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推动拉丁美洲的罕见病政策:行动呼吁。

Advancing rare disease policy in Latin America: a call to action.

作者信息

Wainstock Daniel, Katz Amiel

机构信息

PUC-Rio, Rio de Janeiro, RJ, Brazil.

Harvard University, Cambridge, MA, USA.

出版信息

Lancet Reg Health Am. 2023 Jan 27;18:100434. doi: 10.1016/j.lana.2023.100434. eCollection 2023 Feb.

Abstract

People living with a rare disease are amongst the most vulnerable groups in society. They have been historically marginalised and systematically stigmatised. It is estimated that 300 million people worldwide live with a rare disease. Despite that, many countries today, especially in Latin America, still lack consideration of rare diseases in public policies and national laws. Based on interviews with patient advocacy groups in Latin America, we aim to provide recommendations for lawmakers and policymakers in Brazil, Peru, and Colombia on how to improve public policies and national legislation for persons living with rare diseases in these three countries.

摘要

患有罕见病的人是社会中最弱势群体之一。他们在历史上一直被边缘化,并受到系统性的污名化。据估计,全球有3亿人患有罕见病。尽管如此,如今许多国家,尤其是拉丁美洲的国家,在公共政策和国家法律中仍未对罕见病给予关注。基于对拉丁美洲患者权益倡导组织的访谈,我们旨在就如何改善巴西、秘鲁和哥伦比亚这三个国家针对罕见病患者的公共政策和国家立法,为这三个国家的立法者和政策制定者提供建议。

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2
A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries.
Front Public Health. 2020 Jan 28;7:416. doi: 10.3389/fpubh.2019.00416. eCollection 2019.
3
A civil society view of rare disease public policy in six Latin American countries.
Orphanet J Rare Dis. 2020 Feb 27;15(1):60. doi: 10.1186/s13023-020-1314-z.
4
Spotlight on rare diseases.
Lancet Diabetes Endocrinol. 2019 Feb;7(2):75. doi: 10.1016/S2213-8587(19)30006-3.
5
A compilation of national plans, policies and government actions for rare diseases in 23 countries.
Intractable Rare Dis Res. 2018 Nov;7(4):213-222. doi: 10.5582/irdr.2018.01085.
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10
Pricing and reimbursement of orphan drugs: the need for more transparency.
Orphanet J Rare Dis. 2011 Jun 17;6:42. doi: 10.1186/1750-1172-6-42.

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