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解锁药物可及性:泰国罕见病药物可及性的综合分析

Unlocking access: a comprehensive analysis of medicines accessibility for rare diseases in Thailand.

作者信息

Suwattanapreeda Siriwat, Hirunrassamee Sanita, Sooksriwong Chaoncin, Maluangnon Kusawadee, Chuachantra Thirapich, Kuchaisit Krissana, Osirisakul Niti

机构信息

Somdet Chaopraya Institute of Psychiatry, 112 Somdetchaopraya Street, Klongsarn, Bangkok, 10600, Thailand.

Drug Information and Consumer Protection Center, Center of Excellence in Pharmacy Practice and Management Research, Faculty of Pharmacy, Thammasat University, Khlong Luang District, Pathum Thani, Thailand.

出版信息

Orphanet J Rare Dis. 2025 May 28;20(1):258. doi: 10.1186/s13023-025-03754-9.

Abstract

INTRODUCTION

In Thailand, obtaining medicines for rare diseases presents significant challenges, with limited evidence highlighting these issues.

OBJECTIVES

To evaluate the accessibility of medicines and the extent of health insurance coverage for treatments of rare diseases in Thailand.

METHOD

This study utilized a thorough review of current health policies, drug registration database, and insurance coverage conditions. Additionally, procurement data from the Ministry of Finance was analyzed to verify the acquisition of medicines intended for the treatment of rare diseases.

RESULTS

A review of the availability and procurement of medicines for rare diseases in Thailand revealed considerable limitations in both registration and accessibility. According to the International Rare Diseases Research Consortium, only 46.80% of their recommended medicines were registered in Thailand, and of these, just 22.93% were included in the national essential medicines list. Additionally, a review of the state's pharmaceutical procurement dataset over the past 5 years showed that merely 31.70% of these registered drugs had been purchased from suppliers for use in hospitals.

CONCLUSION

To address these issues, the study recommended accelerating the approval process for rare disease medicines, expanding health insurance coverage, establishing financial support for patients, and creating a specific pricing policy for orphan drugs. Collaborative efforts among stakeholders were emphasized as crucial for improving access to essential medicines and enhancing treatment outcomes for patients with rare diseases in Thailand.

摘要

引言

在泰国,获取治疗罕见病的药物面临重大挑战,仅有有限的证据凸显了这些问题。

目的

评估泰国治疗罕见病药物的可及性以及医疗保险覆盖范围。

方法

本研究全面审查了现行卫生政策、药品注册数据库和保险覆盖条件。此外,还分析了财政部的采购数据,以核实用于治疗罕见病药物的采购情况。

结果

对泰国罕见病药物的可得性和采购情况进行审查后发现,在注册和可及性方面均存在相当大的限制。根据国际罕见病研究联盟的数据,其推荐的药物中只有46.80%在泰国注册,其中仅有22.93%被列入国家基本药物清单。此外,对过去5年国家药品采购数据集的审查表明,这些已注册药物中仅有31.70%从供应商处采购并用于医院。

结论

为解决这些问题,该研究建议加快罕见病药物的审批流程,扩大医疗保险覆盖范围,为患者建立财政支持,并制定孤儿药的特定定价政策。强调利益相关者之间的合作努力对于改善泰国基本药物的可及性和提高罕见病患者的治疗效果至关重要。

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