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Abstract：
This article aims to synthesize the existing literature on the implementation of public policies to incentivize the development of treatments for rare diseases, (diseases with very low prevalence and therefore with low commercial interest) otherwise known as orphan drugs. The implementation of these incentives in the United States (US), Japan, and in the European Union (EU) seems to be related to a substantial increase in treatments for these diseases, and has influenced the way the pharmaceutical research & development (R&D) system operates beyond this policy area. Despite the success of the Orphan Drug model, the academic literature also highlights the negative implications that these public policies have on affordability and access to orphan drugs, as well as on the prioritization of certain disease rare areas over others. The synthesis focuses mostly on the United States\' Orphan Drug Act (ODA) as a model for subsequent policies in other regions and countries. It starts with a historical overview of the creation of the term \"rare diseases\", continues with a summary of the evidence available on the US ODA\'s positive and negative impacts, and provides a summary of the different proposals to reform these incentives in light of the negative outcomes described. Finally, it describes some key aspects of the Japanese and European policies, as well as some of the challenges captured in the literature related to their impact in Low- and Middle-Income Countries (LMICs).
摘要：
本文旨在综合现有的有关公共政策实施的文献，以激励罕见病治疗的发展，（患病率很低，因此商业利益很低的疾病）也称为孤儿药。在美国（US）实施这些激励措施，Japan,在欧盟（EU）似乎与这些疾病的治疗方法大幅增加有关，并影响了药物研发（R＆D）系统在该政策领域之外的运作方式。尽管孤儿药模型取得了成功,学术文献还强调了这些公共政策对孤儿药的可负担性和可获得性的负面影响，以及某些疾病罕见地区优先于其他地区。该综述主要集中在美国的孤儿药物法案（ODA）上，作为其他地区和国家后续政策的典范。它从“罕见疾病”一词的产生的历史概述开始，继续总结美国官方发展援助的积极和消极影响的现有证据，并根据所描述的负面结果，总结了改革这些激励措施的不同建议。最后,它描述了日本和欧洲政策的一些关键方面，以及文献中捕捉到的一些与它们在低收入和中等收入国家(LMICs)影响相关的挑战。
