Orphan drug development: The impact of regulatory and reimbursement frameworks

被引：4

作者：

Huang, Yu-Jun ^{[1
]}

Chao, Wan-Yu ^{[1
,2
]}

Wang, Chi-Chuan ^{[1
,3
,4
]}

Chang, Lin-Chau ^{[1
]}

机构：

[1] Natl Taiwan Univ, Coll Med, Sch Pharm, 33 Linsen S Rd, Taipei City 10050, Taiwan

[2] Taiwan Food & Drug Adm, Minist Hlth & Welf, Div Med Prod, Sect New Drugs, Bldg F, 99 Lane 130,Sect 1,Acad Rd, Taipei City 11571, Taiwan

[3] Natl Taiwan Univ, Grad Inst Clin Pharm, Coll Med, 33 Linsen S Rd, Taipei City 10050, Taiwan

[4] Natl Taiwan Univ Hosp, Dept Pharm, 1 Changde St, Taipei City, Taiwan

来源：

DRUG DISCOVERY TODAY | 2022年 / 27卷 / 06期

关键词：

Orphan drugs; Rare diseases; Regulatory framework; Reimbursement; RARE DISEASES; ACCESS; COVERAGE;

D O I：

10.1016/j.drudis.2022.03.002

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

The enactment of orphan drug-specific legislation pioneered by the USA was subsequently followed by many regions, including the European Union (EU), Australia, Japan, and Taiwan. Here, we discuss the associated regulations established and their impacts in the aforementioned regions, which are among the first with frameworks specific for orphan drugs. Varied scopes of rare diseases or orphan drugs, diverse incentives, and heterogeneous types of reimbursement systems imply the prioritization of the agencies concerned. The numbers of designated and approved drugs reflect the impact of the regulatory and reimbursement frameworks. A comparison of the frameworks and their impact in the respective regions could provide valuable information for developing and improving related frameworks for countries worldwide.

引用

页码：1724 / 1732

页数：9

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