請用此 Handle URI 來引用此文件：
http://tdr.lib.ntu.edu.tw/jspui/handle/123456789/95044完整後設資料紀錄
| DC 欄位 | 值 | 語言 |
|---|---|---|
| dc.contributor.advisor | 王繼娟 | zh_TW |
| dc.contributor.advisor | Chi-Chuan Wang | en |
| dc.contributor.author | 趙婉妤 | zh_TW |
| dc.contributor.author | Wan-Yu Chao | en |
| dc.date.accessioned | 2024-08-26T16:24:54Z | - |
| dc.date.available | 2024-08-27 | - |
| dc.date.copyright | 2024-08-26 | - |
| dc.date.issued | 2024 | - |
| dc.date.submitted | 2024-08-07 | - |
| dc.identifier.citation | Orphan Drug Act of 1983. Public Law. No. 97–414.
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| dc.identifier.uri | http://tdr.lib.ntu.edu.tw/jspui/handle/123456789/95044 | - |
| dc.description.abstract | 罕藥是指適應症用於預防、診斷、治療罕見疾病的藥物。目前，已有許多國家制定罕藥相關的法規或政策，以鼓勵此類藥物的研發、製造和供應，而臺灣也不例外。本研究的首要目的為綜整和比較美國、歐盟、日本、澳洲和臺灣等五個國家/地區的罕藥法規環境，描述了罕藥認定的標準、上市許可、市場專屬期及其他的獎勵措施、健保給付與核價方法。另外，我們也對五個研究國家/地區近年來核准罕藥情形進行了分析與比較，以評估罕藥法規的有效性並呈現罕藥的研發趨勢。此外，為了反映臺灣罕藥的使用現況並探討未滿足的醫療需求，我們檢索並分析了官方網站的罕見疾病患者人數、罕藥使用量、罕藥使用人數等資訊和數據。根據我們的研究，目前仍有許多罕見疾病沒有合適的治療方法。儘管已向罕藥研發廠商提供了許多獎勵措施，但未滿足的醫療需求仍然存在。可能還需要其他獎勵措施來更進一步支持罕藥的研發，也需要法規單位、藥業和學術界等利益關係人之間的合作，以提升罕藥的可用性和可近性。我們的研究可以協助提供未來政策制定之見解。 | zh_TW |
| dc.description.abstract | Orphan drugs are drugs indicated for the prevention, diagnosis, and treatment of rare diseases. To date, orphan drug-related regulations or policies have been established in several countries to encourage research and development, manufacturing, and supply of such drugs, and Taiwan is no exception. The main objective of the present study was to summarize and compare the regulatory environments for orphan drugs in five countries/regions, including the United Sates, European Union, Japan, Australia, and Taiwan. The criteria for orphan drug designation, marketing authorization, market exclusivity and other incentives, reimbursement and pricing methods were described. Besides, we analyzed and compared the orphan drug approval in the five investigated countries/regions in recent years to evaluate the effectiveness of orphan drug regulations and reveal the development trend for orphan drugs. Furthermore, in order to reflect the current status of orphan drug usage and explore the unmet medical needs in Taiwan, we retrieved and analyzed the information and statistics from official websites regarding the number of patients with rare diseases, amount of orphan drug usage and number of users. According to our study, there are still many rare diseases without available treatments at present. Although several incentives have been offered to orphan drug sponsors, unmet medical needs remain. Other incentives or measures may be required to support further development of orphan drugs. Collaboration among relevant stakeholders including regulatory authorities, pharmaceutical industry, and academia is also needed to enhance orphan drug availability and accessibility. Our study could provide insights in assisting future policy making. | en |
| dc.description.provenance | Submitted by admin ntu (admin@lib.ntu.edu.tw) on 2024-08-26T16:24:54Z No. of bitstreams: 0 | en |
| dc.description.provenance | Made available in DSpace on 2024-08-26T16:24:54Z (GMT). No. of bitstreams: 0 | en |
| dc.description.tableofcontents | 口試委員會審定書 i
誌謝 ii 中文摘要 iii Abstract iv CHAPTER 1: INTRODUCTION 1 1.1 Rare Diseases 1 1.2 Orphan Drugs and Orphan Drug Regulations 4 1.3 Motivation and Purposes of the Present Study 7 CHAPTER 2: METHODS 9 2.1 Study Design and Purposes 9 2.2 Data Sources 15 2.3 Analysis 17 CHAPTER 3: RESULTS 21 3.1 Regulatory Environments for Orphan Drugs in Five Countries/Regions 21 3.1.1 Orphan Drug Designation and Marketing Authorization 21 3.1.2 Market Exclusivity and Other Incentives 33 3.1.3 Pricing and Reimbursement 46 3.1.4 Recent Updated Regulations 57 3.2 Regulatory Approval in Five Countries/Regions - Analysis of Orphan Drug Approval in Recent Years 61 3.3 Unmet Needs in Taiwan 68 3.3.1 Number of Patients Reported with Rare Diseases in Taiwan 68 3.3.2 Amount of Orphan Drug Usage and Number of Users in Taiwan 71 CHAPTER 4: DISCUSSION 78 4.1 Exploring and Combating the Unmet Medical Needs 78 4.2 Suggestions for Improvement 82 4.2.1 Other Feasible Incentives 82 4.2.2 Reliance, Review Collaboration, and Regulatory Harmonization 85 4.3 Strengths and Limitations 89 CHAPTER 5: CONCLUSIONS 91 REFERENCES 92 | - |
| dc.language.iso | en | - |
| dc.title | 臺灣的罕藥法規環境：獨特性、現況及未滿足的需求 | zh_TW |
| dc.title | Regulatory Environments for Orphan Drugs in Taiwan: The Uniqueness, the Current Status, and the Unmet Needs | en |
| dc.type | Thesis | - |
| dc.date.schoolyear | 112-2 | - |
| dc.description.degree | 碩士 | - |
| dc.contributor.oralexamcommittee | 張琳巧;黃文鴻 | zh_TW |
| dc.contributor.oralexamcommittee | Lin-Chau Chang;Weng-Foung Huang | en |
| dc.subject.keyword | 獎勵措施,罕藥,罕見疾病,法規與政策,臺灣,未滿足的醫療需求, | zh_TW |
| dc.subject.keyword | Incentives,Orphan drugs,Rare diseases,Regulations and policies,Taiwan,Unmet medical needs, | en |
| dc.relation.page | 103 | - |
| dc.identifier.doi | 10.6342/NTU202403939 | - |
| dc.rights.note | 未授權 | - |
| dc.date.accepted | 2024-08-08 | - |
| dc.contributor.author-college | 醫學院 | - |
| dc.contributor.author-dept | 臨床藥學研究所 | - |
| 顯示於系所單位： | 臨床藥學研究所 | |
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| ntu-112-2.pdf 目前未授權公開取用 | 1.81 MB | Adobe PDF |
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