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

共 50 条

[1] ORPHAN DRUG REIMBURSEMENT IN EUROPE: DO LESS STRINGENT REGULATORY REQUIREMENTS TRANSLATE INTO LESS FAVOURABLE REIMBURSEMENT?
Anastasaki, E.
Macaulay, R.
[J]. VALUE IN HEALTH, 2017, 20 (09) : A464 - A464
[2] Challenges in orphan drug development and regulatory policy in China
Cheng, Alice
Xie, Zhi
[J]. ORPHANET JOURNAL OF RARE DISEASES, 2017, 12 : 1 - 8
[3] Challenges in orphan drug development and regulatory policy in China
Alice Cheng
Zhi Xie
[J]. Orphanet Journal of Rare Diseases, 12
[4] Impact of the EU Orphan Drug Regulation on the Development of Orphan Drug: A 15-Year Analysis
Pan, Irene
[J]. PHARMACOEPIDEMIOLOGY AND DRUG SAFETY, 2016, 25 : 397 - 398
[5] THE EXPENDITURE AND REIMBURSEMENT OF ORPHAN DRUG IN TAIWAN: CHALLENGES AND OPPORTUNITIES
Chiang, P.
Yang, W.
[J]. VALUE IN HEALTH, 2016, 19 (03) : A255 - A255
[6] Orphan Drug Pricing and Reimbursement Policies Lacking in EU
不详
[J]. PHARMACEUTICAL MEDICINE, 2018, 32 (04) : 297 - 298
[7] ORPHAN DRUG REIMBURSEMENT IN ENGLAND AND IMPLICATIONS FOR PATIENT ACCESS
Walker, S.
Lilley, H.
Payne, R.
Fountain, D.
[J]. VALUE IN HEALTH, 2019, 22 : S859 - S859
[8] A systematic review of moral reasons on orphan drug reimbursement
Zimmermann, Bettina M.
Eichinger, Johanna
Baumgartner, Matthias R.
[J]. ORPHANET JOURNAL OF RARE DISEASES, 2021, 16 (01)
[9] Improvement of Pediatric Drug Development: Regulatory and Practical Frameworks
Tsukamoto, Katusra
Carroll, Kelly A.
Onishi, Taku
Matsumaru, Naoki
Brasseur, Daniel
Nakamura, Hidefumi
[J]. CLINICAL THERAPEUTICS, 2016, 38 (03) : 574 - 581
[10] A systematic review of moral reasons on orphan drug reimbursement
Bettina M. Zimmermann
Johanna Eichinger
Matthias R. Baumgartner
[J]. Orphanet Journal of Rare Diseases, 16

← 1 2 3 4 5 →