Mosegui Gabriela Bittencourt Gonzalez, Antoñanzas Fernando
Universidade Federal Fluminense, Instituto de Saúde Coletiva, Departamento de Saúde e Sociedade Universidade Federal Fluminense, Instituto de Saúde Coletiva, Departamento de Saúde e Sociedade Niterói (RJ) Brazil Universidade Federal Fluminense, Instituto de Saúde Coletiva, Departamento de Saúde e Sociedade, Niterói (RJ) Brasil.
Universidad de La Rioja, Departamento de Economia e Empresa Universidad de La Rioja, Departamento de Economia e Empresa La Rioja Espanha Universidad de La Rioja, Departamento de Economia e Empresa, La Rioja, Espanha.
Rev Panam Salud Publica. 2019 Jul 8;43:e57. doi: 10.26633/RPSP.2019.57. eCollection 2019.
To describe and compare the regulatory framework governing policies on rapid/alternative access to medicines (expanded access and compassionate use) in South American countries.
An exploratory descriptive study with analysis of documents was performed. In addition to a literature review, official rules issued by regulatory agencies were reviewed if available. Information was collected on how countries define the concepts of expanded access and compassionate use, clinical phase in which the medicine becomes available for these alternative uses, and role of physicians and sponsors.
Argentina, Brazil, Chile, Peru, and Uruguay were included in the study. The information obtained revealed that the regulatory scenario is more structured in Argentina and Brazil than in the other countries. In Chile, rules on expanded access and compassionate use are available, however without an explicit definition of these concepts. In Peru and Uruguay, important definitions are missing regarding expanded access. The search did not reveal any databases with information on expanded access and compassionate use programs, supporting the notion that empirical data to evaluate the results of these policies are lacking.
All the countries analyzed have a regulatory framework that contemplates rapid/alternative access to medicines by patients at risk. However, databases and transparent information are lacking, preventing a snapshot of the medicines covered and patients benefiting from alternative access programs and assessments of these policies in South America.
描述并比较南美洲国家关于药品快速/替代获取政策（扩大获取和同情用药）的监管框架。
开展一项通过文件分析进行的探索性描述性研究。除文献综述外，还查阅了监管机构发布的官方规定（如有）。收集了有关各国如何定义扩大获取和同情用药概念、药品可用于这些替代用途的临床阶段以及医生和申办方作用的信息。
该研究纳入了阿根廷、巴西、智利、秘鲁和乌拉圭。所获信息表明，阿根廷和巴西的监管情况比其他国家更具结构性。在智利，有关于扩大获取和同情用药的规定，但未对这些概念进行明确界定。在秘鲁和乌拉圭，缺少关于扩大获取的重要定义。检索未发现任何包含扩大获取和同情用药项目信息的数据库，这支持了缺乏评估这些政策结果的实证数据这一观点。
所有分析的国家都有一个监管框架，考虑到了有风险患者对药品的快速/替代获取。然而，缺乏数据库和透明信息，无法了解南美洲替代获取项目所涵盖的药品情况、受益患者情况以及对这些政策的评估。
