{"title":"罕见病孤儿药开发的个人观点:黄金机遇还是不可持续的未来?","authors":"Charles Oo, Lorraine M Rusch","doi":"10.1002/jcph.599","DOIUrl":null,"url":null,"abstract":"1. Tiwari J. Navigating through orphan medicinal product regulations in EU and US—similarities and differences. Regul Toxicol Pharmacol. 2015;71(1):63–67. 2. Tambuyzer E. Rare diseases, orphan drugs and their regulation: questions and misconceptions. Nat Rev Drug Discov. 2010;9(12): 921–929. 3. Kakkis ED, O’Donovan M, Cox G, et al. Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints. Orphanet J Rare Dis. 2015;10:16. doi:10.1186/s13023-014-0195-4 4. Gaddipati H, Liu K, Pariser A, Pazdur R. Rare cancer trial design: lessons from FDA approvals. Clin Cancer Res. 2012;18: 5172–5178. 5. Dupont AG, Van Wilder PB. Access to orphan drugs despite poor quality of clinical evidence. Br J Clin Pharmacol. 2011;71(4): 488–496. 6. Braun MM, Farag-El-Massah S, Xu K, Cot e TR. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nat Rev Drug Discov. 2010;9:519–522. 7. Meekings KN, Williams CSM, Arrowsmith JE. Orphan drug development: an economically viable strategy for biopharma R&D. Drug Discov Today. 2012;17(13–14):660–664. 8. Haffner ME, Whitley J, Moses M. Two decades of orphan product development. Nat Rev Drug Discov. 2002;1(10):821–825. 9. Orphan Drug Act. 21 CFR Part 316 Orphan Drugs. US Food Drug Admin. 2013;78(113):15–33. 10. Simoens S. Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis. 2011;6(1):42. 11. Bashaw ED, Huang S-M, Cot e TR, et al. Clinical pharmacology as a cornerstone of orphan drug development. Nat Rev Drug Discov. 2011;10(11):795–796. 12. Abrahamyan L, Diamond IR, Johnson SR, Feldman BM. A new toolkit for conducting clinical trials in rare disorders. J Popul Ther Clin Pharmacol. 2014;21(1):66–78. 13. Oo C, Tsai J-C, Kao HD. There is no better time than the present: nanotechnology as a disruptive innovation for drug development. Drug Discov Today. 2015;20(6):645–647. 14. Dunoyer M. Accelerating access to treatments for rare diseases. Nat Rev Drug Discov. 2011;10(7):475–476. Oo and Rusch 3 Drug Interactions","PeriodicalId":48908,"journal":{"name":"Journal of Clinical Pharmacology","volume":"56 3","pages":"257-9"},"PeriodicalIF":2.9000,"publicationDate":"2016-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1002/jcph.599","citationCount":"9","resultStr":"{\"title\":\"A personal perspective of orphan drug development for rare diseases: A golden opportunity or an unsustainable future?\",\"authors\":\"Charles Oo, Lorraine M Rusch\",\"doi\":\"10.1002/jcph.599\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"1. Tiwari J. Navigating through orphan medicinal product regulations in EU and US—similarities and differences. Regul Toxicol Pharmacol. 2015;71(1):63–67. 2. Tambuyzer E. Rare diseases, orphan drugs and their regulation: questions and misconceptions. Nat Rev Drug Discov. 2010;9(12): 921–929. 3. Kakkis ED, O’Donovan M, Cox G, et al. Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints. Orphanet J Rare Dis. 2015;10:16. doi:10.1186/s13023-014-0195-4 4. Gaddipati H, Liu K, Pariser A, Pazdur R. Rare cancer trial design: lessons from FDA approvals. Clin Cancer Res. 2012;18: 5172–5178. 5. Dupont AG, Van Wilder PB. Access to orphan drugs despite poor quality of clinical evidence. Br J Clin Pharmacol. 2011;71(4): 488–496. 6. Braun MM, Farag-El-Massah S, Xu K, Cot e TR. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nat Rev Drug Discov. 2010;9:519–522. 7. Meekings KN, Williams CSM, Arrowsmith JE. Orphan drug development: an economically viable strategy for biopharma R&D. Drug Discov Today. 2012;17(13–14):660–664. 8. Haffner ME, Whitley J, Moses M. Two decades of orphan product development. Nat Rev Drug Discov. 2002;1(10):821–825. 9. Orphan Drug Act. 21 CFR Part 316 Orphan Drugs. US Food Drug Admin. 2013;78(113):15–33. 10. Simoens S. Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis. 2011;6(1):42. 11. Bashaw ED, Huang S-M, Cot e TR, et al. Clinical pharmacology as a cornerstone of orphan drug development. Nat Rev Drug Discov. 2011;10(11):795–796. 12. Abrahamyan L, Diamond IR, Johnson SR, Feldman BM. A new toolkit for conducting clinical trials in rare disorders. J Popul Ther Clin Pharmacol. 2014;21(1):66–78. 13. Oo C, Tsai J-C, Kao HD. There is no better time than the present: nanotechnology as a disruptive innovation for drug development. Drug Discov Today. 2015;20(6):645–647. 14. Dunoyer M. Accelerating access to treatments for rare diseases. Nat Rev Drug Discov. 2011;10(7):475–476. 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A personal perspective of orphan drug development for rare diseases: A golden opportunity or an unsustainable future?
