, and various provinces and cities have established a representative rare disease protection model, which includes special funds, medical assistance programs, and serious disease insurance. The national medical insurance catalog has been adjusted to reduce the financial burden on rare disease patients, resulting in an increase in the number of orphan drugs covered by the catalog to 95 as of March 2024. By comparing orphan drug
policies in the United States, the European Union, Japan, Australia, and other countries (or regions), we will provide relevant suggestions to further improve orphan drug
policies in China, thus bringing more treatment options and hope to patients with rare diseases.