1. Tiwari J. Navigating through orphan medicinal product regulations in EU and US—similarities and differences. Regul Toxicol Pharmacol. 2015;71(1):63–67. 2. Tambuyzer E. Rare diseases, orphan drugs and their regulation: questions and misconceptions. Nat Rev Drug Discov. 2010;9(12): 921–929. 3. Kakkis ED, O’Donovan M, Cox G, et al. Recommendations for the development of rare disease drugs using the accelerated approval pathway and for qualifying biomarkers as primary endpoints. Orphanet J Rare Dis. 2015;10:16. doi:10.1186/s13023-014-0195-4 4. Gaddipati H, Liu K, Pariser A, Pazdur R. Rare cancer trial design: lessons from FDA approvals. Clin Cancer Res. 2012;18: 5172–5178. 5. Dupont AG, Van Wilder PB. Access to orphan drugs despite poor quality of clinical evidence. Br J Clin Pharmacol. 2011;71(4): 488–496. 6. Braun MM, Farag-El-Massah S, Xu K, Cot e TR. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nat Rev Drug Discov. 2010;9:519–522. 7. Meekings KN, Williams CSM, Arrowsmith JE. Orphan drug development: an economically viable strategy for biopharma R&D. Drug Discov Today. 2012;17(13–14):660–664. 8. Haffner ME, Whitley J, Moses M. Two decades of orphan product development. Nat Rev Drug Discov. 2002;1(10):821–825. 9. Orphan Drug Act. 21 CFR Part 316 Orphan Drugs. US Food Drug Admin. 2013;78(113):15–33. 10. Simoens S. Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis. 2011;6(1):42. 11. Bashaw ED, Huang S-M, Cot e TR, et al. Clinical pharmacology as a cornerstone of orphan drug development. Nat Rev Drug Discov. 2011;10(11):795–796. 12. Abrahamyan L, Diamond IR, Johnson SR, Feldman BM. A new toolkit for conducting clinical trials in rare disorders. J Popul Ther Clin Pharmacol. 2014;21(1):66–78. 13. Oo C, Tsai J-C, Kao HD. There is no better time than the present: nanotechnology as a disruptive innovation for drug development. Drug Discov Today. 2015;20(6):645–647. 14. Dunoyer M. Accelerating access to treatments for rare diseases. Nat Rev Drug Discov. 2011;10(7):475–476. Oo and Rusch 3 Drug Interactions
期刊介绍:
The Journal of Clinical Pharmacology (JCP) is a Human Pharmacology journal designed to provide physicians, pharmacists, research scientists, regulatory scientists, drug developers and academic colleagues a forum to present research in all aspects of Clinical Pharmacology. This includes original research in pharmacokinetics, pharmacogenetics/pharmacogenomics, pharmacometrics, physiologic based pharmacokinetic modeling, drug interactions, therapeutic drug monitoring, regulatory sciences (including unique methods of data analysis), special population studies, drug development, pharmacovigilance, womens’ health, pediatric pharmacology, and pharmacodynamics. Additionally, JCP publishes review articles, commentaries and educational manuscripts. The Journal also serves as an instrument to disseminate Public Policy statements from the American College of Clinical Pharmacology.